Active — Not RecruitingNot Applicable

Long Term Follow-Up for Safety of AVR-RD-02

United States2 participantsStarted 2024-02-21

Plain-language summary

The goal of this observational study is to assess the safety and tolerability of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Subject must be willing and able to provide written informed consent for the JAB-GD-001 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.
✓. Subject must have been enrolled in the preceding AVROBIO AVRO-RD-02-201 treatment study and have received AVR-RD-02 treatment

What they're measuring

Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder

Timeframe: Baseline to Year 14

Change from baseline over time in hemoglobin concentration

Timeframe: Baseline to Year 14

Change from baseline over time in platelet count

Timeframe: Baseline to Year 14

Trial details

NCT IDNCT06488261

SponsorJohn Bernat

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2037-06

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