BEAM-101 for Anemia, Sickle Cell

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Boston Children's Hospital, Boston, MA
Anemia, Sickle Cell+1 More
BEAM-101 - Biological
Eligibility
18 - 65
All Sexes
What conditions do you have?
Select

Study 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

Eligible Conditions

  • Anemia, Sickle Cell

Treatment Effectiveness

Study Objectives

6 Primary · 12 Secondary · Reporting Duration: Month 6 post BEAM-101 treatment to month 24 as compared to baseline

Month 6
Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline
Proportion of patients experiencing no severe VOCs
Month 24
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)
Day 100
Transplant-related mortality within 100 days after beam-101 treatment
Month 24
Proportion of patients with successful neutrophil engraftment
Time to neutrophil engraftment
Time to platelet engraftment
Baseline to month 24
Change in total Hgb (g/dL) concentration over time
Month 24
Change in RBC transfusions per month and per year for SCD-related indications
Month 24
Change in free Hgb over time
Change in haptoglobin over time
Change in lactate dehydrogenase (LDH) over time
Change in reticulocyte count over time
Change in total bilirubin over time
Month 24
Change in annualized duration of hospitalizations for VOCs
Change in annualized number of hospitalizations for VOCs
Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs
Proportion of patients with HbF ≥30%, for at least 3 months

Trial Safety

Trial Design

1 Treatment Group

BEAM-101
1 of 1
Experimental Treatment

15 Total Participants · 1 Treatment Group

Primary Treatment: BEAM-101 · No Placebo Group · Phase 1 & 2

BEAM-101
Biological
Experimental Group · 1 Intervention: BEAM-101 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: month 6 post beam-101 treatment to month 24 as compared to baseline
Closest Location: Boston Children's Hospital · Boston, MA
Photo of Boston 1Photo of Boston 2Photo of Boston 3
2020First Recorded Clinical Trial
16 TrialsResearching Anemia, Sickle Cell
349 CompletedClinical Trials

Who is running the clinical trial?

Beam Therapeutics Inc.Lead Sponsor

Eligibility Criteria

Age 18 - 65 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.