Active — Not RecruitingPhase 1/2

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

United States15 participantsStarted 2022-08-30

Plain-language summary

This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD

Who can participate

Age range12 Years – 35 Years

SexALL

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

✓. Age ≥12 years to ≤35 years
✓. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
✓. Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures

Exclusion criteria

✕. HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy
✕. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
✕. Available and willing matched sibling donor
✕. Definitive diagnosis of moyamoya syndrome based on screening brain MRA
✕. History of overt stroke

What they're measuring

Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline

Timeframe: 6 months to time of analysis as compared to baseline

Proportion of patients with successful neutrophil engraftment

Timeframe: BEAM-101 administration to month 24

Time to neutrophil engraftment

Timeframe: BEAM-101 administration to month 24

Time to platelet engraftment

Timeframe: BEAM-101 administration to month 24

Transplant-related mortality within 100 days after beam-101 treatment

Timeframe: BEAM-101 administration to day 100

Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)

Timeframe: BEAM-101 administration through month 24

Trial details

NCT IDNCT05456880

SponsorBeam Therapeutics Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-02

View on ClinicalTrials.gov More Sickle Cell Disease trials

Who can participate

Age range12 Years – 35 Years

SexALL

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

✓. Age ≥12 years to ≤35 years
✓. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
✓. Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures

Exclusion criteria

✕. HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy
✕. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
✕. Available and willing matched sibling donor
✕. Definitive diagnosis of moyamoya syndrome based on screening brain MRA
✕. History of overt stroke

What they're measuring

Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline

Timeframe: 6 months to time of analysis as compared to baseline

Proportion of patients with successful neutrophil engraftment

Timeframe: BEAM-101 administration to month 24

Time to neutrophil engraftment

Timeframe: BEAM-101 administration to month 24

Time to platelet engraftment

Timeframe: BEAM-101 administration to month 24

Transplant-related mortality within 100 days after beam-101 treatment

Timeframe: BEAM-101 administration to day 100

Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)

Timeframe: BEAM-101 administration through month 24