Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Severe Hemophilia A Clinical Trial

Official title:

Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01992549
• Other study ID #: GENA-15
• Status: Completed
• Phase: Phase 3
• Start date: April 2014
• Est. completion date: December 27, 2018

Study information

• Verified date: December 2020
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 48
• Est. completion date: December 27, 2018
• Est. primary completion date: December 27, 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Patients who completed GENA-05 in accordance with the study protocol

Exclusion Criteria:

1. Severe liver or kidney disease

2. Concomitant treatment with any systemic immunosuppressive drug;

3. Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

Biological:
• Human-cl rhFVIII

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
Canada	McMaster Children's Hospital	Hamilton	Ontario
Canada	Hospital for Sick Children	Toronto
Canada	BC Children's Hospital	Vancouver	British Columbia
France	Hopital de la Timone	Marseille
France	Hôpital Kremlin Bicètre	Paris
Georgia	Institute of Hematology and Transfusiology	Tbilisi
India	Sahyadri Speciality Hospital	Pune
India	Christian Medical College	Vellore
Moldova, Republic of	IMSP Mother and Child Institute	Chisinau
Poland	University Medical School	Warsaw
Ukraine	The National Children Specialized Hospital "OHMATDET"	Kiev
Ukraine	Danylo Halytsky Lviv National Medical University	Lviv
United Kingdom	Great Ormond Street Hospital for Children	London
United States	UC Davis Medical Center	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

United States,  Canada,  France,  Georgia,  India,  Moldova, Republic of,  Poland,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors	The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre =0.6 BU/mL at any time point during the observation period.	Maximum two years
Secondary	Frequency of Spontaneous Break-through Bleeds	The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).	Maximum 2 years
Secondary	Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.	Maximum 2 years
Secondary	Efficacy of Human-cl rhFVIII for Surgical Prophylaxis	An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".	Maximum 2 years
Secondary	The Occurrence of Any Adverse Event (AE)	The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.	Maximum 2 years