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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02576795
Other study ID # BMN 270-201
Secondary ID 2014-003880-38
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date August 2015
Est. completion date March 2024

Study information

Verified date December 2023
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A.


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Study Design


Related Conditions & MeSH terms


Intervention

Biological:
valoctocogene roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A

Locations

Country Name City State
United Kingdom Queen Elizabeth Hospital Birmingham Birmingham
United Kingdom Addenbrooke's Hospital Cambridge
United Kingdom St. Thomas' Hospital London
United Kingdom The Royal London Hospital London
United Kingdom University Hospital Southampton NHS Foundation Trust Southampton

Sponsors (1)

Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v4.03 for 7 years following valoctocogene roxaparvovec infusion. 85 Months
Primary To determine the dose of AAV5-hFVIII-SQ required to achieve expression of FVIII at or above 5% of normal activity (>5 IU/dL) at 16 weeks after infusion. The kinetics, duration and magnitude of AAV-mediated FVIII activity in individuals with haemophilia A will be determined and correlated to an appropriate BMN 270 dose. 85 Months
Secondary To describe the immune response to the FVIII transgene product and AAV capsid proteins following systemic administration of AAV5-hFVIII-SQ 85 Months
Secondary Frequency of FVIII replacement therapy during the study 85 Months
Secondary Number of bleeding episodes requiring treatment during the study 85 Months
See also
  Status Clinical Trial Phase
Completed NCT02256917 - Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A Phase 3
Completed NCT01863758 - Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A Phase 3
Recruiting NCT02314325 - Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens Phase 4
Completed NCT02697370 - Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A Phase 4