Trials / Terminated
TerminatedNCT04999059
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
A Long-Term Follow-Up Study of Subjects With Fabry Disease Who Previously Received Ex-Vivo, Lentiviral Vector-Mediated Gene-Modified Autologous Cell Therapy AVR-RD-01 in Study AVRO-RD-01-201
- Status
- Terminated
- Phase
- —
- Study type
- Observational
- Enrollment
- 5 (actual)
- Sponsor
- AVROBIO · Industry
- Sex
- Male
- Age
- 16 Years – 50 Years
- Healthy volunteers
- Not accepted
Summary
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.
Detailed description
Participants enrolled in the AVRO-RD-01-201 study will be offered participation in the AVRO-RD-01-LTF01 study. The Baseline visit for the AVRO-RD-01-LTF01 study will coincide with the participant's last visit in the AVRO-RD-01-201 study. Participants confirmed eligible for the AVRO-RD-01-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for 10 years (for a total of 15 years from AVR-RD-01 infusion) during which time continued safety, engraftment, and efficacy of treatment will be assessed.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | Safety and Efficacy Assessments | Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention. |
Timeline
- Start date
- 2019-05-08
- Primary completion
- 2023-08-16
- Completion
- 2023-08-16
- First posted
- 2021-08-10
- Last updated
- 2023-08-31
Locations
3 sites across 2 countries: Australia, Brazil
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04999059. Inclusion in this directory is not an endorsement.