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Gene Therapy

Gene Therapy for Sickle Cell Anemia

Phase 1
Waitlist Available
Research Sponsored by Daniel Bauer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 13-40 years
Confirmed sickle cell disease or β-thalassemia diagnosis by molecular genetic testing
Must not have
Major surgery in the past 30 days
Contraindication to administration of conditioning medication (busulfan)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
No Placebo-Only Group

Summary

This trial explores a new treatment called gene therapy for genetic diseases. Gene therapy involves using the patient's own genetic material to fix or replace a diseased gene with the goal of curing the disease. The

Who is the study for?
This trial is for individuals with severe beta-thalassemia or sickle cell disease. Candidates must have a specific genetic variant (rs114518452). They should be eligible for bone marrow transplant but will use their own modified cells to avoid complications like GVHD.
What is being tested?
The trial tests gene editing on the patient's own blood stem cells, aiming to increase fetal hemoglobin production which could cure the disease. It involves modifying genes using Cas9 and then reintroducing these cells after chemotherapy.
What are the potential side effects?
Potential side effects may include those associated with bone marrow transplants such as infection risk due to immune suppression from chemotherapy, reactions at the infusion site, and possible off-target gene editing effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 13 and 40 years old.
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I have been diagnosed with sickle cell or β-thalassemia through genetic testing.
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I have been diagnosed with a specific type of sickle cell disease or β-thalassemia.
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My blood cell counts are within the required range.
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Hydroxyurea treatment did not work for me.
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I don't have a family member who is a perfect match for a bone marrow donation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have not had major surgery in the last 30 days.
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I cannot take busulfan due to health reasons.
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I have a high level of iron in my body.
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I have had a stem cell transplant.
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I do not have any uncontrolled infections, active cancers, or blood disorder complications.
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I have severe blood vessel problems in my brain, receive regular blood transfusions, or have had a stroke due to sickle cell disease.
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I carry a specific genetic variation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Primary engraftment
Secondary study objectives
Acute chest syndrome (for sickle cell disease (SCD) patients)
Bilirubin (for sickle cell disease (SCD) patients)
Hemoglobin
+11 more

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: Sickle Cell Disease and Transfusion-Dependent Beta-ThalassemiaExperimental Treatment2 Interventions

Find a Location

Who is running the clinical trial?

Daniel BauerLead Sponsor
~7 spots leftby Dec 2028
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