EUCTR2021-000903-19-IT
Active, not recruiting
Phase 1
A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III - UX053-CL101
TRAGENYX PHARMACEUTICAL INC.0 sites30 target enrollmentMay 24, 2021
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Glycogen Storage Disease Type III (GSD III)
- Sponsor
- TRAGENYX PHARMACEUTICAL INC.
- Enrollment
- 30
- Status
- Active, not recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Confirmed diagnosis of Glycogen Storage Disease type III (GSD III) (all subtypes) based on pathogenic mutations in the amylo\-a\-1,6\-glucosidase 4\-alpha\-glucanotransferase (AGL) gene on both alleles or glycogen debranching enzyme (GDE) deficiency based on biopsy of liver, muscle, or fibroblasts
- •2\. History of any of the following:
- •a. Severe hypoglycemia, defined as neuroglycopenia (eg, altered mental status, seizure, dizziness, slurred speech, blurry vision, abnormal behavior, perioral paresthesia, requiring intervention by a caregiver) or blood glucose \< 54 mg/dL (3 mmol/L) within the last year
- •b. \= 2 incidents of symptomatic hypoglycemia (defined as blood glucose \< 70 mg/dL \[3\.9 mmol/L] if measured at the time of symptoms) within the last year, despite nutrition management
- •c. Ongoing liver injury, defined as alanine aminotransferase (ALT) \> 2\.5x the upper limit of normal (ULN) within the last year
- •3\. ALT \= 5x ULN during the 3 months prior to the Baseline Visit
- •4\. Males or females \= 18 years of age
- •5\. After nutrition optimization (if necessary) for subjects in the repeat dose (RD) Period, during the beginning of the Screening Period, willing and able to maintain nutritional intake consistent with the nutrition guidelines based on expert recommendations for the remainder of the study
- •6\. Willing and able to provide access to medical records surrounding medical treatment that occurred prior to enrollment
- •7\. Willing and able to provide written informed consent, or in the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any test procedures or assessments
Exclusion Criteria
- •1\. History of liver transplant, including hepatocyte cell therapy/transplant, or active listing for liver transplant
- •2\. History of cirrhosis, or presence of any of the following:
- •a. Total bilirubin \= 1\.3 mg/dL and international normalized ratio (INR) \= 1\.3
- •b. Evidence of portal hypertension, including, but not limited to the following symptoms splenomegaly, ascites, thrombocytopenia, esophageal varices, or history of hepatic encephalopathy
- •c. Model for End Stage Liver Disease (MELD) score \> 12
- •3\. Current Hepatitis B or C infection or history of chronic Hepatitis B or C infection
- •4\. Severe renal impairment defined as a glomerular filtration rate (GFR) \= 29 mL/min
- •5\. Any prior history of hepatocellular carcinoma or presence of liver adenoma \> 5 cm at the longest diameter or \> 3 cm and \= 5 cm in size that has an annual growth rate of \= 0\.5 cm per year
- •6\. Current or history of malignancies in the 3 years prior to the Screening Visit (ISV for RD)
- •7\. Hospitalizations related to GSD III disease between the Screening (ISV for RD) and Baseline Visit
Outcomes
Primary Outcomes
Not specified
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