Overview
Sponsor-declared trial summary
Phase
Therapeutic use (Phase IV)
Status
Ended
Participants planned
250
Countries
10
Sites
51
Hemophilia A and B
To evaluate the overall joint status as detected by ultrasound (US) in haemophilia A and B patients treated with efmoroctocog alfa (rFVIIIFc) or eftrenonacog alfa (rFIXFc) prophylaxis over the 18-month study period.
Key facts
- Sponsor
- Swedish Orphan Biovitrum AB (publ)
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 21 Jul 2023 → 1 Dec 2023
- Decision date (initial)
- 2023-06-19
- Transition trial
- No
- Low-intervention
- Yes
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Swedish Orphan Biovitrum AB
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Prophylaxis
To evaluate the overall joint status as detected by ultrasound (US) in haemophilia A and B patients treated with efmoroctocog alfa (rFVIIIFc) or eftrenonacog alfa (rFIXFc) prophylaxis over the 18-month study period.
Secondary objectives 5
- To evaluate the joint status as detected by US in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) specific items of hypertrophic synovium, cartilage and bone damage in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period
- To evaluate the clinical joint status as detected by Hemophilia Joint Health Score (HJHS) in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period
- To evaluate the presence, resolution, recurrence and new development of target joints in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over 18 months
- To describe the bleeding episodes in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period
- To evaluate the quality of life in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period
Conditions and MedDRA coding
Hemophilia A and B
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10018939 | Haemophilia B (Factor IX) | 10010331 |
| 20.0 | LLT | 10018937 | Haemophilia A | 10010331 |
| 20.0 | LLT | 10018937 | Haemophilia A | 10010331 |
| 20.0 | LLT | 10018939 | Haemophilia B (Factor IX) | 10010331 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 7
- Age ≥ 6 years
- Diagnosis of haemophilia A or B
- Having at least 6 months documented pre-study treatment data regarding treatment prescriptions and bleeding episodes prior to the baseline visit
- Previous treatment for haemophilia A or B with any marketed recombinant and/or plasma-derived FVIII or FIX concentrate for at least 6 months
- Start of prophylactic treatment with rFVIIIFc or rFIXFc prior to study enrolment or latest at the baseline visit, in accordance with local regulations
- Signed and dated informed consent provided by the patient, or the patient’s legally authorized representative for patients under the legal age. Assent should be obtained from paediatric patients in accordance with local regulations
- To be eligible for the florio HAEMO sub-study a patient should have used florio HAEMO (a CE marked medical device used in routine clinical practice) for at least 3 months and must agree to have data collected from the florio HAEMO app by providing a separate informed consent or assent
Exclusion criteria 5
- Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion
- Prophylactic treatment with non-factor therapy during the 6 months prior to enrolment
- Presence of factor VIII or FIX inhibitory antibodies (inhibitors) (≥0.60 Bethesda Units [BU]/mL) at the latest available inhibitor test
- Enrolment in a concurrent clinical interventional study, or intake of an Investigational medicinal product, within 3 months prior to inclusion in this study
- Foreseeable inability to cooperate with given instructions or study procedures
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Change from baseline in total HEAD-US score up to month 18 (end of study - EOS)
Secondary endpoints 9
- Change from baseline in HEAD-US score for hypertrophic synovium at 6, 12 and 18 months
- Change from baseline in HEAD-US score for cartilage at 6, 12 and 18 months
- Change from baseline in HEAD-US score for bone at 6, 12 and 18 months
- Change from baseline in total HJHS at month 18 (EOS)
- Number and location of target joints at baseline, 6, 12 and 18 months
- Total Annualized bleeding rate (ABR), joint ABR, target joint ABR, traumatic/spontaneous ABR
- Patient-Reported Outcomes Measurement Information System (PROMIS) physical function/activity scores at baseline and 18 months
- PROMIS pain intensity and interference scores at baseline and 18 months
- IPAQ-SF scores at baseline and 18 months
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 13
ELOCTA 2000 IU Powder and solvent for solution for injection
PRD3961084 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/006
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 750 IU Powder and solvent for solution for injection
PRD3960978 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/003
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 500 IU Powder and solvent for solution for injection
PRD3960959 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/002
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 1500 IU Powder and solvent for solution for injection
PRD3961065 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/005
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 3000 IU powder and solvent for solution for injection
