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Clinical Trials in the USA / NCT06155500
Active, not recruiting Phase 1

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

NCT06155500 · tracked via the Priya Life Science USA tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 1
Started
2024-04-16
Last updated
2026-07-28

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

OTQ923

OTQ923: There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)

Study summary

CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.

Eligibility

Sex
ALL
Min age
18 Years
Max age
100 Years
Healthy volunteers
No
Inclusion Criteria: 1. Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101). 2. Patients must provide informed consent prior to their entry into this study. Exclusion Criteria: 1\. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).

Primary outcome measure(s)

  • Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy — Up to 15 years
    Number of participants with delayed adverse events including new secondary malignancies, new incidence or exacerbation of a prior autoimmune disorder, new incidence or exacerbation of a prior rheumatologic disorder, new hematologic disorder, and other adverse events considered to be related to OTQ923 therapy.

Trial sites (3)

FacilityCityRegionStatus
University of Chicago Chicago Illinois
Memorial Sloan Kettering Cancer Ctr New York New York
St Jude Childrens Research Hospital Memphis Tennessee

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Two Arm, Double-blind, Phase III Study Assessing Efficacy and Safety of Ianalumab Versus Placebo, in Participants With Sjögren's Disease With High Symptom Burden Recruiting

Other trials for the same condition

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease Recruiting
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Recruiting
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) Recruiting
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease Recruiting
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06155500 on ClinicalTrials.gov ↗ ← All trials in the USA