An international investigator initiated trial to capture different approaches in the treatment of Haemophilia A Patients With FVIII Inhibitors

2024-516741-39-00 Protocol MOTIVATE Therapeutic use (Phase IV) Ongoing, recruiting

Start 14 Jan 2020 · Status Ongoing, recruiting · 7 EU/EEA countries · 14 sites · Protocol MOTIVATE

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)
Status Ongoing, recruiting
Participants planned 123
Countries 7
Sites 14

Haemophilia A

The primary objective for Groups 1 and 2 is to evaluate ITI outcomes as determined by achievement of the following ITI criteria: 1. Inhibitor titre < 0.6 Bethesda units (BU)/mL for at least 2 consecutive measurements 2. FVIII recovery ≥ 66% of the predefined reference value of 1.5% international units (IU)/kg body weig…

Key facts

Sponsor
HZRM Haemophilie-Zentrum Rhein Main GmbH, Emory University
Participant type
Pediatric, Patients
Age range
0-17 years, 18-64 years, 65+ years
Gender
Male
Therapeutic area
Diseases [C] - Hemic and Lymphatic Diseases [C15]
Trial duration
14 Jan 2020 → ongoing
Decision date (initial)
2025-10-28
Transition trial
Yes
Low-intervention
Yes
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
Octapharma AG, Switzerland

External identifiers

EU CT number
2024-516741-39-00
EudraCT number
2019-003427-38
ClinicalTrials.gov
NCT04023019

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenetic, Therapy, Prophylaxis, Pharmacoeconomic, Efficacy, Pharmacokinetic, Safety

The primary objective for Groups 1 and 2 is to evaluate ITI outcomes as determined by achievement of the following ITI criteria:
1. Inhibitor titre < 0.6 Bethesda units (BU)/mL for at least 2 consecutive measurements
2. FVIII recovery ≥ 66% of the predefined reference value of 1.5% international units (IU)/kg body weight (BW)
3. FVIII half-life ≥ 6 h
The primary objective for Group 3 is to evaluate the annualised bleeding rate (ABR) compared with the ABR in Group 1 and Group 2.

Secondary objectives 1

  1. Secondary objectives for participants in Group 1 and Group 2 include: 1. Time to achieve ITI outcome 2. Use of emicizumab, aPCC, rFVIIa during ITI 3. Rate of FVIII inhibitor relapses during a follow-up period in participants who have achieved complete ITI success Secondary objectives for all 3 groups include the assessment of: 1. Frequency and severity of all bleeding episodes (BEs), all treated BEs, all spontaneous BEs, all joint BEs, and target joint BEs over time (≥ 3 bleeds in the same joint within 24 weeks) 2. Number of infusions required to control BEs 3. Frequency and severity of bleeding during and after surgical procedures 4. Proportion of participants experiencing adverse drug reactions (ADRs) against haemophilia treatment 5. Thrombotic events (location, treatment, outcome) 6. Treatment costs

Conditions and MedDRA coding

Haemophilia A

VersionLevelCodeTermSystem organ class
20.0 LLT 10060612 Hemophilia A 10010331

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

  1. 1. Participants can be of any age at the time of enrolment into the trial. 2. Male persons with hemophilia A (HA), of any severity, who have a historical inhibitor titre ≥ 0.6 BU/mL, including those who have failed previous ITI attempt(s) 3. Persons undergoing ITI with Nuwiq®, octanate® or wilate® or undergoing ITI with Nuwiq®, octanate® or wilate® and receiving prophylactic therapy with emicizumab, aPCC or rFVIIa 4. Participants or participants’ parent(s)/legal guardian(s) must be capable of giving signed informed consent and be able to understand the trial documents

Exclusion criteria 1

  1. 1. Participants are excluded from the trial if any coagulation disorder other than HA is diagnosed 2. Partly retrospective patients will be excluded if detailed documentation on treatment, all BEs, inhibitor titres and FVIII levels is not available for the retrospective period

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

  1. The primary endpoint in Group 1 and Group 2 is to evaluate ITI outcome as determined by achievement of the following ITI criteria: 1. Inhibitor titre < 0.6 BU/mL for at least 2 consecutive measurements 2. FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg BW 3. FVIII half-life ≥ 6 h
  2. The primary endpoint for Group 3 is to evaluate the ABR compared with the ABR in Group 1 and Group 2.

