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Gene Therapy

Stem Cell Gene Therapy for Sickle Cell Disease

Phase 1 & 2

Recruiting

Led By Gary Schiller, MD

Research Sponsored by Donald B. Kohn, M.D.

Eligibility Criteria Checklist

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

Must have

Inadequate clinical response to hydroxyurea (HU), defined as any one of the following outcomes, while on HU for at least 3 months:

- 2 or more acute sickle pain crises requiring hospitalization

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

Study Summary

This trial will test a new treatment for sickle cell disease that uses the patient's own blood cells.

Who is the study for?

Adults with severe sickle cell disease who have had complications like pain crises, stroke, or acute chest syndrome despite treatment can join this trial. They must not be pregnant, have HIV/HCV/HTLV-1/hepatitis B/Cancer, and should not have a suitable sibling donor for transplant.Check my eligibility

What is being tested?

The trial is testing a gene therapy where patients' own blood stem cells are modified with the βAS3-FB vector to potentially treat sickle cell disease. It's in Phase I to check safety and initial effectiveness.See study design

What are the potential side effects?

Potential side effects may include reactions at the infusion site, immune response against the modified cells, or other unforeseen issues due to genetic modification of blood cells.

Eligibility Criteria

Inclusion Criteria

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

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My condition did not improve after taking hydroxyurea for 3 months.

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I have been hospitalized for sickle cell pain crises at least twice.

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I can care for myself but may need occasional help.

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I am 18 years old or older.

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I've had a severe lung issue not caused by asthma in the last 2 years despite treatment.

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I have had a stroke or a lasting brain problem for more than a day.

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I had episodes of swollen fingers or toes in my childhood.

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I have severe bone tissue death.

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I have been receiving regular blood transfusions for over a year to manage my sickle cell disease.

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My hemoglobin levels have not increased by more than 1.5 gm/dl from the baseline, or I need transfusions to keep my hemoglobin above 6.0 gm/dL.

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My sickle cell disease is confirmed by genetic testing.

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I was hospitalized for a severe chest problem in the last 2 years.

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I have been hospitalized for sickle cell pain crises at least twice in the last 2 years.

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I have had priapism at least twice.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Evaluation of Safety

Trial Design

1Treatment groups

Experimental Treatment

Group I: βAS3-FB vector transduced peripheral blood CD34+ cellsExperimental Treatment1 Intervention

This is a single arm study without randomization. All subjects will receive the intervention of BetaAS3 lentiviral vector-modified autologous peripheral blood stem cell transplant.

0 / 14Eligibility criteria met

Find a Location

Who is running the clinical trial?

Donald B. Kohn, M.D.Lead Sponsor

1 Previous Clinical Trials

10 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

64 Previous Clinical Trials

3,151 Total Patients Enrolled

Giulia Parisi, PhDStudy DirectorUniversity of California, Los Angeles

Media Library

βAS3-FB vector transduced peripheral blood CD34+ cells (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02247843 — Phase 1 & 2

Sickle Cell Disease Clinical Trial 2023: βAS3-FB vector transduced peripheral blood CD34+ cells Highlights & Side Effects. Trial Name: NCT02247843 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: βAS3-FB vector transduced peripheral blood CD34+ cells

βAS3-FB vector transduced peripheral blood CD34+ cells (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02247843 — Phase 1 & 2

Frequently Asked Questions

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

Is this research effort recruiting new participants?

"This research endeavour, which was initially made available for public access on December 1st 2014, is currently looking to enrol participants. The trial listing has been updated as recently as April 9th 2022 according to clinicaltrials.gov."

Answered by AI

How many participants have been recruited for this trial thus far?

"Affirmative. According to the information posted on clinicaltrials.gov, this medical trial is actively looking for participants and was first introduced in December 2014 before being recently amended in April 2022. This research requires 6 volunteers from a single location."

Answered by AI

Recent research and studies

Journal of Clinical InvestigationJournal

Β-globin Gene Transfer to Human Bone Marrow for Sickle Cell Disease

Romero, Zulema, Fabrizia Urbinati, Sabine Geiger, Aaron R. Cooper, Jennifer Wherley, Michael L. Kaufman, Roger P. Hollis, et al.. 2013. “Β-globin Gene Transfer to Human Bone Marrow for Sickle Cell Disease”. Journal of Clinical Investigation. American Society for Clinical Investigation. doi:10.1172/jci67930.

clinicaltrials.govStudy

Stem Cell Gene Therapy for Sickle Cell Disease

Donald B. Kohn, M.D. 2014. "Stem Cell Gene Therapy for Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02247843.

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