Gene Therapy Study in Severe Haemophilia A Patients With Antibodies Against AAV5 (270-203)
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|ClinicalTrials.gov Identifier: NCT03520712|
Recruitment Status : Enrolling by invitation
First Posted : May 10, 2018
Last Update Posted : April 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Haemophilia A Gene Therapy Clotting Disorders Blood Disorder||Biological: Valoctocogene Roxaparvovec||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5|
|Actual Study Start Date :||April 3, 2018|
|Estimated Primary Completion Date :||June 2024|
|Estimated Study Completion Date :||June 2024|
Experimental: valoctocogene roxaparvovec Open Label
Single administration of BMN270 at a dose of 6E13 vg/kg
Biological: Valoctocogene Roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Other Name: BMN 270
- Percentage of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v4.03 for 5 years following valoctocogene roxaparvovec infusion. [ Time Frame: 61 months ]Safety of a single intravenous administration of BMN 270 in severe HA subjects with pre-existing antibody to AAV5 vector capsid, including development of FVIII neutralizing antibody.
- Efficacy of BMN 270 defined as FVIII activity at or above 5 IU/dL at Week 26 [ Time Frame: 26 weeks ]
- Impact of BMN 270 on usage of exogenous FVIII replacement therapy [ Time Frame: 61 months ]
- Impact of BMN 270 on the number of bleeding episodes requiring exogenous FVIII therapy [ Time Frame: 61 months ]
- Pharmacodynamics of FVIII expression following IV infusion of BMN 270 roxaparvovec [ Time Frame: 61 months ]
- Impact of BMN 270 on patient-reported outcomes (PROs) [ Time Frame: 61 months ]
- Assess the impact of valoctocogene roxaparvovec following Patient Reported Outcome (PRO) Haemo-QoL-A. [ Time Frame: 61 months ]Haemo-QoL-A is a hemophilia-specific, health-related quality of life questionnaire for adults on a scale of 0-5 with the lower value representing a better outcome.
- Assess the impact of valoctocogene roxaparvovec following Patient Reported Outcome (PRO) EQ-5D-5L [ Time Frame: 61 months ]EQ-5D-5L is a general questionnaire designed to measure health status on a scale of 0-100 with the higher value representing a better outcome.
- Assess the impact of valoctocogene roxaparvovec following Patient Reported Outcome (PRO) Haemophilia Activities List (HAL) [ Time Frame: 52 weeks ]HAL is a questionnaire that has several activities are listed that could be difficult for people with hemophilia. The aim of the questionnaire is to see how easy it is for participates to do those activities.
- Assess the impact of valoctocogene roxaparvovec following Patient Reported Outcome (PRO) Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI+CIQ:HS) [ Time Frame: 52 weeks ]WPAI+CIQ:HS is a questionnaire that is designed to measure the effect of symptom severity due to hemophilia on work productivity and activity/classroom impairment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03520712
|Royal Free Hospital|
|London, United Kingdom|
|University Hospital Southampton NHS Foundation Trust|
|Southampton, United Kingdom|
|Study Director:||Medical Director, MD||BioMarin Pharmaceutical|