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Gene Therapy

CRISPR-Cas9 Gene Editing for Beta Thalassemia

Phase 2 & 3

Waitlist Available

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

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

Must have

History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening

Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 month post-ctx001 infusion through month 24 visit

Awards & highlights

Study Summary

This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.

Who is the study for?

This trial is for individuals with transfusion-dependent β-thalassemia, which means they need regular blood transfusions due to their condition. They should have a history of significant blood transfusion needs and be suitable for an autologous stem cell transplant. People with certain genetic variations of thalassemia or active infections, low white blood cell or platelet counts can't participate.Check my eligibility

What is being tested?

The study is testing CTX001, which involves modifying the patient's own stem cells using CRISPR-Cas9 technology to potentially treat β-thalassemia. It's a single-dose study looking at both safety and how well it works in improving the condition.See study design

What are the potential side effects?

Potential side effects are not specified here but generally may include reactions related to the modification process of stem cells, body's response to genetically edited cells, and typical risks associated with stem cell transplants such as infection risk.

Eligibility Criteria

Inclusion Criteria

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

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I've had significant blood transfusions in the last 2 years.

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I have been diagnosed with transfusion-dependent β-thalassemia.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 month post-ctx001 infusion through month 24 visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 month post-ctx001 infusion through month 24 visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 month post-ctx001 infusion through month 24 visit for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

All-cause mortality

Frequency and severity of collected adverse events (AEs)

Incidence of transplant-related mortality (TRM)

+3 more

Secondary outcome measures

Change in PRO over time assessed using pediatric quality of life inventory (PedsQL)

Change in fetal hemoglobin concentration over time

Change in health-related quality of life (HRQoL) from baseline over time using EuroQol Questionnaire (5 dimensions - 5 levels of severity - EQ-5D-5L)

+11 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: CTX001Experimental Treatment1 Intervention

CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Subjects will receive a single infusion of CTX001 through a central venous catheter.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,342 Total Patients Enrolled

CRISPR TherapeuticsIndustry Sponsor

5 Previous Clinical Trials

261 Total Patients Enrolled

Media Library

CTX001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03655678 — Phase 2 & 3

Beta Thalassemia Research Study Groups: CTX001

Beta Thalassemia Clinical Trial 2023: CTX001 Highlights & Side Effects. Trial Name: NCT03655678 — Phase 2 & 3

CTX001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03655678 — Phase 2 & 3

Frequently Asked Questions

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

Can you provide more context about CTX001 research?

"CTX001 is currently being investigated in 2 active clinical trials, both of which are Phase 3. There are a total of 29 locations running these clinical trials, although the majority are situated in Rome, Illinois."

Answered by AI

What is the total sample size for this experiment?

"No, this research is no longer looking for new participants. The trial was posted on September 14th, 2018 and updated for the final time on May 26th, 2023. If you are interested in other studies, there are 173 trials actively recruiting patients with hemoglobinopathies and 2 studies searching for patients to participate in CTX001 trials."

Answered by AI

Does this trial mark a new development in medical research?

"CRISPR Therapeutics sponsored the first clinical trial for CTX001 in 2018. The global study completed its Phase 2 & 3 drug approval stage that year and involved 45 participants from 9 cities across 7 countries. Since then, 0 studies have been completed."

Answered by AI

Could you list the hospitals where this clinical trial is taking place?

"There are 6 clinics participating in this trial, 3 of which are located in Vancouver, Chicago and Manhattan. The other locations for the remaining sites have yet to be decided but will be released soon. If you enroll in the program, it is best to choose a clinic near your residence to limit travel."

Answered by AI

Does this experiment only test on elderly individuals?

"The age bracket for patients that this study is recruiting falls between 12 years old to 35 years old."

Answered by AI

If a patient meets the requirements, can they join this trial?

"This study is no longer recruiting patients. Although it was originally posted on September 14th, 2018, the last edit occurred on May 26th, 2022. There are currently 175 other trials looking for participants with hemoglobinopathies and 2 trials specifically for CTX001 that are actively searching for patients."

Answered by AI

Are there any specific inclusions or exclusions for this research?

"This study is looking for 45 patients with hemoglobinopathies between the ages of 12 and 35. To be eligible, patients must have either documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning., History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last"

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

Crispr-cas9 Gene Editing for Sickle Cell Disease and Β-thalassemia

Frangoul, Haydar, David Altshuler, M. Domenica Cappellini, Yi-Shan Chen, Jennifer Domm, Brenda K. Eustace, Juergen Foell, et al.. 2021. “Crispr-cas9 Gene Editing for Sickle Cell Disease and Β-thalassemia”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa2031054.

Progress in Molecular Biology and Translational ScienceJournal

Genome Editing Approaches to Β-hemoglobinopathies

Brusson, Mégane, and Annarita Miccio. 2021. “Genome Editing Approaches to Β-hemoglobinopathies”. Progress in Molecular Biology and Translational Science. Elsevier. doi:10.1016/bs.pmbts.2021.01.025.

clinicaltrials.govStudy

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

2018. "A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03655678.

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