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Recruiting NCT06735755

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)

Phase I / Phase II Interventional Glycogen Storage Disease Type Ia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: BEAM-301: Single dose of BEAM-301 administered by IV.
Who it may be relevant to
Registry conditions: Glycogen Storage Disease Type Ia. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

  • Drug BEAM-301: Single dose of BEAM-301 administered by IV
    BEAM-301 is designed to correct the G6PC1 c.247C\>T allele via an A:T-to-G:C base-pair substitution, resulting in restoration of G6Pase-α catalytic activity.

Primary outcome measures

  • To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL [Time frame: BEAM-301 administration through month 24]
Secondary outcome measures (6)
  • Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge [Time frame: Baseline to month 24]
  • Changes from baseline in starch supplementation dose [Time frame: Baseline to month 24]
  • Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time [Time frame: Baseline to month 24]
  • BEAM-301 PK parameters (Cmax) [Time frame: Baseline to month 24]
  • BEAM-301 PK parameters (AUC) [Time frame: Baseline to month 24]
  • BEAM-301 PK parameters (half-life) [Time frame: Baseline to month 24]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

  • Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
  • 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.
  • Presence of liver adenoma >3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
  • Have aspartate transaminase or alanine transaminase >upper limit of normal (ULN).
  • Total bilirubin levels >ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
  • Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
  • 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).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 3 centers
  • Clinical Study Site — Orange
  • Clinical Study Site — Farmington
  • Clinical Study Site — Houston

Identifiers

NCT: NCT06735755 · BTX-301-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