Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

• Status: Recruiting
• Trial end date: 2029-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Selecta Biosciences, Inc.
• Collaborator: National Human Genome Research Institute (NHGRI)

Criteria

Inclusion Criteria:

1. Age 3 to <18 years at time of consent (assent where possible)

2. Confirmed diagnosis of MMUT type methylmalonic acidemia by molecular genetic testing

3. Clinical and biochemical diagnosis of severe MMA as defined by:

1. sMMA level between 100 to 3,000 μmol/L

2. A clinical history consistent with severe MMA

3. Subjects must have fully recovered from any hospitalization for metabolic ketoacidosis or surgery at least 4 weeks prior to the start of the screening period.

4. Parent or legal guardian are willing and able to provide informed consent. Written assent will be obtained from minors older than age seven whenever possible.

5. Subject and caregiver must be willing to comply with study-related assessments and adhere to lifestyle considerations throughout study duration.

Exclusion Criteria:

1. History of any organ transplantation.

2. High MMUT liver enzymatic activity in the range seen in healthy subjects or MMA patients after corrective liver transplant, as demonstrated by POBT levels.

3. Presence of Nab against AAV8 or polyethylene glycol (PEG)

4. An estimated glomerular filtration rate (GFR)<45 mL/min/1.73 m2 (disease stage 3a)

5. Hemoglobin <10 g/dL

6. Platelet count <100,000 per mm3

7. History of any malignancy or immunocompromising condition.

8. History of anaphylaxis or severe allergic reaction to drug therapy, foods, PEG or polysorbates.

9. Previously received gene therapy or messenger ribonucleic acid (mRNA) treatments for MMA.

10. Participated in a clinical trial of another (non-gene or mRNA therapy) investigational agent within 30 days prior to screening, or within 5 elimination half-lives of the investigational agent, whichever is longer.

Note: additional inclusion/exclusion criteria may apply, per protocol.