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Gene Therapy

EDIT-301 for Beta Thalassemia

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

Diagnosis of Transfusion Dependent B-Thalassemia as defined by documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records

History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 months post edit-301 infusion through up to 24 months post edit-301 infusion

Awards & highlights

Study Summary

This trial is testing a new drug, EDIT-301, to treat people with beta thalassemia who need transfusions. They will evaluate how safe and effective the drug is.

Who is the study for?

This trial is for adults with Transfusion-Dependent Beta Thalassemia, who have needed regular blood transfusions and are in stable condition to undergo a stem cell transplant. They should not have had previous transplants or gene therapy, no significant organ issues, infections, other hemoglobin disorders, cancer or immunodeficiency.Check my eligibility

What is being tested?

The study tests EDIT-301's safety and effectiveness in treating Beta Thalassemia. Participants will receive this treatment before undergoing an autologous Hematopoietic Stem Cell Transplant (HSCT), where they use their own stem cells.See study design

What are the potential side effects?

While specific side effects of EDIT-301 aren't listed here, similar treatments may cause immune reactions, infection risk increase due to bone marrow involvement, fatigue from the procedure and potential organ inflammation.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with Transfusion Dependent B-Thalassemia.

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I've had significant blood transfusions in the last 2 years.

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I am mostly able to care for myself.

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I am healthy enough for a stem cell transplant using my own cells.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 months post edit-301 infusion through up to 24 months post edit-301 infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 months post edit-301 infusion through up to 24 months post edit-301 infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 months post edit-301 infusion through up to 24 months 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

Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)

Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)

Secondary outcome measures

Change in parameters of iron overload compared to baseline over time

Body Weight Changes

Hemoglobin

+9 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 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Editas Medicine, Inc.Lead Sponsor

4 Previous Clinical Trials

159 Total Patients Enrolled

Media Library

EDIT-301 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05444894 — Phase 1 & 2

Beta Thalassemia Research Study Groups: EDIT-301

Beta Thalassemia Clinical Trial 2023: EDIT-301 Highlights & Side Effects. Trial Name: NCT05444894 — Phase 1 & 2

EDIT-301 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05444894 — 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 are the eligibility criteria for participating in this research?

"This clinical study requires 6 individuals aged 18 to 35, who have hemoglobinopathies, and fulfill other specific conditions. These include being homozygous or compound heterozygous for β-thalassemia/Hemoglobin E based on medical documentation as well as having received 100 mL/kg of packed red blood cells per annum in the two years preceding consenting to participate. Additionally, participants must be clinically stable and possess a Karnofsky Performance Status of at least 70%."

Answered by AI

What is the current enrollment rate for this trial?

"Affirmative, the publicly accessible information on clinicaltrials.gov reveals that this medical experiment is actively taking applicants. First posted on April 29th 2022 and most recently updated October 10th of the same year, it requires 6 volunteers at 2 different sites."

Answered by AI

Are there still openings for individuals to participate in this research endeavor?

"Affirmative. According to the information found at clinicaltrials.gov, this experiment is still in search of volunteers and was initially uploaded on April 29th 2022 with a most recent revision on October 10th 2022. The investigation requires 6 participants between 2 medical sites."

Answered by AI

Are seniors excluded from participating in this experiment?

"This trial specifically requires that participants must be aged between 18 to 35. Additionally, there are 55 separate studies for minors and 23 trials for senior citizens over the age of 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

2022. "EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05444894.

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