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Gene Therapy

EDIT-301 for Sickle Cell Disease

Phase 1 & 2

Recruiting

Research Sponsored by Editas Medicine, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Documented severe SCD genotype (βS/βS, βS/β0, or βS/β+)

Karnofsky Performance Status ≥ 80

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years post edit-301 infusion

Awards & highlights

Study Summary

This trial is testing a new drug to treat sickle cell disease in adults. The goal is to see if it is safe and effective.

Who is the study for?

This trial is for adults with severe sickle cell disease who have had at least two major pain episodes per year despite treatment. They must be generally well-functioning (Karnofsky Performance Status ≥ 80). People can't join if they've had a stem cell transplant, have certain allergies or infections, advanced liver disease, cancer, immune disorders, or an HLA-matched donor.Check my eligibility

What is being tested?

The study tests EDIT-301's effectiveness and safety in treating severe sickle cell disease. Participants will receive this experimental therapy to see how well it works and what side effects it may cause compared to standard treatments.See study design

What are the potential side effects?

Potential side effects of EDIT-301 could include reactions related to the mobilization of hematopoietic stem cells using plerixafor and myeloablative conditioning with busulfan. Specific side effects are not listed but would relate to these procedures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a severe form of sickle cell disease.

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I can carry out normal activities with minimal symptoms.

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I've had 2 or more severe pain crises a year despite treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years post edit-301 infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years post edit-301 infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years post edit-301 infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)

Secondary outcome measures

Change from baseline in HbF concentration (g/dL)

Change from baseline in markers of hemolysis (absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, haptoglobin)

Change from baseline in total Hb concentration (g/dL)

+14 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: EDIT-301Experimental Treatment1 Intervention

EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Editas Medicine, Inc.Lead Sponsor

4 Previous Clinical Trials

123 Total Patients Enrolled

Media Library

EDIT-301 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04853576 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: EDIT-301

Sickle Cell Disease Clinical Trial 2023: EDIT-301 Highlights & Side Effects. Trial Name: NCT04853576 — Phase 1 & 2

EDIT-301 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04853576 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the upper cap on participants in this trial?

"This medical trial requires 40 participants meeting its predetermined selection criteria. Localities such as Cook Children's in Fort Worth, TX and UCSF Benioff Children's Hospital in Oakland, CA are available for potential enrolment."

Answered by AI

What is the geographic scope of this trial's management?

"This clinical trial is currently enrolling out of 16 different medical centres, including Cook Children's in Fort Worth, UCSF Benioff Children's Hospital in Oakland, and the esteemed Children's Hospital of Philadelphia. Additionally there are a further 13 sites operating around America."

Answered by AI

Does your study include participants aged 45 or older?

"This medical study has a narrow age range of 18 to 50. There are also other trials available for minors (144) and seniors (85)."

Answered by AI

Who is eligible for enrollment in this clinical experiment?

"The pool of possible candidates for this medical experiment is limited to those between 18 and 50 years old who are diagnosed with a hemoglobinopathy. A total of 40 people need to be recruited in order for the trial to proceed as planned."

Answered by AI

Are there any opportunities to join this experiment at present?

"As per the clinicaltrials.gov, this trial is presently looking for subjects to participate. This research project was first uploaded on May 4th 2021, with its latest changes made October 10th 2022."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

2021. "A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04853576.

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