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Tests treatment safety and results for Glycogen Storage Disease Type Ia
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)
ClinicalTrials.gov ID: NCT06735755
What this study is testing
What is BEAM-301: Single dose of BEAM-301 administered by IV?
BEAM-301: Single dose of BEAM-301 administered by IV is an investigational medicine, being studied as a potential treatment for glycogen storage disease type ia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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.
You likely can't join if
- 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...
See the full eligibility 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.
- 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).
The study team makes the final eligibility decision.
Where it's taking place
- Orange, California, United States
- Farmington, Connecticut, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Orange, California, United States; Farmington, Connecticut, United States; Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.