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Gene Therapy

Gene Transfer for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Erica Esrick, MD

Research Sponsored by David Williams

Eligibility Criteria Checklist

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

Must have

Age 3 years to 40 years

Failure of hydroxyurea therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

Awards & highlights

Study Summary

This trial will test whether using gene therapy to change the expression of a particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.

Who is the study for?

This trial is for people aged 3-40 with severe Sickle Cell Disease (SCD) who've had multiple pain events, acute chest syndrome, or need chronic transfusions. They must have tried hydroxyurea without success and can't have a matching bone marrow donor. Participants should be in stable health otherwise and willing to follow up for 15 years.Check my eligibility

What is being tested?

The trial tests gene therapy using the patient's own modified blood stem cells to treat SCD. It involves a single infusion of these engineered cells aiming to increase fetal hemoglobin production which could potentially cure SCD.See study design

What are the potential side effects?

Potential side effects may include reactions similar to those experienced during bone marrow transplants such as infection risk due to immune suppression from chemotherapy, discomfort at the infusion site, and possible organ inflammation.

Eligibility Criteria

Inclusion Criteria

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

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I am between 3 and 40 years old.

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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.

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I have sickle cell disease with a specific genetic type.

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I have severe symptoms from sickle cell disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Rescue of hematopoiesis after conditioning

Secondary outcome measures

Expression of transgene

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment armExperimental Treatment1 Intervention

open-label, non-randomized, single center, pilot and feasibility, single arm cohort study of a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with lentiviral vector containing a short-hairpin RNA targeting BC11A.

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

David WilliamsLead Sponsor

4 Previous Clinical Trials

48 Total Patients Enrolled

Erica Esrick, MDPrincipal InvestigatorBoston Children's Hospital

1 Previous Clinical Trials

6 Total Patients Enrolled

Media Library

Gene Transfer for Sickle Cell Disease (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03282656 — Phase 1

Sickle Cell Disease Research Study Groups: Treatment arm

Sickle Cell Disease Clinical Trial 2023: Gene Transfer for Sickle Cell Disease Highlights & Side Effects. Trial Name: NCT03282656 — Phase 1

Gene Transfer for Sickle Cell Disease (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03282656 — Phase 1

Frequently Asked Questions

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

Are there still opportunities for participants to enroll in this research study?

"Unfortunately, clinicaltrials.gov information indicates that this trial is not presently accepting patients; the initial post date was February 13th 2018 and it was last updated on August 2nd 2022. Nevertheless, 197 other studies are actively recruiting participants at the moment."

Answered by AI

To what degree do autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a pose risks to patients upon single infusion?

"Assessing the safety of single infusion autologous bone marrow derived CD34+ HSC cells transduced with a lentiviral vector containing a short-hairpin RNA targeting BCL11a, our team at Power has given it an overall score of 1. This is due to this being Phase I trial, which indicates that there is limited research regarding both efficacy and safety."

Answered by AI

Is there a way for me to become involved with this clinical research?

"This clinical trial is actively looking for 10 participants, aged between 3 and 40 years old, suffering from anemia or sickle cell. Furthermore, potential enrollees must have experienced three or more severe pain events in the last two years that required hospitalisation with parenteral opioids; had at least two episodes of acute chest syndrome during this time frame; recurrent priapism twice over the same period; red-cell alloimmunization due to long term transfusions; been prescribed chronic blood transfusions as primary or secondary stroke prophylaxis; failed hydroxyurea therapy due to lack of efficacy or side effects despite having"

Answered by AI

Does the age restriction for this medical trial restrict those younger than thirty?

"This medical study welcome participants aged between 3 and 40. Those younger than 18 are eligible for 131 different trials, whereas those older than 65 can apply to 85 alternative studies."

Answered by AI

Recent research and studies

bcl11aJournal

Post-transcriptional Genetic Silencing Of<i>bcl11a</i>to Treat Sickle Cell Disease

Esrick, Erica B., Leslie E. Lehmann, Alessandra Biffi, Maureen Achebe, Christian Brendel, Marioara F. Ciuculescu, Heather Daley, et al.. 2021. “Post-transcriptional Genetic Silencing Ofbcl11ato Treat Sickle Cell Disease”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa2029392.

clinicaltrials.govStudy

Gene Transfer for Sickle Cell Disease

David Williams 2018. "Gene Transfer for Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03282656.

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