A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Meth… (NCT07163364) | Clinical Trial Compass
Not Yet RecruitingPhase 3
A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type)
20 participantsStarted 2025-08-31
Plain-language summary
This study is a Single-Center, Single-Arm, open-label, Phase III clinical study to evaluate the efficacy, safety characteristics of Hydroxocobalamin Chloride Injection (20 mg/mL) for Maintenance Therapy in participants with Methylmalonic Acidemia (MMA) with Elevated Homocysteine (Cobalamin C Deficiency).
Who can participate
Age range6 Months – 18 Years
SexALL
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Inclusion criteria
✓. Age 6 months (inclusive) to \< 18 years at the time of first investigational product administration; both sexes eligible.
✓. Confirmed diagnosis of cobalamin C (cbl C)-type methylmalonic acidemia (MMA) fulfilling ALL of the following:
✓. Documented vitamin B12 responsiveness: ≥ 50 % reduction from pre-treatment baseline in plasma C3/C2 ratio and urinary methylmalonic acid following vitamin B12 therapy.
✓. Presence of pathogenic MMACHC gene variants in a participant with MMA associated with hyperhomocysteinemia.
✓. Investigator-assessed clinical stability, defined as:
✓. Continuous treatment with injectable hydroxocobalamin for ≥ 3 months immediately preceding first dose of study drug.
✓. Written informed consent obtained from participant and/or legally authorised representative; willingness and ability to comply with all study visits and procedures.
✓. Female participants of childbearing potential (post-menarche) must have a negative serum β-hCG test at screening. All participants of reproductive potential (post-menarche females or males with documented spermarche) must use a highly effective contraceptive method throughout the study and for an appropriate post-study period as defined by local regulations.
Exclusion criteria
✕. Use of any vitamin B12 preparation other than injectable hydroxocobalamin within 3 months prior to screening.
✕. Participation in another clinical trial within 28 days or 5 half-lives of the investigational agent (whichever is longer) before screening initiation, except for screening-only participants who did not receive study drug.
What they're measuring
1
Proportion of participants achieving normalization of plasma or urinary methylmalonic acid levels post-dose.