PRD7210768 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/007
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 4000 IU powder and solvent for solution for injection
PRD6832304 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/008
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 250 IU Powder and solvent for solution for injection
PRD3960817 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/001
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ELOCTA 1000 IU Powder and solvent for solution for injection
PRD3961045 · Product
- Active substance
- Efmoroctocog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1, BIIB031
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 65 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD02 — COAGULATION FACTOR VIII
- Marketing authorisation
- EU/1/15/1046/004
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ALPROLIX 500 IU powder and solvent for solution for injection
PRD4458004 · Product
- Active substance
- Eftrenonacog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, BIIB-029, rFIXFc, COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 100 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD04 — COAGULATION FACTOR IX
- Marketing authorisation
- EU/1/16/1098/002
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/07/453
- Modified vs. Marketing Authorisation
- No
ALPROLIX 3000 IU powder and solvent for solution for injection
PRD4458046 · Product
- Active substance
- Eftrenonacog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, BIIB-029, rFIXFc, COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 100 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD04 — COAGULATION FACTOR IX
- Marketing authorisation
- EU/1/16/1098/005
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/07/453
- Modified vs. Marketing Authorisation
- No
ALPROLIX 1000 IU powder and solvent for solution for injection
PRD4458017 · Product
- Active substance
- Eftrenonacog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, BIIB-029, rFIXFc, COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 100 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD04 — COAGULATION FACTOR IX
- Marketing authorisation
- EU/1/16/1098/003
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/07/453
- Modified vs. Marketing Authorisation
- No
ALPROLIX 250 IU powder and solvent for solution for injection
PRD4457980 · Product
- Active substance
- Eftrenonacog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, BIIB-029, rFIXFc, COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 100 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD04 — COAGULATION FACTOR IX
- Marketing authorisation
- EU/1/16/1098/001
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/07/453
- Modified vs. Marketing Authorisation
- No
ALPROLIX 2000 IU powder and solvent for solution for injection
PRD4458040 · Product
- Active substance
- Eftrenonacog Alfa
- Substance synonyms
- Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, BIIB-029, rFIXFc, COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INJECTION
- Max daily dose
- 100 % percent
- Max total dose
- 100 IU/Kg iu/kilogram
- Max treatment duration
- 1200 Month(s)
- Authorisation status
- Authorised
- ATC code
- B02BD04 — COAGULATION FACTOR IX
- Marketing authorisation
- EU/1/16/1098/004
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/07/453
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Swedish Orphan Biovitrum AB (publ)
- Sponsor organisation
- Swedish Orphan Biovitrum AB (publ)
- Address
- Tvistevagen 48, Alidhem Alidhem
- City
- Umea
- Postcode
- 907 36
- Country
- Sweden
Scientific contact point
- Organisation
- Swedish Orphan Biovitrum AB (publ)
- Contact name
- Ekaterina GRESKO
Public contact point
- Organisation
- Swedish Orphan Biovitrum AB (publ)
- Contact name
- Rolf SCHLOSSHAUER
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| TREMENDOUS INC ORL-000000367
|
NEW YORK, United States | Other |
| Iqvia Limited ORG-100008655
|
Reading, United Kingdom | On site monitoring, Code 10, Code 11, Code 2, Code 5, Data management, E-data capture |
Locations
10 EU/EEA countries · 51 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Bulgaria | Ended | 8 | 2 |
| Croatia | Ended | 10 | 2 |
| Czechia | Ended | 15 | 5 |
| France | Ended | 97 | 20 |
| Hungary | Ended | 10 | 1 |
| Ireland | Ended | 5 | 2 |
| Italy | Not authorised | 20 | 2 |
| Romania | Ended | 30 | 5 |
| Slovenia | Ended | 5 | 1 |
| Spain | Ended | 47 | 11 |
| Rest of world
United Kingdom
|
— | 3 | — |
Investigational sites
Uniteversity Muliprofile Hospital For Active Treatment Tsaritsa Yoanna-Isul EAD
Expert centre for Haemophilaand other rare bleedin, Oborishte Distr., Ul.Byalo More 8, Sofia
Specialized Hospital For Active Treatment Of Oncological Diseases Sofia Region EOOD
Dept. of Clin Hematology in Hemorrhagic Diathesis, Banishora Residential Area, Bulevard Gen. Stoletov 67, Sofiya
University Hospital Centre Zagreb
-, Ulica Mije Kispatica 12, Zagreb, Grad Zagreb
University Hospital Centre Zagreb
-, Ulica Mije Kispatica 12, Zagreb, Grad Zagreb
Institute Of Hematology And Blood Transfusion
Center of Thrombosis and Hemostasis, U Nemocnice 2094/1, Nove Mesto, Prague
Krajska nemocnice Liberec a.s.
