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A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

NCT05778877 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2024-09-24

No results posted yet for this study

Summary

This Phase 1/2 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.

Conditions

  • Methylmalonic Acidemia (MMA)

Interventions

DRUG

SEL-302

SEL-302 Drug: MMA-101 (1.0E13 vg/kg) Drug: SEL-110 (0.15 mg/kg or up to 0.3 mg/kg) Other Names: SEL-110.36, ImmTOR

Sponsors & Collaborators

  • National Human Genome Research Institute (NHGRI)

    collaborator NIH

  • Selecta Biosciences, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-19
Primary Completion
2025-08-31
Completion
2029-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05778877 on ClinicalTrials.gov