Secondary endpoints 3

  1. For participants in Group 1 and Group 2: 1. Time to achieve ITI outcome 2. Use of emicizumab, aPCC, rFVIIa during ITI 3. Rate of FVIII inhibitor relapses during a follow-up period in participants who have achieved complete ITI success
  2. For participants in all 3 groups: 1. Frequency and severity of all bleeding episodes (BEs), all treated BEs, all spontaneous BEs, all joint BEs, and target joint BEs over time (≥ 3 bleeds in the same joint within 24 weeks) 2. Number of infusions required to control BEs 3. Frequency and severity of bleeding during and after surgical procedures
  3. For participants in all 3 groups: 4. Proportion of participants experiencing ADRs 5. Thrombotic events (location, treatment, outcome) 6. Treatment costs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 29

Wilate 500, 500 I.E. VWF/500 I.E. FVIII, Pulver und Lösungsmittel zur Herstellung einer intravenösen Injektionslösung.

PRD326413 · Product

Active substance
Human Coagulation Factor Viii
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD06 — VON WILLEBRAND FACTOR AND COAGULA-TION FACTOR VIII IN COMBINATION
Marketing authorisation
PEI.H.01918.03.1
MA holder
OCTAPHARMA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Wilate 1000, 1000 I.E. VWF/1000 I.E. FVIII, Pulver und Lösungsmittel zur Herstellung einer intravenösen Injektionslösung.

PRD326415 · Product

Active substance
Human Coagulation Factor Viii
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD06 — VON WILLEBRAND FACTOR AND COAGULA-TION FACTOR VIII IN COMBINATION
Marketing authorisation
PEI.H.01918.04.1
MA holder
OCTAPHARMA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

NovoSeven 1 mg (50 KIU) powder and solvent for solution for injection

PRD3583255 · Product

Active substance
Eptacog Alfa (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/96/006/008
MA holder
NOVO NORDISK A/S
MA country
Norway
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

NovoSeven 5 mg (250 KIU) powder and solvent for solution for injection

PRD3583261 · Product

Active substance
Eptacog Alfa (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/96/006/010
MA holder
NOVO NORDISK A/S
MA country
Norway
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

NovoSeven 8 mg (400 KIU) powder and solvent for solution for injection

PRD3583263 · Product

Active substance
Eptacog Alfa (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/96/006/011
MA holder
NOVO NORDISK A/S
MA country
Norway
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

NovoSeven 2 mg (100 KIU) powder and solvent for solution for injection

PRD3583257 · Product

Active substance
Eptacog Alfa (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/96/006/009
MA holder
NOVO NORDISK A/S
MA country
Norway
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

OCTANATE 250 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung

PRD323644 · Product

Active substance
Human Coagulation Factor Viii
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
10500A/97-1
MA holder
OCTAPHARMA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

OCTANATE 500 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung

PRD323643 · Product

Active substance
Human Coagulation Factor Viii
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
10500A/97-2
MA holder
OCTAPHARMA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

OCTANATE 1000 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung

PRD323645 · Product

Active substance
Human Coagulation Factor Viii
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
10500A/97-3
MA holder
OCTAPHARMA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

CEVENFACTA 5 mg (225 KIU) powder and solvent for solution for injection

PRD9836194 · Product

Active substance
Eptacog Beta (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/22/1664/003
MA holder
LABORATOIRE FRANCAIS DU FRACTIONNEMENT ET DES BIOTECHNOLOGIES
MA country
Iceland
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

CEVENFACTA 2 mg (90 KIU) powder and solvent for solution for injection

PRD9835951 · Product

Active substance
Eptacog Beta (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/22/1664/002
MA holder
LABORATOIRE FRANCAIS DU FRACTIONNEMENT ET DES BIOTECHNOLOGIES
MA country
Iceland
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

CEVENFACTA 1 mg (45 KIU) powder and solvent for solution for injection

PRD9835764 · Product

Active substance
Eptacog Beta (Activated)
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
00 µg/Kg microgram(s)/kilogram
Max total dose
00 µg/Kg microgram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD08 — EPTACOG ALFA (ACTIVATED)
Marketing authorisation
EU/1/22/1664/001
MA holder
LABORATOIRE FRANCAIS DU FRACTIONNEMENT ET DES BIOTECHNOLOGIES
MA country
Iceland
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 150 mg/mL solution for injection

PRD10287054 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SUSPENSION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/005
MA holder
ROCHE REGISTRATION GMBH
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 150 mg/mL solution for injection

PRD5960582 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/003
MA holder
ROCHE REGISTRATION GMBH
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 150 mg/mL solution for injection

PRD5960581 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/kg milligram(s)/kilogram
Max total dose
00 mg/Kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/002
MA holder
ROCHE REGISTRATION GMBH
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 30 mg/mL solution for injection

