Glycogen Storage Disease Type IaPhase 1/2NCT06735755
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)
A Phase 1/2 study testing BEAM-301: Single dose of BEAM-301 administered by IV in people with Glycogen Storage Disease Type Ia, run by Beam Therapeutics Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glycogen Storage Disease Type Ia
- What is being tested?
- BEAM-301: Single dose of BEAM-301 administered by IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 3 sitesUnited States
- Study dates
- 6 Dec 2024to 30 Dec 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Clinical Study Site
Farmington, Connecticut, 06030
- Recruiting
Clinical Study Site
Houston, Texas, 77030
- Recruiting
Clinical Study Site
Orange, California, 92868
About this study
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
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.
- Conditions
- Glycogen Storage Disease Type Ia
- Being tested
- BEAM-301: Single dose of BEAM-301 administered by IV
- Keywords
- GSDIa, GSD1a
- Medical subject headings
- Hepatorenal form of glycogen storage disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BEAM-301: Single dose of BEAM-301 administered by IVDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| BEAM-301 | Experimental | BEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration. Receives: Drug: BEAM-301: Single dose of BEAM-301 administered by IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: BEAM-301 administration through month 24
Secondary outcomes (6)
Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge
Measured: Baseline to month 24
Changes from baseline in starch supplementation dose
Measured: Baseline to month 24
Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time
Measured: Baseline to month 24
BEAM-301 PK parameters (Cmax)
Measured: Baseline to month 24
BEAM-301 PK parameters (AUC)
Measured: Baseline to month 24
BEAM-301 PK parameters (half-life)
Measured: Baseline to month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2024
- Study start6 Dec 2024
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- up to 4 planned, single-ascending-dose, dose-exploration cohorts
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beam Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06735755
- Sponsor's protocol ID
- BTX-301-001
- Status last verified
- Dec 2025
- First submitted
- 6 Dec 2024
- Last updated
- 15 Dec 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06735755
- Acronym / sponsor ID
- BTX-301-001
- Conditions
- Glycogen Storage Disease Type Ia
- Keywords
- GSDIa, GSD1a
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Beam Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Dec 2024 (actual)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 16 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- up to 4 planned, single-ascending-dose, dose-exploration cohorts
| Arm | Type | Receives |
|---|---|---|
| BEAM-301 | Experimental | Drug: BEAM-301: Single dose of BEAM-301 administered by IVBEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BEAM-301: Single dose of BEAM-301 administered by IVBEAM-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. | Drug | BEAM-301 |
Primary outcome
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
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BEAM-301: Single dose of BEAM-301 administered by IV in people with Glycogen Storage Disease Type Ia, run by Beam Therapeutics Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical Information (857-327-8641, clinicalinfo@beamtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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 (time frame: BEAM-301 administration through month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Dec 2025.