Department of Emergency, Husova 357/10, Liberec I-Stare Mesto, Liberec (neclenene Mesto)
University Hospital Olomouc
Department of Hemato-Oncology, I. P. Pavlova 185/6, Nova Ulice, Olomouc
Fakultni Nemocnice Brno
Dept. of Oddeleni detske hematologie, Cernopolni 9, Cerna Pole, Brno-Sever
Fakultni Nemocnice V Motole
Dept of Klinika detske hematologie a onkologie UK, V Uvalu 84, 150 06, Prague 5
Centre Hospitalier Universitaire De Caen Normandie
Service d'Hématologie Biologique, Avenue De La Cote De Nacre, Cs 30001, Caen Cedex 9
Assistance Publique Hopitaux De Paris
Centre Régional de Traitement de l'Hémophilie, 149 Rue De Sevres, 75015, Paris
Hospices Civils De Lyon
Unité d'Hémostase Clinique, 59 Boulevard Pinel, 69500, Bron
Hospital Hotel Dieu
-, 1 Place Alexis Ricordeau, 44000, Nantes
Centre Hospitalier Universitaire De Poitiers
Service d'Hématologie Clinique, 2 Rue De La Miletrie, 86000, Poitiers
Centre Hospitalier Regional Universitaire De Tours
Service d'Hématologie Hémostase, Avenue De La Republique, 37170, Chambray Les Tours
CHRU De Nancy
Hématologie Biologique, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Centre Hospitalier Universitaire De Dijon
Centre Mémoire Ressources Recherche (CMRR), 14 Rue Paul Gaffarel, 21000, Dijon
Centre Hospitalier Universitaire De Rennes
Service d'Hématologie Clinique, 2 Rue Henri Le Guilloux, 35033, Rennes Cedex 9
University Hospital Of Clermont-Ferrand
Service d'Hématologie, 1 Place Lucie Et Raymond Aubrac, 63100, Clermont-Ferrand
Les Hopitaux Universitaires De Strasbourg
Centre de Référence pour le Traitement des Hémophilies, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Assistance Publique Hopitaux De Marseille
Centre Régional du Traitement de l'Hémophilie CRT, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Universitaire De Lille
Hémostaste Clinique - Institut Coeur Poumons, Boulevard Du Professeur Jules Leclercq, 59000, Lille
Centre Hospitalier Universitaire De Bordeaux
Service d'Hématologie Pédiatrique, Place Amelie Raba Leon, 33000, Bordeaux
Centre Hospitalier Universitaire De Nimes
-, 4 Place Du Professeur Robert Debre, Bp 40026, Nimes Cedex 9
Centre Hospitalier Universitaire De Toulouse
Centre d'Hémophilie, 330 Avenue De Grande Bretagne, 31059, Toulouse Cedex 9
Hospital Maison Blanche
-, 45 Rue Cognacq Jay, 51092, Reims Cedex
Bicetre Hospital
Service Hématologie et Immunologie Clinique, 78 Rue Du General Leclerc, 94275, Le Kremlin Bicetre Cedex
Centre Hospitalier Metropole Savoie
-, Place Lucien Biset, Bp 31125, Chambery
CHU De Rouen
Haemophilia Care Center, 1 Rue De Germont, Bp 96031, Rouen Cedex
Medical Centre Hungarian Defence Forces
Hematologiai Centrum, Robert Karoly Korut 44, 1134, Budapest XIII
Childrens Health Ireland
-, Cooley Road, Crumlin, Dublin
St James's Hospital
-, James's Street, Ireland, Dublin 8
Humanitas Research Hospital
Centro Trombosi, Via Alessandro Manzoni 56, 20089, Rozzano
Ospedale Santa Chiara
