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A Phase 3 Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia

A Phase 3 Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia

Kajiwara Makoto

PPD-SNBL K.K.

St. Luke's Tower 12F, 8-1, Akashi-cho, Chuo-ku, Tokyo

ppdsnbl-9-dtx401-cl301_jpn_all@ppd.com

Kajiwara Makoto

PPD-SNBL K.K.

St. Luke's Tower 12F, 8-1, Akashi-cho, Chuo-ku, Tokyo

+81-80-6771-1846

ppdsnbl-9-dtx401-cl301_jpn_all@ppd.com

Not Recruiting

May. 31, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

- Male and female patients >= 8 years of age at the time of informed consent or assent.
- Subject has a diagnosis of GSDIa confirmed by deficient enzymatic activity (on liver biopsy), or by molecular testing of G6PC gene revealing 2 pathogenic mutations. In the case where only a single pathogenic mutation is identified, clinical diagnosis is compatible with GSDIa and absence of characteristic features of GSDIb (ie, chronic neutropenia, inflammatory bowel disease).
- Subject is currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status.

- Detectable pre-existing antibodies to the AAV8 capsid during Screening.
- History of liver transplant, including hepatocyte cell therapy/transplant.
- History of severe hepatic fibrosis or cirrhosis as evidenced by any of the following: portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or a liver biopsy with evidence of stage III fibrosis.
- Presence of liver adenoma > 5 cm in size or presence of liver adenoma > 3 cm and =< 5 cm in size with a documented annual growth rate of >= 0.5 cm per year.
- Significant hepatic injury or dysfunction as evidenced by imaging or any of the following laboratory abnormalities.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 X the upper limit of normal (ULN)
- Total bilirubin > ULN unless the subject has Gilbert syndrome
- Alkaline phosphatase > ULN, with gamma-glutamyl transferase > ULN
- Non-fasting triglycerides greater than or equal to 1000 mg/dL. For the purposes of this study, non-fasting refers to the longest fasting period that each individual subject is able to tolerate.
- Current or previous participation in another gene transfer study.
Note: Additional inclusion/exclusion criteria may apply, per protocol.

Both

Glycogen storage disease type Ia

Gene Therapy Products: DTX401
On Day1, subjects will receive a single, open-labeled, peripheral IV infusion of DTX401.

- To evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy needed to maintain glucose control

- To evaluate the effect of DTX401 on reducing the frequency of exogenous glucose replacement therapy
- To evaluate the effect of DTX401 on glucose control
- To evaluate the effect of DTX401 on subject experience of disease
- To evaluate the effect of DTX401 on glucose control
- To evaluate the safety of DTX401

+81-96-344-2111

Mar. 11, 2024

No

United States/Brazil/Canada/Denmark/Germany/Italy/Netherland/Spain