A Fabry Disease Gene Therapy Study (MARVEL1)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04040049|
Recruitment Status : Recruiting
First Posted : July 31, 2019
Last Update Posted : February 3, 2021
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease Lysosomal Storage Diseases||Genetic: FLT190||Phase 1 Phase 2|
Patients who provide consent to participate in this study will be screened for eligibility.
Eligible patients will attend the study site on the day prior to infusion (Day -1) for a baseline visit. On Day 0, FLT190 will be administered as a single dose, slow intravenous infusion. Following FLT190 treatment the patient will be discharged from the investigational site and will continue to be monitored at outpatient visits for a period of 9 months; following which, the patient will enter a period of long-term follow-up conducted under a separate protocol.
The study will be conducted in 2 parts;
Part 1: Enrolment of previously treated patients (Dose escalation)
Part 2: Enrolment of previously untreated patients (Dose expansion).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease|
|Actual Study Start Date :||July 8, 2019|
|Estimated Primary Completion Date :||December 2022|
|Estimated Study Completion Date :||December 2022|
FLT190 is a replication-incompetent adeno- associated viral (AAV) vector. Administered by a single intravenous infusion.
Gene Therapy product.
- Frequency of treatment-emergent adverse events (AEs) [ Time Frame: From screening to 9 months post infusion ]To investigate the safety of systemic administration of FLT190.
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): NCT04040049
|Contact: Clinical Operations||+44 1438 firstname.lastname@example.org|
|Charité - Universitätsmedizin Berlin||Recruiting|
|University of Würzburg||Recruiting|
|Universita Federico II di Napoli||Recruiting|
|Haukeland University Hospital||Recruiting|
|Royal Free Hospital||Recruiting|
|London, United Kingdom|