Hematology, Via Roma 67, 56126, Pisa
Fundeni Clinical Institute
Hematology, Soseaua Fundeni 258, 022328, Bucharest
Spitalul Judetean De Urgenta Dr. Constantin Opris Baia Mare
Hematology, Strada Cosbuc George Nr 31, 430031, Baia Mare
Spitalul Clinic Municipal De Urgenta Timisoara
Hematology, Strada Dima Gheorghe Nr.5, 300079, Timisoara
Spitalul Clinic Municipal Filantropia Craiova
Hematology, Str. Filantropiei, 1, Craiova
Spitalul Clinic Judetean De Urgenta Sf. Spiridon Iasi
Hematology, Bulevardul Independentei 1, 700111, Jassi
Hospital Universitario De Guadalajara SESCAM
Hematology Department, Calle De Los Donantes De Sangre S/n, 19002, Guadalajara
Hospital de la Santa Creu i Sant Pau
Servicio de Hematologia, Calle de Sant Quintí, 89, Barcelona
Hospital Universitario De Salamanca
Servicio de Hematologia, Paseo De San Vicente 58-182, 37007, Salamanca
Hospital General Universitario Dr. Balmis
Servicio de Hematología, Avenida Pintor Baeza, 12, Alicante
Complexo Hospitalario Universitario A Coruna
Department of Hematology, Lugar Jubias De Arriba 84, 15006, A Coruna
Hospital Unviersitario Miguel Servet
Servicio de Hematologia y Hemoterapia, Paseo De Isabel La Catolica 1-3, 50009, Zaragoza
Hospital Universitario Central De Asturias
Servicio de Hematologia y Hemoterapia, Avenida De Roma S/n, 33011, Oviedo
Hospital Universitario Rio Hortega
Servicio de Hematologica, Calle Dulzaina 2, 47012, Valladolid
Hospital Universitario Regional De Malaga
Hematology Department, Avenida De Carlos De Haya S/n, 29010, Malaga
Hospital Universitario La Paz
Hematology Department, Paseo Castellana 261, 28046, Madrid
Hospital Universitario Y Politecnico La Fe
Haemostasis and Thrombosis Unit, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Bulgaria | 2023-07-25 | ||||
| Croatia | 2023-09-12 | ||||
| Czechia | 2023-10-04 | 2023-10-16 | |||
| France | 2023-08-23 | 2023-08-24 | |||
| Ireland | 2023-08-03 | ||||
| Romania | 2023-07-21 | 2023-09-27 | |||
| Slovenia | 2023-07-24 | ||||
| Spain | 2023-08-18 | 2023-11-16 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Low Intervention Clinical Trial Report Summary SUM-26747
|
2024-05-27T17:31:42 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Lay Person Summary of Results | 2024-05-27T17:33:18 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | Sobi_JOIN us_Plain Language Summary of Results_Version_06May2024a_clean | 1 |
| Summary of results (for publication) | Sobi_JOIN Us_aCSR_FinalV1_08May2024_Summary | 1 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-02-17 | France | Acceptable 2023-06-12
|
2023-06-13 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-07-19 | France | Acceptable | 2023-08-28 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2023-08-01 | Acceptable | 2023-08-23 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2023-08-29 | France | Acceptable | 2023-10-16 |
| 5 | SUBSEQUENT ADDITION OF MSC | APP-5 | 2023-09-18 | Acceptable |