Ignite Creation Date:
2024-05-06 @ 12:58 PM
Last Modification Date:
2024-10-26 @ 1:07 PM
Study NCT ID:
NCT03897361
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-12
First Post:
2019-02-06
Brief Title:
Stem Cell Gene Therapy for Cystinosis
Sponsor:
University of California San Diego
Organization:
University of California San Diego
Study Overview
Official Title:
A Phase 12 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34 Hematopoietic Stem Cells HSC From Mobilized Peripheral Blood Stem Cells PBSC of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDYEFSCTNST260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is a Phase 12 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
Detailed Description:
Cystinosis is a rare inherited recessive disease belonging to the family of Lysosomal Storage Disorders and is characterized by lysosomal accumulation of cystine in all the cells of the body leading to multi-organ failure Cystinosis has a devastating impact on the affected individuals primarily children and young adults even with cysteamine treatment The prevalence of cystinosis is 1 in 100000 to 1 in 200000 The gene involved in cystinosis is the gene CTNS that encodes for the transmembrane lysosomal cystine transporter - cystinosin The current standard of care does not prevent the progression of the disease and significantly impacts the quality of life of patients with cystinosis
For this study up to 6 subjects meeting eligibility criteria will be transplanted following a 3-cohort staggered treatment design with 2 subjects per cohort The first 2 cohorts will consist of 4 adults 18 years or older potentially followed by a cohort consisting of 2 adolescents or adults 14 years old Following the informed consent process enrolled subjects will be screened to confirm full eligibility for participation Eligible subjects will undergo hematopoietic stem cell HSC mobilization and collection leukapheresis A portion of cells will be kept as back-up for rescue purpose if necessary and a portion will be ex vivo gene-modified with a lentiviral vector pCCL-CTNS or pCDYEFSCTNST260I to express CTNS gene product name CTNS-RD-04 Clinical manufacturing for patients in Cohort 3 will introduce a transduction enhancer LentiBOOST product name for these patients will be CTNS-RD-04-LB where the suffix -LB stands for LentiBOOST The subjects will receive marrow cytoreduction with busulfan prior to infusion of CTNS-RD-04 Subjects will discontinue cysteamine treatment during the assessment period The assessment follow-up period will include an initial 2 years of active end-point evaluations where the subjects will be evaluated at 3- 6- 9- 12- 18- and 24-months post-transplantation A Long-Term Follow-Up study LTFU for a total 15-year follow-up period will be offered to all subjects
The objectives of this Phase 12 clinical study are to assess the safetytolerability of CTNS-RD-04 and its efficacy through a number of clinical molecular and biochemical assessments
For this study up to 6 subjects meeting eligibility criteria will be transplanted following a 3-cohort staggered treatment design with 2 subjects per cohort The first 2 cohorts will consist of 4 adults 18 years or older potentially followed by a cohort consisting of 2 adolescents or adults 14 years old Following the informed consent process enrolled subjects will be screened to confirm full eligibility for participation Eligible subjects will undergo hematopoietic stem cell HSC mobilization and collection leukapheresis A portion of cells will be kept as back-up for rescue purpose if necessary and a portion will be ex vivo gene-modified with a lentiviral vector pCCL-CTNS or pCDYEFSCTNST260I to express CTNS gene product name CTNS-RD-04 Clinical manufacturing for patients in Cohort 3 will introduce a transduction enhancer LentiBOOST product name for these patients will be CTNS-RD-04-LB where the suffix -LB stands for LentiBOOST The subjects will receive marrow cytoreduction with busulfan prior to infusion of CTNS-RD-04 Subjects will discontinue cysteamine treatment during the assessment period The assessment follow-up period will include an initial 2 years of active end-point evaluations where the subjects will be evaluated at 3- 6- 9- 12- 18- and 24-months post-transplantation A Long-Term Follow-Up study LTFU for a total 15-year follow-up period will be offered to all subjects
The objectives of this Phase 12 clinical study are to assess the safetytolerability of CTNS-RD-04 and its efficacy through a number of clinical molecular and biochemical assessments
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None