Clinical trial 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)
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Glycogen Storage Disease Type Ia
Keywords: GSDIa
GSD1a
Lead sponsor: Beam Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2024-12-16
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: up to 4 planned, single-ascending-dose, dose-exploration cohorts
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion 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.

Exclusion Criteria:

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).

Primary outcomes:
measure: 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
timeFrame: BEAM-301 administration through month 24
Secondary outcomes:
measure: Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge
timeFrame: Baseline to month 24
measure: Changes from baseline in starch supplementation dose
timeFrame: Baseline to month 24
measure: Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time
timeFrame: Baseline to month 24
measure: BEAM-301 PK parameters (Cmax)
timeFrame: Baseline to month 24
measure: BEAM-301 PK parameters (AUC)
timeFrame: Baseline to month 24
measure: BEAM-301 PK parameters (half-life)
timeFrame: Baseline to month 24
Other outcomes:
Locations:
city: Orange
country: United States
facility: Clinical Study Site
state: California
city: Farmington
country: United States
facility: Clinical Study Site
state: Connecticut
Study officials:
References: