uProtect: Immunogenicity, efficacy and safety of treatment with Human-cl rhFVIII in previously untreated patients with severe haemophilia A

Phase 3

Completed

• Conditions: Severe haemophilia A; Haematological Disorders; Hereditary factor VIII deficiency

• Registration Number: ISRCTN50040185
• Lead Sponsor: Octapharma AG (Switzerland)

Brief Summary

2018 Interim results article in https://www.ncbi.nlm.nih.gov/pubmed/28815880 interim results (added 14/05/2019)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-10-22
• Study Completion: 2020-03-24
• Last Update Posted: 6 June 2022

Recruitment & Eligibility

• Status: Completed
• Sex: Male
• Target Recruitment: 100

Inclusion Criteria

1. Male, no age limitations, but due to the required patient population it can be expected that the majority of patients going to be included are babies and small children.
2. Severe haemophilia A (FVIII:C < 1%)
3. No previous treatment with FVIII concentrates or other blood products containing FVIII
4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient?s parent/legal guardian)

Exclusion Criteria

1. Diagnosis with a coagulation disorder other than haemophilia A
2. Severe liver or kidney disease (alanine amino transferase (ALT) or aspartate transaminase (AST) levels >5 times of upper limit of normal, creatinine >120 µmol/L)
3. Concomitant treatment with any systemic immunosuppressive drug
4. Participation in another interventional clinical study currently or during the past 4 weeks.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The immunogenic potential of the IMP. Each patient is tested for the development of inhibitors at treatment start, every three to four exposure days to the IMP, latterly every ten exposure days (latest every three months).

Secondary Outcome Measures

Name	Time	Method
Safety, efficacy and tolerability: Efficacy (by assessing each treatment of a bleeding episode, or the rate of bleeds in case of prophylactic treatment) and safety (adverse events) are observed during the entire study duration, which is planned for a total of 100 exposure days with the IMP, but not longer than 5 years.