A clinical trial to study Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII in Previously Untreated Patients with Severe Haemophilia A

Phase 3

Completed

• Conditions: Health Condition 1: D66- Hereditary factor VIII deficiency

• Registration Number: CTRI/2013/09/003958
• Lead Sponsor: Octapharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2019-06-22
• Last Update Posted: 24 November 2021

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 13

Inclusion Criteria

Subjects who fulfill the following criteria are eligible for inclusion into this study:

1. Male patients

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 patientâ??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

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
To investigate the immunogenicity of Human-cl rhFVIII in 100 previously <br/ ><br>untreated patients (PUPs) suffering from severe Haemophilia A (FVIII:C less than 1%) <br/ ><br>Timepoint: 1) At baseline (Screening Visit) <br/ ><br>2) Every 3-4 EDs until ED 20 <br/ ><br>3) Every 10-12 EDs or every 3 months ± 2 weeks (whichever comes first) after ED 20 <br/ ><br>4) At study completion <br/ ><br>5) Any time in the case of a suspicion of inhibitor development

Secondary Outcome Measures

Name	Time	Method
â?¢Assessment of the efficacy of Human-cl rhFVIII during prophylactic treatment (based on the frequency of spontaneous break-through bleeds).Timepoint: The efficacy of Human-cl rhFVIII in prophylactic treatment will be evaluated based on the frequency of spontaneous breakthrough bleeds which means each time a breakthrough bleed occurs.;â?¢Assessment of the efficacy of Human-cl rhFVIII in surgical prophylaxis after the end of surgical prophylactic treatment phaseTimepoint: Efficacy will be assessed at the end of surgery by the surgeon and post-operatively by the surgeon and the haematologist;â?¢ Assessment of the efficacy of Human-cl rhFVIII during treatment of bleedsTimepoint: At the end of a BE, the following efficacy assessment will be made by the patientâ??s parent(s)/legal guardian(s) (together with the Investigator in case of on-site treatment