PRD5960580 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/Kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/001
MA holder
ROCHE REGISTRATION GMBH
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 30 mg/mL solution for injection

PRD11004249 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/006
MA holder
ROCHE REGISTRATION GMBH
MA country
Iceland
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Hemlibra 150 mg/mL solution for injection

PRD5960585 · Product

Active substance
Emicizumab
Substance synonyms
RO5534262, ACE910
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS USE
Max daily dose
00 mg/kg milligram(s)/kilogram
Max total dose
00 mg/kg milligram(s)/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BX06 — -
Marketing authorisation
EU/1/18/1271/004
MA holder
ROCHE REGISTRATION GMBH
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

FEIBA 2500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.

PRD10335978 · Product

Active substance
Factor Viii Inhibitor Bypassing Fraction
Substance synonyms
HUMAN PLASMA FRACTION WITH FACTOR VIII INHIBITOR BYPASSING ACTIVITY, ANTI-INHIBITOR COAGULANT COMPLEX, ACTIVATED PROTHROMBIN COMPLEX CONCENTRATE
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS
Max daily dose
00 U/g unit(s)/gram
Max total dose
00 U/g unit(s)/gram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD03 — FACTOR VIII INHIBITOR BYPASSING ACTIVITY
Marketing authorisation
PEI.H.12158.03.1
MA holder
TAKEDA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

FEIBA 500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.

PRD10335895 · Product

Active substance
Factor Viii Inhibitor Bypassing Fraction
Substance synonyms
HUMAN PLASMA FRACTION WITH FACTOR VIII INHIBITOR BYPASSING ACTIVITY, ANTI-INHIBITOR COAGULANT COMPLEX, ACTIVATED PROTHROMBIN COMPLEX CONCENTRATE
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS
Max daily dose
00 U/g unit(s)/gram
Max total dose
00 U/g unit(s)/gram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD03 — FACTOR VIII INHIBITOR BYPASSING ACTIVITY
Marketing authorisation
PEI.H.12158.01.1
MA holder
TAKEDA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

FEIBA 1000 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung

PRD10335931 · Product

Active substance
Factor Viii Inhibitor Bypassing Fraction
Substance synonyms
HUMAN PLASMA FRACTION WITH FACTOR VIII INHIBITOR BYPASSING ACTIVITY, ANTI-INHIBITOR COAGULANT COMPLEX, ACTIVATED PROTHROMBIN COMPLEX CONCENTRATE
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INTRAVENOUS
Max daily dose
00 U/g unit(s)/gram
Max total dose
00 U/g unit(s)/gram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD03 — FACTOR VIII INHIBITOR BYPASSING ACTIVITY
Marketing authorisation
PEI.H.12158.02.1
MA holder
TAKEDA GMBH
MA country
Germany
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 500 IU powder and solvent for solution for injection

PRD1696358 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/002
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 3000 IU powder and solvent for solution for injection

PRD5992276 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/006
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 250 IU powder and solvent for solution for injection

PRD1696357 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/001
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 1000 IU powder and solvent for solution for injection

PRD1696359 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/003
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 2000 IU powder and solvent for solution for injection

PRD1696360 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/004
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 2500 IU powder and solvent for solution for injection

PRD5992277 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/005
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 4000 IU powder and solvent for solution for injection

PRD5992275 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/007
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Nuwiq 1500 IU powder and solvent for solution for injection

PRD9437977 · Product

Active substance
Simoctocog Alfa
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
200 IU/Kg iu/kilogram
Max total dose
00 IU/Kg iu/kilogram
Max treatment duration
60 Month(s)
Authorisation status
Authorised
ATC code
B02BD02 — COAGULATION FACTOR VIII
Marketing authorisation
EU/1/14/936/008
MA holder
OCTAPHARMA AB
MA country
EU
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

HZRM Haemophilie-Zentrum Rhein Main GmbH

Sponsor organisation
HZRM Haemophilie-Zentrum Rhein Main GmbH
Address
Stresemannallee 15, Sachsenhausen Sachsenhausen
City
Frankfurt Am Main
Postcode
60596
Country
Germany

Scientific contact point

Organisation
HZRM Haemophilie-Zentrum Rhein Main GmbH
Contact name
Carmen Escuriola

Public contact point

Organisation
HZRM Haemophilie-Zentrum Rhein Main GmbH
Contact name
Carmen Escuriola

