A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients W… (NCT06735755) | Clinical Trial Compass
RecruitingPhase 1/2
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)
United States36 participantsStarted 2024-12-06
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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
. 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).