CompletedPhase 1/2

Stem Cell Gene Therapy for Cystinosis

United States6 participantsStarted 2019-07-08

Plain-language summary

This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Cohorts 1 and 2: Male or female subject is ≥ 18 years of age.
✓. Cohort 3: Male or female subject is ≥ 14 years of age.
✓. Subject is diagnosed with cystinosis, i.e., early onset of Fanconi syndrome, and history of elevated white blood cell cystine level and/or history of or presence of cystine crystals in the eye.
✓. Subject has a Karnofsky Performance Status or age-dependent Lansky Performance of ≥ 60.
✓. If subject has had a kidney transplant, he or she must be at least one-year post kidney transplant status.
✓. Subject has adequate hematologic function:
✓. Absolute neutrophil count (ANC) ≥ 1.5 x 1000/mm\^3
✓. Platelet count ≥ 100 x 1000/mm\^3

Exclusion criteria

✕. Subject has an active, uncontrolled, acute bacterial, viral, or fungal infection during screening or within 30 days prior to starting the conditioning regimen.
✕. Subject has positive serology at screening for any of the following:
✕. Human Immunodeficiency Virus (HIV) 1-2
✕. Human T-cell Lymphotropic Virus (HTLV) - I/II
✕. Hepatitis B core and Hepatitis B PCR positive

What they're measuring

Safety and tolerability: Total number of adverse events (AEs) per participant (pre- and post-dose to end of study)

Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.

Safety and tolerability: Aggregate number of adverse events (AEs) by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) severity (pre-dose and post-dose to end of study)

Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.

Safety and tolerability: Aggregate number of adverse events (AEs) by relationship to study treatment (pre-dose and post-dose to end of study)

Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.

Safety and tolerability: Clinical laboratory values for white blood cell, absolute monocyte, and platelet counts at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.

Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion

Safety and tolerability: Systolic and diastolic blood pressure at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.

Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion

Trial details

NCT IDNCT03897361

SponsorUniversity of California, San Diego

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-09-18

View on ClinicalTrials.gov More Lysosomal Storage Diseases trials

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Cohorts 1 and 2: Male or female subject is ≥ 18 years of age.
✓. Cohort 3: Male or female subject is ≥ 14 years of age.
✓. Subject is diagnosed with cystinosis, i.e., early onset of Fanconi syndrome, and history of elevated white blood cell cystine level and/or history of or presence of cystine crystals in the eye.
✓. Subject has a Karnofsky Performance Status or age-dependent Lansky Performance of ≥ 60.
✓. If subject has had a kidney transplant, he or she must be at least one-year post kidney transplant status.
✓. Subject has adequate hematologic function:
✓. Absolute neutrophil count (ANC) ≥ 1.5 x 1000/mm\^3
✓. Platelet count ≥ 100 x 1000/mm\^3

Exclusion criteria

✕. Subject has an active, uncontrolled, acute bacterial, viral, or fungal infection during screening or within 30 days prior to starting the conditioning regimen.
✕. Subject has positive serology at screening for any of the following:
✕. Human Immunodeficiency Virus (HIV) 1-2
✕. Human T-cell Lymphotropic Virus (HTLV) - I/II
✕. Hepatitis B core and Hepatitis B PCR positive