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Gene Therapy

Genome-Edited Cells for Sickle Cell Disease

Q: How many locations are participating in the evaluation process?

This clinical trial is enrolling participants at Childrens Hospital Los Angeles Dept.ofChildrensHospital/LA in the Golden State, Memorial Sloan Kettering <a href="https://www.withpower.com/clinical-trials/cancer">Cancer</a> Center within the Empire State of <a href="https://www.withpower.com/clinical-trials/new-york">New york</a>, University of Chicago SC - 2 in <a href="https://www.withpower.com/clinical-trials/illinois">Illinois</a> and 7 additional sites across America.

Q: Is enrollment for this trial still available?

As documented on clinicaltrials.gov, recruitment for this trial is ongoing. It was first published on August 26th 2020 and has recently been edited as of September 2nd 2022.

Q: How large is the pool of participants involved in this experiment?

Novartis Pharmaceuticals, the sponsor of this research project, is looking for 20 volunteers that meet their inclusion criteria. The study will be conducted at two different sites: Children's Hospital Los Angeles Dept. ofChildrensHospital/LA in <a href="https://www.withpower.com/clinical-trials/california">California</a> and Memorial Sloan Kettering <a href="https://www.withpower.com/clinical-trials/cancer">Cancer</a> Centre in <a href="https://www.withpower.com/clinical-trials/new-york">New york</a>.

Q: Are octogenarians being included in the investigation?

The requirements for this study stipulate that candidates must be aged between 2 and 40. Additionally, 131 clinical trials are available to those below 18 years of age while 85 studies exist for those over 65.

Q: What aims are being pursued through this research endeavor?

This 24-month trial primarily aims to measure the amount of fetal hemoglobin expression among participants. Secondary objectives include overall survival, annualized <a href="https://www.withpower.com/clinical-trials/sickle-cell">sickle cell</a> disease complications rate, a transfusion rate reduction by 65%, and an evaluation of effect on age-appropriate patient reported outcomes using ASCQ-ME <a href="https://www.withpower.com/clinical-trials/pain">pain</a> impact instruments.

Q: Do I qualify for entry into this investigation?

This clinical trial seeks 20 individuals with <a href="https://www.withpower.com/clinical-trials/sickle-cell-anemia">sickle cell anemia</a> aged between 2 and 40. To be considered for the study, patients must meet one of several criteria including acute chest syndrome, a history of recurrent priapism, prior <a href="https://www.withpower.com/clinical-trials/stroke">stroke</a> or chronic transfusions as well as being unresponsive to hydroxyurea therapy.

Phase 1

Waitlist Available

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Subjects who have failed, not tolerated or refused hydroxyurea therapy

Performance status >70% (Karnofsky for subjects >16 years of age and Lansky for subjects <16 years of age)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

Study Summary

This trial is testing a new treatment for sickle cell disease that involves editing the patient's cells to reduce the activity of a protein that causes sickle cell disease.

Who is the study for?

This trial is for people aged 2-40 with sickle cell disease who've had severe symptoms like pain crises, acute chest syndrome, or stroke. It's open to those who can't tolerate or haven't benefited from hydroxyurea therapy and have a performance status over 70%. People with liver issues, iron overload, certain infections or cancers, previous transplants or gene therapy are not eligible.Check my eligibility

What is being tested?

The study tests OTQ923 - a genome-edited stem cell treatment aimed at increasing fetal hemoglobin to alleviate sickle cell complications. Participants receive their own modified cells back to potentially reduce the severity of their condition.See study design

What are the potential side effects?

While specific side effects of OTQ923 aren't listed here, similar treatments may cause immune reactions, infection risk due to bone marrow suppression during stem cell transplant preparation, and potential long-term risks related to genome editing.

Eligibility Criteria

Inclusion Criteria

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

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I have stopped or refused hydroxyurea due to side effects or choice.

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I am mostly active and can care for myself.

Select...

I have had severe symptoms like pain crises, chest issues, or needed regular blood transfusions.

Select...

I am between 2 and 40 years old.

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I have been diagnosed with sickle cell disease.

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

Fetal hemoglobin (HbF) expression after hematopoietic stem cell transplant (HSCT)

Number of participants with adverse events and serious adverse events

Time to reach absolute neutrophil count (ANC) ≥500/μL for 3 consecutive days

Secondary outcome measures

Durability of hematologic engraftment

Evaluation of effect on patient-reported outcomes from baseline and post-HSCT with age appropriate patient reported measures

Number of participants with change from baseline of annualized SCD complications (aggregate of VOC, ACS, priapism and stroke) and if relevant, rate of transfusion by 65%

+8 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: OTQ923Experimental Treatment1 Intervention

Single intravenous infusion of OTQ923 Part A - Adults treated with OTQ923; Part B - Children age 2-17 treated with OTQ923 based on review of data from Part A by Health agency after a formal interim analysis.

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor

2,855 Previous Clinical Trials

4,197,348 Total Patients Enrolled

Media Library

Sickle Cell Disease Research Study Groups: OTQ923

Sickle Cell Disease Clinical Trial 2023: HIX763 Highlights & Side Effects. Trial Name: NCT04443907 — Phase 1

HIX763 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04443907 — Phase 1

Frequently Asked Questions

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

How many locations are participating in the evaluation process?

"This clinical trial is enrolling participants at Childrens Hospital Los Angeles Dept.ofChildrensHospital/LA in the Golden State, Memorial Sloan Kettering Cancer Center within the Empire State of New york, University of Chicago SC - 2 in Illinois and 7 additional sites across America."

Answered by AI

Is enrollment for this trial still available?

"As documented on clinicaltrials.gov, recruitment for this trial is ongoing. It was first published on August 26th 2020 and has recently been edited as of September 2nd 2022."

Answered by AI

How large is the pool of participants involved in this experiment?

"Novartis Pharmaceuticals, the sponsor of this research project, is looking for 20 volunteers that meet their inclusion criteria. The study will be conducted at two different sites: Children's Hospital Los Angeles Dept. ofChildrensHospital/LA in California and Memorial Sloan Kettering Cancer Centre in New york."

Answered by AI

Are octogenarians being included in the investigation?

"The requirements for this study stipulate that candidates must be aged between 2 and 40. Additionally, 131 clinical trials are available to those below 18 years of age while 85 studies exist for those over 65."

Answered by AI

What aims are being pursued through this research endeavor?

"This 24-month trial primarily aims to measure the amount of fetal hemoglobin expression among participants. Secondary objectives include overall survival, annualized sickle cell disease complications rate, a transfusion rate reduction by 65%, and an evaluation of effect on age-appropriate patient reported outcomes using ASCQ-ME pain impact instruments."

Answered by AI

Do I qualify for entry into this investigation?

"This clinical trial seeks 20 individuals with sickle cell anemia aged between 2 and 40. To be considered for the study, patients must meet one of several criteria including acute chest syndrome, a history of recurrent priapism, prior stroke or chronic transfusions as well as being unresponsive to hydroxyurea therapy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

2020. "Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04443907.

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