Ignite Creation Date:
2024-05-06 @ 6:45 PM
Last Modification Date:
2024-10-26 @ 2:54 PM
Study NCT ID:
NCT05778877
Status:
WITHDRAWN
Last Update Posted:
2023-05-08
First Post:
2023-01-23
Brief Title:
A Study to Evaluate the Safety Tolerability and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA
Sponsor:
Selecta Biosciences Inc
Organization:
Selecta Biosciences Inc
Study Overview
Official Title:
Phase 12 Study to Evaluate the Safety Tolerability and Pharmacodynamics of SEL-302 MMA-101 Following Administration of SEL-110 in Pediatric Subjects With Mut Subtype Isolated Methylmalonic Acidemia MMA
Status:
WITHDRAWN
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Decided by sponsor
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
reiMMAgine
Brief Summary:
This Phase 12 study will evaluate the safety and pharmacodynamics PD of SEL-302 which consists of the gene transfer vector MMA-101 following administration of an immunomodulatory SEL-110 agent in pediatric subjects with Methylmalonyl-CoA Mutase MMUT MMA
Detailed Description:
MMA is a rare inborn error of branched chain amino acid metabolism Despite strict dietary adherence and vigilant monitoring and care affected individuals have recurrent episodes of severe illness and develop complications from different organ systems that can be life-threatening Liver transplants can help but gene transfer therapy could offer an alternative treatment option This study will be an open-label single dose single center study of SEL-302 consisting of two investigative therapeutics a gene transfer therapy that is using an inactive virus called adeno-associated-virus 8 AAV8 to deliver the MMUT gene to the liver by itself called MMA-101 and an immunotherapy called SEL-110 a nano-encapsulated form of sirolimus
The study will enroll two cohorts treating up to a total of 6 subjects
Cohort 1 3 adolescents 12 and 18 years of age Cohort 2 3 children 3 and 12 years of age with a minimum body weight of 15 kg
The dose of MMA-101 administered to each subject will be 10E13 vgkg Each progression to the next subject dosed in the study will be reviewed and approved by a data safety monitoring committee
The first subject in Cohort 1 will receive only MMA-101 The second adolescent subject in Cohort 1 will be treated with 015 mgkg of SEL-110 followed by MMA-101 on Day 1 and two repeat doses of 015 mgkg of SEL-110 at Day 28 and Day 56 The dose of SEL-110 in the third subject in Cohort 1 may be increased up to 03 mgkg depending on results from the second subject After assessment of safety and efficacy of Cohort 1 Cohort 2 will be started in younger children
The dose of SEL-110 in Cohort 2 for the first subject will be 015 mgkg of SEL-110 immediately prior to the dose of MMA-101 on Day 1 and two repeat doses of 015 mgkg of SEL-110 at Day 28 and Day 56 The dose of SEL-110 in the second and third subject in Cohort 2 may be increased up to 03 mgkg at one or more of the three doses depending on results of all previously treated subjects
The primary efficacy endpoints of reduction in serum methylmalonic acid sMMA levels and increases in the 1-13C sodium propionate oxidation breath test POBT will be assessed at an interim timepoint for safety evaluation Day 84 and at the primary endpoint of 1 year
All subjects will be monitored closely for safety for the first year of the study and then annually for an additional 4 years
The study will enroll two cohorts treating up to a total of 6 subjects
Cohort 1 3 adolescents 12 and 18 years of age Cohort 2 3 children 3 and 12 years of age with a minimum body weight of 15 kg
The dose of MMA-101 administered to each subject will be 10E13 vgkg Each progression to the next subject dosed in the study will be reviewed and approved by a data safety monitoring committee
The first subject in Cohort 1 will receive only MMA-101 The second adolescent subject in Cohort 1 will be treated with 015 mgkg of SEL-110 followed by MMA-101 on Day 1 and two repeat doses of 015 mgkg of SEL-110 at Day 28 and Day 56 The dose of SEL-110 in the third subject in Cohort 1 may be increased up to 03 mgkg depending on results from the second subject After assessment of safety and efficacy of Cohort 1 Cohort 2 will be started in younger children
The dose of SEL-110 in Cohort 2 for the first subject will be 015 mgkg of SEL-110 immediately prior to the dose of MMA-101 on Day 1 and two repeat doses of 015 mgkg of SEL-110 at Day 28 and Day 56 The dose of SEL-110 in the second and third subject in Cohort 2 may be increased up to 03 mgkg at one or more of the three doses depending on results of all previously treated subjects
The primary efficacy endpoints of reduction in serum methylmalonic acid sMMA levels and increases in the 1-13C sodium propionate oxidation breath test POBT will be assessed at an interim timepoint for safety evaluation Day 84 and at the primary endpoint of 1 year
All subjects will be monitored closely for safety for the first year of the study and then annually for an additional 4 years
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 000957-HG | OTHER | NHGRI | None |