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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06155500
• Other study ID #: CADPT03A12001
• Status: Recruiting
• Start date: April 16, 2024
• Est. completion date: January 11, 2039

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: 1-888-669-6682
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

This study is monitoring patients treated with OTQ923, an investigational drug product of ex vivo genome-edited autologous hematopoietic stem and progenitor cells (HSPCs) that induces fetal hemoglobin (HbF) production, for a total of 15 years following infusion to monitor long-term safety and efficacy.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: January 11, 2039
• Est. primary completion date: January 11, 2039
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Accepted invitation to join based on prior treatment with gene therapy.

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)

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

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

Locations

Country	Name	City	State
United States	St Jude Childrens Rsrch Hospital	Memphis	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy	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.	Up to 15 years
Secondary	Persistence of fetal hemoglobin expression	Assessment of fetal hemoglobin (HbF) expression persistence in peripheral blood will be done by assessing hemoglobin fractionation	Up to 15 years
Secondary	WBC chimerism in peripheral blood	Concern for loss of engraftment by monitoring the WBC chimerism annually. Modified white blood cells (WBCs) measured by droplet digital PCR (ddPCR) and NGS will be performed annually through year 5 with subsequent samples stored and run annually in the event of concern for loss of engraftment	5 years