WithdrawnPhase 1/2

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

Stopped: Decided by sponsor

United States0Started 2022-12-19

Plain-language 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.

Who can participate

Age range

3 Years – 17 Years

Sex

ALL

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

. Age 3 to \<18 years at time of consent (assent where possible)
. Confirmed diagnosis of MMUT type methylmalonic acidemia by molecular genetic testing
. Clinical and biochemical diagnosis of severe MMA as defined by:
. sMMA level between 100 to 3,000 μmol/L
. A clinical history consistent with severe MMA
. 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.
. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Safety and tolerability of SEL-302

Timeframe: From the initial administration of SEL-302 up to 5 years for long-term follow-up.

PD Activity of SEL-302

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

PD Activity of SEL-302

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

Assess the change in Neutralizing antibody (Nab) titers for MMA-101 with treatment of SEL-110

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

Trial details

NCT IDNCT05778877

SponsorSelecta Biosciences, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-08

View on ClinicalTrials.gov More Methylmalonic Acidemia (MMA) trials

Who can participate

Age range

3 Years – 17 Years

Sex

ALL

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

. Age 3 to \<18 years at time of consent (assent where possible)
. Confirmed diagnosis of MMUT type methylmalonic acidemia by molecular genetic testing
. Clinical and biochemical diagnosis of severe MMA as defined by:
. sMMA level between 100 to 3,000 μmol/L
. A clinical history consistent with severe MMA
. 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.
. 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.

What they're measuring

Safety and tolerability of SEL-302

Timeframe: From the initial administration of SEL-302 up to 5 years for long-term follow-up.

PD Activity of SEL-302

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

PD Activity of SEL-302

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

Assess the change in Neutralizing antibody (Nab) titers for MMA-101 with treatment of SEL-110

Timeframe: From initial treatment with SEL-302 up to 5 years for long-term follow-up.

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria