TerminatedPhase 1/2

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

Stopped: Due to low likelihood of clinical benefit in treated participants.

United States4 participantsStarted 2021-05-29

Plain-language summary

The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non-disruptively integrate the human methylmalonyl-CoA mutase gene at the albumin locus. The trial is expected to enroll pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years.

Who can participate

Age range6 Months – 12 Years

SexALL

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Inclusion criteria

✓. Isolated MMA with genetically confirmed, pathogenic mutations in the MMUT gene
✓. Screening serum/plasma methylmalonic acid level of \>100 µmol/L
✓. One or more of the following considered by the PI to be MMA-related: (i) An unscheduled ER visit, hospitalization or requirement for sick day diet in the year prior to screening visit (ii) Developmental delay, movement disorder, optic neuropathy or feeding disorder with tube feeding requirement
✓. Medically stable for the 2 months prior to the start of screening

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Timeframe: From first dose of study drug up to Week 52

Number of Participants With Infusional Toxicities

Timeframe: Baseline up to Week 52

Trial details

NCT IDNCT04581785

SponsorLogicBio Therapeutics, Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-01-10

Results submitted2024-01-05

View on ClinicalTrials.gov More Methylmalonic Acidemia trials