EDIT-301 for Sickle Cell Disease

Recruiting at 23 trial locations

Overseen By Editas Medicine's Clinical Trial Team

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Editas Medicine, Inc.

Must not be taking: Hydroxyurea, Voxelotor, Crizanlizumab, L-glutamine

Disqualifiers: HLA-matched donor, Prior HSCT, Vasculopathy, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

45 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.

Will I have to stop taking my current medications?

Yes, you will need to stop taking certain medications like hydroxyurea, voxelotor, crizanlizumab, or L-glutamine as part of the trial requirements.

What makes the treatment EDIT-301 unique for sickle cell disease?

EDIT-301 is a novel treatment for sickle cell disease that uses gene editing to modify the patient's own cells, potentially offering a more targeted and long-lasting solution compared to traditional treatments like hydroxyurea or blood transfusions. ^{1 2 3 4 5}

Eligibility Criteria

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.

Inclusion Criteria

I have a severe form of sickle cell disease.

I can carry out normal activities with minimal symptoms.

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

Exclusion Criteria

My organs are not functioning properly.

I am not allergic to plerixafor, busulfan, or any related medications.

I have or had another cancer or a condition that weakens my immune system.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of autologous gene-edited CD34+ hematopoietic stem cells (EDIT-301) via intravenous infusion

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Treatment Details

Interventions

• EDIT-301

Trial Overview 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.

Participant Groups

Group I: EDIT-301 Experimental Treatment 1 Intervention

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

EDIT-301 is already approved in United States, Canada for the following indications:

Approved in United States as EDIT-301 for:

• None approved yet; currently in clinical trials for Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Approved in Canada as EDIT-301 for:

• None approved yet; currently in clinical trials for Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Who Is Running the Clinical Trial?

Editas Medicine, Inc.

Lead Sponsor

Trials

5

Recruited

170+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

An Analysis of Racial and Ethnic Backgrounds Within the CASiRe International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

[Sickle cell disease]. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Newborn screening for sickle cell diseases in the United States: A review of data spanning 2 decades. [2015]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Systematic Literature Review Shows Gaps in Data on Global Prevalence and Birth Prevalence of Sickle Cell Disease and Sickle Cell Trait: Call for Action to Scale Up and Harmonize Data Collection. [2023]