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Gene Therapy

Gene Editing for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Akshay Sharma, MBBS

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

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

Must have

Age ≥18 years and ≤24.9 years

Failure, intolerance, or refusal of hydroxyurea therapy

Must not have

Cardiac abnormalities

History of a significant bleeding disorder

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from time of screening to 3 years post infusion

Awards & highlights

No Placebo-Only Group

Summary

"This trial aims to test a new treatment called gene editing in Sickle Cell Disease patients. The goal is to see if a single dose of this treatment can increase the amount of a specific type of hem

Who is the study for?

This trial is for patients with severe Sickle Cell Disease. Participants must have a history of symptoms related to SCD and be eligible for stem cell transplantation. Specific criteria will determine who can join, but details are not provided here.

What is being tested?

The study tests the safety and effectiveness of gene editing on CD34+ cells in SCD patients. It aims to see if this treatment increases fetal hemoglobin levels and reduces disease symptoms using CRISPR/Cas9 technology.

What are the potential side effects?

Potential side effects may include reactions from the infusion process, complications from gene editing like off-target effects, or issues related to Busulfan conditioning used before transplant.

Eligibility Criteria

Inclusion Criteria

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

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I am between 18 and 24 years old.

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I cannot or will not use hydroxyurea due to side effects or personal choice.

Exclusion Criteria

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

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I have heart problems.

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I have a history of serious bleeding problems.

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I have recently undergone a procedure related to a stroke or brain blood flow.

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My liver tests are not normal.

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I have had a stroke in the past.

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My blood cancer is linked to a specific genetic mutation.

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I have a sibling match for a bone marrow transplant.

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I have had a stem cell transplant or gene therapy before.

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My kidneys do not work properly.

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I have lung problems.

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I have an active cancer, blood disorder, genetic abnormalities, or a weak immune system.

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I have Moyamoya disease.

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My daily activity is significantly limited by my health.

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I have liver problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from time of screening to 3 years post infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from time of screening to 3 years post infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and from time of screening to 3 years post infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Frequency of off-target editing after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.

Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.

Incidence of platelet engraftment by day +60 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.

+2 more

Secondary study objectives

Change in incidence of packed RBC transfusions.

Compare the change from baseline in the fraction of red blood cells (RBCs) containing HbF.

Compare the change from baseline in the total blood hemoglobin concentration.

+1 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: Autologous, genetically modified CD34+ HSPCs TreatmentExperimental Treatment3 Interventions

All eligible participants receive intervention as described in the Detailed Description with the following: plerixafor, busulfan, and autologous, gene-modified CD34+ cells

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Plerixafor

2011

Completed Phase 3

~710

Busulfan

2008

Completed Phase 4

~1710