Third parties 9

OrganisationCity, countryDuties
Allucent (UK) Limited
ORG-100008801
 Bracknell, United Kingdom Other
Universitaetsklinik Balgrist
ORG-100045444
 Zurich, Switzerland Laboratory analysis
Universitaetsklinikum Bonn AöR
ORG-100009711
 Bonn, Germany Other
LKF Laboratorium fuer Klinische Forschung GmbH
ORG-100017343
 Schwentinental, Germany Other
nspm AG
ORG-100054278
 Meggen, Switzerland Code 11
Emory Children Center
ORG-100052289
 Atlanta, United States Other
HZRM Haemophilie-Zentrum Rhein Main GmbH
ORG-100047532
 Frankfurt Am Main, Germany Laboratory analysis
Hospices Civils De Lyon
ORG-100006597
 Bron, France Other, Laboratory analysis
Medidata Solutions Inc.
ORG-100016256
 New York, United States E-data capture

Emory University

Sponsor organisation
Emory University
Address
1599 Clifton Road Northeast Fl 3
City
Atlanta
Postcode
30322-4250
Country
United States

Scientific contact point

Organisation
Emory University
Contact name
Robert Sidonio

Public contact point

Organisation
Emory University
Contact name
Robert Sidonio

Sponsor responsibilities

Article 77 compliance
HZRM Haemophilie-Zentrum Rhein Main GmbH
Contact point sponsor
HZRM Haemophilie-Zentrum Rhein Main GmbH
Article 77 implementation
HZRM Haemophilie-Zentrum Rhein Main GmbH

Locations

7 EU/EEA countries · 14 investigational sites

By country

CountryMS statusPlanned subjectsSites
Bulgaria Ended 3 1
Croatia Ongoing, recruiting 3 1
Finland Ended 1 1
Germany Ongoing, recruiting 6 5
Norway Ongoing, recruiting 5 1
Spain Ongoing, recruiting 2 4
Sweden Ongoing, recruiting 2 1
Rest of world
Colombia, Canada, Mexico, United States, Ukraine, Switzerland, Guatemala, United Kingdom, North Macedonia, Saudi Arabia
 — 101 —

Investigational sites

Bulgaria

1 site · Ended
Specialized Hospital For Active Treatment Of Hematological Diseases EAD
Clinic of Pediatric Hematology for treatment of children with benign hematological diseases 0-18y, Bulevard Kliment Ohridski 1a, 1797, Sofiya

Croatia

1 site · Ongoing, recruiting
University Hospital Centre Zagreb
Internal medicine, Ulica Mije Kispatica 12, 10000, Zagreb

Finland

1 site · Ended
HUS-Yhtymae
New Children’s Hospital, Stenbackinkatu 9, 00290, Helsinki

Germany

5 sites · Ongoing, recruiting
Universitaetsklinikum Bonn AöR
Insititut für Experimentelle Haematologie und Transfusionsmedizin (IHT), Venusberg-Campus 1, Venusberg, Bonn
HZRM Haemophilie-Zentrum Rhein Main GmbH
NA, Stresemannallee 15, Sachsenhausen, Frankfurt Am Main
Justus-Liebig-Universitaet Giessen
Sektion Haemostaseologie, Langhansstrasse 2, 35392, Giessen
Gerinnungszentrum Rhein-Ruhr Aerztepartnerschaft Dr. med. Hannelore Rott Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Susan Halimeh Fachaerztin fuer Transfusionsmedizin Haemostaseologie Dr. med. Guenther Kappert Facharzt fuer Laboratoriumsmedizin Haemostaseologie
NA, Koenigstrasse 13, Altstadt, Duisburg
Universitaetsklinikum Muenster AöR
Klinik für Kinder- und Jugendmedizin, Albert-Schweitzer-Campus 1, Sentrup, Muenster

Norway

1 site · Ongoing, recruiting
Oslo University Hospital HF
Center for rare diagnostics, P. O. Box 4950, 0424, Oslo

Spain

4 sites · Ongoing, recruiting
Hospital General Universitario Dr. Balmis
Dept of Haemostasis and Thrombosis, Avinguda Del Pintor Baeza 12, 03010, Alicante
Hospital Universitari Vall D Hebron
Dept of Hematology and Hemotherapy, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
University Hospital Virgen Del Rocio S.L.
Hematology Department, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Universitario San Juan De Alicante
Hematology Department, Carretera N-332 Alicante-Valencia S/n, 03550, Sant Joan D'alacant

Sweden

1 site · Ongoing, recruiting
Region Skane Skanes Universitetssjukhus
Department of hematology, Jan Waldenstroms Gata 16 Plan 5, Malmo St Johannes, Malmo

