RECRUITING

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients with Glycogen Storage Disease Type Ia (GSDIa)

Conditions

Glycogen Storage Disease Type Ia GSDIa GSD1a

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.

Official Title

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients with Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 C.247C>T (p.R83C) Variant

Quick Facts

Study Start:2024-12-06

Study Completion:2027-12-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06735755

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Males or females age ≥18 years of age at the time of consent. 2. Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant (confirmed by genetic testing). 3. History of at least 1 episode of hypoglycemia \<60 mg/dL within the 2 years prior to signing the ICF.	1. Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis. 2. Presence of liver adenoma \>5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening. 3. Presence of liver adenoma \>3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year. 4. Have aspartate transaminase or alanine transaminase \>upper limit of normal (ULN). 5. Total bilirubin levels \>ULN; if documented Gilbert's Syndrome, total bilirubin \>2 × ULN. 6. Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF. 7. Have triglycerides \>1000 mg/dL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).

Inclusion Criteria

Exclusion Criteria

1. Males or females age ≥18 years of age at the time of consent.
2. Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant (confirmed by genetic testing).
3. History of at least 1 episode of hypoglycemia \<60 mg/dL within the 2 years prior to signing the ICF.

1. Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
2. Presence of liver adenoma \>5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening.
3. Presence of liver adenoma \>3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
4. Have aspartate transaminase or alanine transaminase \>upper limit of normal (ULN).
5. Total bilirubin levels \>ULN; if documented Gilbert's Syndrome, total bilirubin \>2 × ULN.
6. Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
7. Have triglycerides \>1000 mg/dL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).

Contacts and Locations

Study Contact

Medical Information

CONTACT

857-327-8641

clinicalinfo@beamtx.com

Study Locations (Sites)

Clinical Study Site

Farmington, Connecticut, 06030

United States

Collaborators and Investigators

Sponsor: Beam Therapeutics Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12-06

Study Completion Date2027-12-30

Study Record Updates

Study Start Date2024-12-06

Study Completion Date2027-12-30

Terms related to this study

Keywords Provided by Researchers

GSDIa
GSD1a

Additional Relevant MeSH Terms

Glycogen Storage Disease Type Ia