NCT07163364

A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type)

A Single-arm Phase III Clinical Study to Evaluate the Efficacy and Safety of Hydroxocobalamin Chloride Injection in Participants With Methylmalonic Acidemia (MMA) With Elevated Homocysteine (Cobalamin C Deficiency)

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: CSPC ZhongQi Pharmaceutical Technology Co., Ltd. · Industry
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Methylmalonic Acidemia (MMA)

Interventions

Type	Name	Description
DRUG	Hydroxocobalamin Chloride Injection	1 - 20 mg per dose, 1 - 5 times per week

Timeline

• Start date: 2025-08-31
• Primary completion: 2026-10-08
• Completion: 2027-04-28
• First posted: 2025-09-09
• Last updated: 2025-09-09