This is a first in human study, in patients with severe bleeding disorder (hemophilia A), who has developed inhibitory antibodies against coagulation factor VIII.The aim is to study the safety and efficacy of dendritic cells, originating from the patient itself. The dendritic cells has been loaded with coagulation factor VIII for an improved immunological tolerance. (TOLER8)

2022-502030-12-01 Protocol 21-ItolDC-028-01 Human pharmacology (Phase I) - First administration to humans Authorised, recruiting

Start 13 Oct 2022 · Status Authorised, recruiting · 2 EU/EEA countries · 2 sites · Protocol 21-ItolDC-028-01

Overview

Sponsor-declared trial summary

Phase Human pharmacology (Phase I) - First administration to humans
Status Authorised, recruiting
Participants planned 6
Countries 2
Sites 2

Treatment of patients with Hemophilia A who have developed inhibitory antibodies to clotting Factor VIII and have failed Immune Tolerance Induction

Key facts

Sponsor
Cell4Cure AB
Participant type
Patients
Age range
18-64 years
Gender
Male
Therapeutic area
Phenomena and Processes [G] - Immune system processes [G12]
Trial duration
13 Oct 2022 → ongoing
Decision date (initial)
2022-12-13
Transition trial
Yes
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
No

External identifiers

EU CT number
2022-502030-12-01
EudraCT number
2021-000608-39

Trial design

CTIS Part I — objectives, methods, condition coding

Conditions and MedDRA coding

Treatment of patients with Hemophilia A who have developed inhibitory antibodies to clotting Factor VIII and have failed Immune Tolerance Induction

VersionLevelCodeTermSystem organ class
20.0 LLT 10053751 Hemophilia A with anti factor VIII 10010331

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Cell4Cure AB

Sponsor organisation
Cell4Cure AB
Address
Gustav IIIs Boulevard 10
City
Solna
Postcode
169 72
Country
Sweden

Scientific contact point

Organisation
Cell4Cure AB
Contact name
CEO

Public contact point

Organisation
Cell4Cure AB
Contact name
CEO

Locations

2 EU/EEA countries · 2 investigational sites

By country

CountryMS statusPlanned subjectsSites
Norway Authorised, recruiting 2 1
Sweden Authorised, recruitment pending 2 1
Rest of world
United Kingdom
2

Investigational sites

Norway

1 site · Authorised, recruiting
Oslo University Hospital Hf
Department of Hematology, Sognsvannsveien 20, 0372, Oslo

Sweden

1 site · Authorised, recruitment pending
Lund University Hospital
Department of Hematology and Coagulation, Getingevaegen 4, 222 42, Lund

Country notifications

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

Country Trial startTrial end Recruitment startRecruitment end Early termination
Norway 2022-10-13
Sweden

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2022-10-26 Sweden Acceptable
2022-12-01
2022-12-05
2 SUBSTANTIAL MODIFICATION SM-1 2023-01-19 Sweden Acceptable
2023-04-19
2023-04-20
3 SUBSTANTIAL MODIFICATION SM-2 2024-02-13 Sweden Acceptable
2024-03-15
2024-03-20
4 SUBSTANTIAL MODIFICATION SM-3 2024-07-25 Sweden Acceptable
2024-08-28
2024-08-28
5 SUBSTANTIAL MODIFICATION SM-5 2025-02-17 Sweden Acceptable
2025-03-18
2025-03-19