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 range
6 Months – 18 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 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
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
1
Proportion of participants achieving normalization of plasma or urinary methylmalonic acid levels post-dose.
. 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.
. Prior liver or kidney transplantation, or any prior cell-based therapy.
. Any of the following laboratory abnormalities:
. Evidence of clinically significant hepatic dysfunction defined as:
. Alanine aminotransferase (ALT) \> 2.0 × upper limit of normal (ULN);
. Aspartate aminotransferase (AST) \> 2.0 × ULN or total bilirubin \> 1.5 × ULN;