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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Other: OTQ923


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Accepted invitation to join based on prior treatment with gene therapy. 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)

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| OTQ923<br>Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Enrolled patients on this study will not be administered any study treatment. | Other: OTQ923<br>* There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gene therapy, genome-edited hematopoietic stem and progenitor cellular therapy, sickle cell, autologous transplant, BCL11A