Long-term observational study of Gene Therapy for AADC deficiency

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT2033220245

Enrollment

Registered

2022-08-05

Start date

2022-08-05

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Aromatic L-amino acid decarboxylase deficiency

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -Patients who enrolled in the Phase I/II Study of Gene Therapy for AADC deficiency, and who completed or discontinued the study. -Female patients who are able to become pregnant, and whose partners agree to use medically effective contraceptive methods throughout the study period.

Exclusion criteria

Exclusion criteria: -Patients who cannot undergo MRI.

Design outcomes

Primary

Measure	Time frame
To investigate the long-term tolerability, safety, and efficacy of the study product in patients with AADC deficiency, after intracerebral administrations of AAV-hAADC-2 into the putamen.	—

Contacts

Public ContactHiroaki Matsumoto

SRD, Inc.

aadc_gt0002x-01@cro-srd.co.jp+81-3-5543-0306

Outcome results