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

• Register: ISRCTN
• Last refreshed on: 6 June 2022
• Main ID: ISRCTN50040185
• Date of registration: 22/10/2013
• Prospective Registration: No
• Primary sponsor: Octapharma AG (Switzerland)
• Public title: NuProtect: Immunogenicity, efficacy and safety of treatment with Human-cl rhFVIII in previously untreated patients with severe haemophilia A
• Scientific title: Immunogenicity, efficacy and safety of treatment with Human-cl rhFVIII in previously untreated patients with severe haemophilia A: a prospective, multinational, open-label, non-controlled study
• Date of first enrolment: 01/03/2013
• Target sample size: 100
• Recruitment status: Completed
• URL: https://www.isrctn.com/ISRCTN50040185
• Study type: Interventional
• Study design: Prospective multicentre multinational open-label non-controlled study (Screening)
• Phase: Phase III

Countries of recruitment

Brazil, Canada, Colombia, England, France, Georgia, Germany, India, Moldova, Morocco, Poland, Russian Federation, Spain, Ukraine, United Kingdom, United States of America, Venezuela

Contacts

• Name: Raina Liesner

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender: Male

Health Condition(s) or Problem(s) studied

Severe haemophilia A
Haematological Disorders
Hereditary factor VIII deficiency

Intervention(s)

There is only one study arm. All patients receive the same investigational medicinal product (IMP) intravenously. The dose, frequency and duration are flexible, and depend on the individual clinical condition of the patient.

Primary Outcome(s)

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(s)

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.

Secondary ID(s)

GENA-05

2012-002554-23

NCT01712438

Source(s) of Monetary Support

Octapharma AG (Switzerland)

Ethics review

Status:
Approval date:
Contact:

Canada, HIREB Hamilton: 11 March 2013 Germany, Ethics Committee University Münster: 08 July 2013 Spain, Vall d`Hebron, Barcelona: 11 January 2013 France, CPP Ouest V, Nanterre: 07 February 2013 UK, NRES Committee London-Central: 19 February 2013 Georgia, Committee of Institute of Haematology, Tiflis: 17 January 2013 Moldova, National Ethics Committee, Chisi

Results

• Results available: Yes
• Date Completed: 24/03/2020