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

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2013/09/003958

Enrollment

100

Registered

2013-09-05

Start date

Unknown

Completion date

Unknown

Last updated

2021-11-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: D66- Hereditary factor VIII deficiency

Interventions

Intervention1: Human-cl rhFVIII: Human-cl rhFVIII is a purified B-domain deleted FVIII glycoprotein that is synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F). Human-c

Eligibility

Inclusion criteria

Inclusion criteria: Subjects who fulfill the following criteria are eligible for inclusion into this study: 1. Male patients 2. Severe haemophilia A (FVIII: C 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

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 Âµmol/L) 3.Concomitant treatment with any systemic immunosuppressive drug Participation in another interventional clinical study currently or during the past 4 weeks

Design outcomes

Primary

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

Measure

Time frame

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

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Secondary

Measure	Time frame
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	—

Measure

Time frame

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

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Countries

Belarus, Canada, France, Georgia, Germany, India, Italy, Morocco, Poland, Portugal, Republic of Moldova, Russian Federation, Slovenia, Spain, Ukraine, United Kingdom, United States of America

Contacts

Public ContactDr Shariq Anwar

JSS Medical Research India Private Limited

sonika.newar@jssresearch.com91-8800799887

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026