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)
← catalyst calendarNCT06735755 · readout in 498 d
Sponsored by Beam Therapeutics Inc. (industry) · BEAM — their whole pipeline →.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
36estimated
Sites
3
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-12-06 | actual | When the study began enrolling |
| Primary completion | 2027-12-30 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2027-12-30 | estimated | The whole study's end, after follow-up |
| First posted | 2024-12-16 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-12-15 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Glycogen Storage Disease Type Ia
Intervention
- Drug: BEAM-301: Single dose of BEAM-301 administered by IV
Primary outcome
what the primary-completion date above is the date OFTo 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
Permalink · BEAM's whole pipeline · Catalyst calendar · Every registered study · What changed