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Croatia 2020-12-18 2021-01-28
Finland 2021-02-25
Germany 2020-01-14 2020-10-26
Norway 2020-12-16 2021-04-21
Spain 2021-04-27 2021-05-12
Sweden 2021-08-18 2021-11-25

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 87 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol_2024-516741-39-00_Redacted 10.0
Protocol (for publication) D4_Patient facing documents_Patient Diary 3.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_BG 1.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_DE 2.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_ES 2.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_FI 2.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_HR 2.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_NO 2.0
Protocol (for publication) D4_Patient facing documents_Patient Diary_SE 2.0
Recruitment arrangements (for publication) K1_Blank document N/A
Recruitment arrangements (for publication) K1_Blank document N/A
Recruitment arrangements (for publication) K1_Blank document N/A
Recruitment arrangements (for publication) K1_Blank document N/A
Recruitment arrangements (for publication) K1_ES_Recruitment arrangements 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements 1
Recruitment arrangements (for publication) K1_Recruitment arrangements 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements 1.0
Recruitment arrangements (for publication) K1_Recruitment arrangements_BG-BG_For publication 1.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 14-17_BG-BG_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 14-17_BG-EN_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 2-6_BG-BG_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 2-6_BG-EN_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 7-13_BG-BG_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Assent 7-13_BG-EN_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Parent_BG-BG_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Parent_BG-EN_For publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Patient_BG-BG_for publication 2.0
Subject information and informed consent form (for publication) L1_BG_PIS and ICF Patient_BG-EN_For publication 2.0
Subject information and informed consent form (for publication) L1_ES-ES_SIS and ICF_Main Adult 6.0
Subject information and informed consent form (for publication) L1_ES-ES_SIS and ICF_Parents 6.0
Subject information and informed consent form (for publication) L1_ES-ES_SIS and ICF_Teenager Assent (12-17 yrs) 6.0
Subject information and informed consent form (for publication) L1_PIS and ICF Assent 13-17_master_EN_For publication 5.0
Subject information and informed consent form (for publication) L1_PIS and ICF Assent 8-12_master_EN_For publication 5.0
Subject information and informed consent form (for publication) L1_PIS and ICF Parent_master_EN_For publication 5.0
Subject information and informed consent form (for publication) L1_PIS and ICF Patient_master_EN_For publication 5.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Adult (16yrs and older) 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Appendix to Guardian ICF 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Appendix to Patient ICF 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Attachment to Parent Information ICF 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Attachment to Patient 15-17yrs ICF 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Attachment to Patient Information ICF 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (10-14yrs) 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (6-10yrs) 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (8-11yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (8-12 yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (8-12 yrs)_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Children Assent (8-12yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Guardian ICF 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Parent Information ICF_Redacted 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Parents Information 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Parents_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Parents_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient (15-17yrs) ICF_Redacted 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient ICF 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient Information ICF_Redacted 7.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient_TC 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Patient_UA-DE_Redacted NA
Subject information and informed consent form (for publication) L1_SIS and ICF_Teenager Assent (12-15yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Teenager Assent (13-14yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Teenager Assent (13-17 yrs) 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Teenager Assent (13-17 yrs)_Redacted 6.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Young adults Assent (15-17yrs) 6.0
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Cevenfacta N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Feiba N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Hemlibra N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Novoseven N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Nuwiq N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Octanate N/A
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Wilate N/A
Synopsis of the protocol (for publication) D1_Protocol Lay Summary__2024-516741-39-00 10.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_BG 10.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_DE 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_ES 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_HR 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_NO 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_SE 8.0
Synopsis of the protocol (for publication) D1_Protocol Lay Summary_2024-516741-39-00_TC 10.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis__2024-516741-39-00 10.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2024-516741-39-00 8.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2024-516741-39-00_BG 10.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2024-516741-39-00_ES 8.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2024-516741-39-00_TC 10.0

Application history

5 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-07-30 Finland Acceptable
2024-08-27
 2024-08-27
2 NON SUBSTANTIAL MODIFICATION NSM-1 2025-02-13 Finland Acceptable
2024-08-27
 2025-02-13
3 SUBSTANTIAL MODIFICATION SM-1 2025-04-11 Finland Acceptable
2025-07-08
 2025-07-08
4 SUBSTANTIAL MODIFICATION SM-2 2025-08-07 Acceptable 2025-08-29
5 SUBSEQUENT ADDITION OF MSC APP-5 2025-08-21 2025-10-28

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