What side effects are associated with this type of intervention?

The most common treatments for Sickle Cell Disease include hydroxyurea, blood transfusions, and investigational therapies like gene editing. Hydroxyurea can cause side effects such as bone marrow suppression, leading to lower blood cell counts, gastrointestinal disturbances, and potential long-term risks like leukemia. Blood transfusions carry risks of iron overload, alloimmunization, and transfusion reactions. Gene editing therapies, such as those using CRISPR/Cas9, are still under investigation, but potential side effects may include off-target genetic modifications and immune responses to the introduced components. Long-term safety data for gene editing are still needed.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease, including hydroxyurea, which reduces vaso-occlusive pain and other complications, and chronic blood transfusions for managing severe cases. Hematopoietic stem cell transplantation, including gene therapy and gene editing approaches, is a potential curative option, though not yet in routine use. These advanced therapies aim to correct the underlying genetic mutations causing SCD, similar to the investigational treatment EDIT-301, which uses gene editing to correct the hemoglobin mutation.

What other types of research exist?

Promising alternatives to EDIT-301 for Sickle Cell Disease treatment in 2024 include: 1) CRISPR/Cas9 gene editing, which is being actively researched to correct the genetic mutations causing SCD. 2) Hydroxyurea, an established treatment that reduces complications and may increase life expectancy, especially in severe cases. 3) Ongoing gene therapy trials targeting hematopoietic stem cells to prevent erythrocyte sickling. These alternatives offer a range of options from established therapies to cutting-edge genetic treatments.

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Gene Therapy

EDIT-301 for Sickle Cell Disease

Phase 1 & 2

Waitlist Available

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

Must not have

Inadequate end organ function

Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells (HSCs) and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My organs are not functioning properly.

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

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

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I cannot or will not adjust my current medications for a stem cell transplant.

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I have a history of severe brain blood vessel problems.

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I do not have any active and serious infections.

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I have had a stem cell transplant or cannot have one due to health reasons.

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I have a family donor who is a perfect match for me.

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I cannot receive blood transfusions.

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My liver disease is in an advanced stage.

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A close family member has or might have a hereditary cancer syndrome.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, blood transfusions, and gene therapy/editing. Hydroxyurea works by increasing fetal hemoglobin production, which reduces the sickling of red blood cells and decreases vaso-occlusive episodes. Blood transfusions help by diluting the sickled cells with normal red blood cells, reducing the risk of complications like stroke. Gene therapy/editing, such as the investigational treatment EDIT-301, aims to correct the hemoglobin mutation at the genetic level, potentially offering a curative approach by enabling the production of normal hemoglobin. These treatments are crucial for SCD patients as they help manage pain, prevent complications, and improve overall quality of life.

Gene therapy for sickle cell disease.Advances in the management of sickle cell disease.