臨床研究等提出・公開システム

Phase I/II Study of Gene Therapy for AADC deficiency

Phase I/II Study of Gene Therapy for AADC deficiency

Yamagata Takanori

Jichi Medical University Hospital

3311-1,Yakushiji, Shimotsuke-shi, Tochigi

takanori@jichi.ac.jp

Sekiguchi Shinya

SRD, Inc.

3-4-8 Hacchobori, Chuo-ku, Tokyo

+81-3-5543-0306

aadc_gt0002x-01@cro-srd.co.jp

Complete

Mar. 14, 2022

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

-Patients with clinical symptoms of motor dysfunctions and/or dystonia, whose severities are estimated as "Definite" or "Probable", according to the "323 Aromatic L-amino acid decarboxylase deficiency" of the Intractable Diseases Information Center.
-Patients whose ages are over two(2) at the time of obtaining informed consent,and who have skulls for whom stereotaxic brain surgeries could be performed. The upper limit of ages was not established.
-Patients without any clinical evidence of other neurodegerative disorders.
-Patients who do not need to add new therapeutic agents or patients who do not need dosage changes for the AADC deficiency treatment for at least 4 weeks, prior to the start of the study substance administration.
-Female patients who are able to become pregnant, and whose partners agree to use medically effective contraceptive methods during the study period.

-Patients with apparent vascular diseases, including cerebrovascular disorders.
-Patients with clinically prominent evidence of other neurological disorders.
-Patients with clinically prominent evidence of immunodeficiency who require immunosuppressant treatments excluding steroid therapy.
-Patients who cannot undergo MRI.
-Patients with the following complications that are hard to manage.
1:Patients with severe renal disorders of the magnitude of both the serum creatinine levels of > 2.0 mg/dl and BUN>25 mg/dl.
2: Patients with severe hepatic disorders of the magnitude of either AST/GOT or ALT/GPT is above 2.5 folds of the upper limit of the normal levels.
3: Patients with diabetes of the magnitude of both >200 mg/dl casual or postprandial blood glucose levels and >9% HbA1c.

Both

Aromatic L-amino acid decarboxylase deficiency

AAV-hAADC-2 is designed to be injected into the striatum (putamen) of the patients with AADC deficiency, according to the stereotactic neurosurgical method.

To examine the totlerability and safety in patients with AADC deficiency, after intracerebral administrations of AAV-hAADC-2 into the putamen

To examine the efficacy in patients with AADC deficiency, after intracerebral administrations of AAV-hAADC-2 into the putamen

+81-285-58-7195

Oct. 27, 2021

No

none