Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Severe Haemophilia A Clinical Trial

Official title:
A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A

Status Active, not recruiting

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02576795
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	August 2015
Completion date	March 2024

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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