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JAPANESE
国立保健医療科学院
JRCT ID: jRCT2073230126

Registered date:15/03/2024

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

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedGlycogen storage disease type Ia
Date of first enrollment21/08/2024
Target sample size4
Countries of recruitmentUnited States,Japan,Brazil,Japan,Canada,Japan,Denmark,Japan,Germany,Japan,Italy,Japan,Netherland,Japan,Spain,Japan
Study typeInterventional
Intervention(s)Gene Therapy Products: DTX401 On Day1, subjects will receive a single, open-labeled, peripheral IV infusion of DTX401.

Outcome(s)

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

Key inclusion & exclusion criteria

Age minimum>= 8age old
Age maximumNot applicable
GenderBoth
Include criteria- 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.
Exclude criteria- 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.

Related Information

Contact

Public contact
Name Makoto Kajiwara
Address St. Luke&#039;s Tower 12F, 8-1, Akashi-cho, Chuo-ku, Tokyo Tokyo Japan 104-0044
Telephone +81-80-6771-1846
E-mail ppdsnbl-9-dtx401-cl301_jpn_all@ppd.com
Affiliation PPD-SNBL K.K.
Scientific contact
Name Makoto Kajiwara
Address St. Luke&#039;s Tower 12F, 8-1, Akashi-cho, Chuo-ku, Tokyo Tokyo Japan 104-0044
Telephone +81-80-6771-1846
E-mail ppdsnbl-9-dtx401-cl301_jpn_all@ppd.com
Affiliation PPD-SNBL K.K.