CTX001 for Hematologic Diseases

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy
Hematologic Diseases+5 More
CTX001 - Biological
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study Summary

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Eligible Conditions

  • Hematologic Diseases
  • Thalassemia
  • Hereditary Diseases
  • Hemoglobinopathies

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 15 Secondary · Reporting Duration: Within 42 Days After CTX001 Infusion

Month 24
Change in Fetal Hemoglobin Concentration Over Time
Change in Proportion of Circulating Erythrocytes Expressing Fetal Hemoglobin (F-cells) Over Time
Change in Total Hemoglobin Concentration Over Time
Month 24
Proportion of Participants Achieving at Least 95 Percent (%), 90%, 85%, 75% and 50% Reduction in Annualized Transfusions
Relative Change in Annualized Volume of RBC Transfusions
Month 24
Incidence of All-cause Mortality
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Month 24
Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time
Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time
Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
Proportion of Participants who Achieve Transfusion Independence for at Least 6 Consecutive Months (TI6)
Time to Engraftment
Transfusion Free Duration for Participants who Achieve TI12
Day 100
Incidence of Transplant-related Mortality (TRM) Within 100 Days After CTX001 Infusion
Month 12
Incidence of TRM Within 12 Months After CTX001 Infusion
Day 42
Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count [ANC] ≥500 per Microliter [mcgL] on 3 Different Days)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

1 Treatment Group

CTX001
1 of 1
Experimental Treatment

12 Total Participants · 1 Treatment Group

Primary Treatment: CTX001 · No Placebo Group · Phase 3

CTX001
Biological
Experimental Group · 1 Intervention: CTX001 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: within 42 days after ctx001 infusion
Closest Location: SCRI at the Children's Hospital at TriStar Centennial · Nashville, TN
Photo of Nashville 1Photo of Nashville 2Photo of Nashville 3
N/AFirst Recorded Clinical Trial
2 TrialsResearching Hematologic Diseases
0 CompletedClinical Trials

Who is running the clinical trial?

CRISPR TherapeuticsIndustry Sponsor
5 Previous Clinical Trials
228 Total Patients Enrolled
5 Trials studying Hematologic Diseases
228 Patients Enrolled for Hematologic Diseases
Vertex Pharmaceuticals IncorporatedLead Sponsor
212 Previous Clinical Trials
28,752 Total Patients Enrolled
5 Trials studying Hematologic Diseases
228 Patients Enrolled for Hematologic Diseases

Eligibility Criteria

Age < 18 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are eligible for autologous stem cell transplant as per investigator's judgment.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.