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

NCT04443907 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2026-01-08

No results posted yet for this study

Summary

This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Conditions

Interventions

BIOLOGICAL

OTQ923

Single intravenous infusion of OTQ923 cell suspension

BIOLOGICAL

OTQ923

Single intravenous infusion of OTQ923, based on review of data from Part A by Health agencies after a formal interim analysis

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-25
Primary Completion
2025-01-06
Completion
2025-01-06
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04443907 on ClinicalTrials.gov