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)
| NCT ID | NCT06735755 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Beam Therapeutics Inc. |
| Condition | Glycogen Storage Disease Type Ia |
| Study Type | INTERVENTIONAL |
| Enrollment | 36 participants |
| Start Date | 2024-12-06 |
| Primary Completion | 2027-12-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 36 participants in total. It began in 2024-12-06 with a primary completion date of 2027-12-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06735755 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Glycogen Storage Disease Type Ia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06735755 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06735755 currently recruiting?
Yes, NCT06735755 is actively recruiting participants. Contact the research team at clinicalinfo@beamtx.com for enrollment information.
Where is the NCT06735755 trial being conducted?
This trial is being conducted at Orange, United States, Farmington, United States, Houston, United States.
Who is sponsoring the NCT06735755 clinical trial?
NCT06735755 is sponsored by Beam Therapeutics Inc.. The trial plans to enroll 36 participants.