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Gene Therapy

Gene Therapy for Sickle Cell Disease (GRASP Trial)

Phase 2

Recruiting

Research Sponsored by David Williams

Eligibility Criteria Checklist

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

Must have

Age between 13-40 years

Clinically severe disease, defined as at least 4 vaso-occlusive events (VOEs) within the past 24 months prior to consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months prior to consent and 6 months to 24 months post-infusion of gene modified cells

Awards & highlights

GRASP Trial Summary

This trial is testing a new way to treat sickle cell disease that involves changing the patient's own genes to try to cure the disease.

Who is the study for?

This trial is for people aged 13-40 with severe sickle cell disease (HbSS or HbS/β0 thalassemia) who've had at least 4 pain crises in the last 2 years. They need good organ function, no matching bone marrow donor, and can't be on chronic blood transfusions or have a history of stroke, certain infections like HIV/Hepatitis, liver issues from iron overload, or other conditions that could interfere with treatment.Check my eligibility

What is being tested?

The study tests gene therapy to increase fetal hemoglobin which doesn't sickle. Patients' own stem cells are modified using a virus vector targeting BCL11A gene to reduce sickling hemoglobin levels. This could potentially cure or improve their condition without needing a donor and may use less chemotherapy.See study design

What are the potential side effects?

Potential side effects include those related to chemotherapy used before transplanting the modified cells back into patients such as nausea, hair loss, increased infection risk; and any unknown risks associated with the new gene therapy approach.

GRASP Trial 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 had 4 or more severe pain episodes in the last 2 years.

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I don't have a family member who is a perfect match for a bone marrow donation.

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I have sickle cell disease with either HbSS or HbS/β0 thalassemia genotype.

GRASP Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months prior to consent and 6 months to 24 months post-infusion of gene modified cells

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months prior to consent and 6 months to 24 months post-infusion of gene modified cells

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months prior to consent and 6 months to 24 months post-infusion of gene modified cells for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Occurrence of VOEs by Month 24 post-infusion

Secondary outcome measures

Hemoglobin Function

Hemolysis

Occurrence of VOEs by Month 18 post-infusion

+2 more

GRASP Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment ArmExperimental Treatment1 Intervention

Open-label, non-randomized, single arm study of a single infusion of autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

David WilliamsLead Sponsor

4 Previous Clinical Trials

33 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

64 Previous Clinical Trials

3,132 Total Patients Enrolled

bluebird bioIndustry Sponsor

20 Previous Clinical Trials

2,018 Total Patients Enrolled

Media Library

Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05353647 — Phase 2

Sickle Cell Disease Research Study Groups: Treatment Arm

Sickle Cell Disease Clinical Trial 2023: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a Highlights & Side Effects. Trial Name: NCT05353647 — Phase 2

Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05353647 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are new patients being enrolled in this research project?

"This information is accurate according to the clinicaltrials.gov website. The study was first posted on 7/12/2022 and was last edited on 10/10/2022."

Answered by AI

How many volunteers are needed for this research project?

"That is correct. The online clinicaltrials.gov registry reflects that this study is still recruiting patients, with an updated posted on 10/10/2022. This particular trial began posting seeking participants on 7/12/2022 and plans to enroll 25 patients across 10 sites."

Answered by AI

Does this research include geriatric patients?

"Eligibility for this trial starts at age 13 and ends at 40. In total, there are 133 studies currently underway for patients under 18 and 85 for senior citizens."

Answered by AI

Where are the different places this trial is being conducted?

"10 hospitals are currently participating in this trial, with locations including but not limited to Milwaukee, Los Angeles and Chicago. To limit participant burden, it is best to select the location nearest you."

Answered by AI

Which type of patient would be a good fit for this particular medical study?

"This study is testing a new medication for anemia, sickle cell. The ideal candidate for this clinical trial is between the ages of 13 and 40. So far, around 25 people have been accepted into the program."

Answered by AI

Are patients in danger when they receive Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a?

"This therapy, which is currently in Phase 2 clinical trials, has not yet been proven effective. Although, there is data supporting its safety profile; our team gives it a score of 2."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

David Williams 2022. "A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05353647.

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