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Gene Editing

Gene Editing (CRISPR) for Sickle Cell Disease

Phase 1 & 2
Recruiting
Research Sponsored by Mark Walters, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Karnofsky performance score ≥60.
History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea);
Must not have
Females who are pregnant or breast feeding.
Participants with evidence of HIV infection or seropositivity for HIV or active hepatitis B or C.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, and 1 and 2 years post-transplant
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a one-time treatment for patients with severe Sickle Cell Disease. The treatment aims to correct the genetic defect causing their disease, allowing their bodies to produce healthy red blood cells. The study will initially focus on ensuring the treatment is safe in older patients before expanding to younger ones.

Who is the study for?
This trial is for individuals aged 12 to 35 with severe Sickle Cell Disease who've had multiple pain events or acute chest syndrome despite treatment, and have good kidney, liver, heart, and lung function. It's not for those with certain infections, pregnant or breastfeeding women, men unwilling to use contraception, or anyone who has received a transplant.
What is being tested?
The study tests a one-time infusion of CRISPR_SCD001-modified stem cells in patients with severe Sickle Cell Disease. This gene-editing approach aims to correct the sickle cell allele in hematopoietic stem cells to alleviate disease symptoms.
What are the potential side effects?
Potential side effects are not explicitly listed but may include typical risks associated with stem cell transplantation such as immune reactions, infection risk increase due to immunosuppression during the procedure and potential off-target genetic effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can care for myself but may need occasional help.
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I've had two or more acute chest syndrome episodes in the last 2 years despite treatment.
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I've had 4 or more severe pain crises due to sickle cell in the last 2 years despite treatment.
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My lung function tests show my oxygen levels and breathing capacity are within safe limits.
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My heart's pumping ability is within a healthy range.
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I am between 12 and 34 years old.
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My kidney function tests are within normal limits.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not pregnant or breastfeeding.
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I do not have HIV or active hepatitis B or C.
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My sickle cell disease is not the HbSS type.
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I have had a stroke or I get blood transfusions to prevent strokes.
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I have a sibling donor who is a perfect HLA match.
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I haven't had an uncontrolled infection in the last 6 weeks.
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I have received a stem cell transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, and 1 and 2 years post-transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, and 1 and 2 years post-transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of adverse events and grade 3 or higher serious adverse events, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE)
Secondary study objectives
Change in the annualized vaso-occlusive pain event (VOE) rates.
Frequency of central nervous system (CNS) toxicity (reversible posterior leukoencephalopathy syndrome [RPLS] or posterior reversible encephalopathy syndrome [PRES], hemorrhage, and seizures).
Frequency of cytomegalovirus (CMV) infection, invasive fungal infection, and any other serious viral or bacterial infection
+9 more
Other study objectives
Patient-reported quality of life (pain and fatigue domains) as measured by the use of Patient Reported Outcome Measurement Information System (PROMIS) modules.
Rate of sickle-related events other than severe VOE and end organ function.

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: CRISPR_SCD001 Drug ProductExperimental Treatment1 Intervention
CRISPR_SCD001 Drug Product (autologous CD34+ cell-enriched population that contains cells modified by the CRISPR-Cas9 ribonucleoprotein) dose will be ≥3.0×106 CD34+ cells/kg recipient weight for each subject and the upper limit cell dose is 20 ×106 CD34+ cells/kg.

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
The most common treatments for Sickle Cell Disease (SCD) include hydroxyurea, blood transfusions, and gene therapy. Hydroxyurea works by increasing fetal hemoglobin (Hb F) levels, which reduces the sickling of red blood cells and decreases vaso-occlusive events. Blood transfusions lower the proportion of sickle hemoglobin (Hb S) in the blood, helping to prevent complications such as pain crises and stroke. Gene therapy, including CRISPR/Cas9 gene editing, aims to correct the sickle cell mutation in hematopoietic stem cells, potentially offering a long-term cure by enabling the production of normal hemoglobin. These treatments are vital for SCD patients as they address the root causes of the disease, improving quality of life and reducing the risk of severe complications.

Find a Location

Who is running the clinical trial?

Mark Walters, MDLead Sponsor
University of California, BerkeleyOTHER
187 Previous Clinical Trials
640,967 Total Patients Enrolled
University of California, Los AngelesOTHER
1,569 Previous Clinical Trials
10,314,280 Total Patients Enrolled

Media Library

CRISPR_SCD001 (Gene Editing) Clinical Trial Eligibility Overview. Trial Name: NCT04774536 — Phase 1 & 2
Sickle Cell Disease Research Study Groups: CRISPR_SCD001 Drug Product
Sickle Cell Disease Clinical Trial 2023: CRISPR_SCD001 Highlights & Side Effects. Trial Name: NCT04774536 — Phase 1 & 2
CRISPR_SCD001 (Gene Editing) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04774536 — Phase 1 & 2
~6 spots leftby Mar 2027