Evaluation of response rate to hydroxycobalamin treatment in patients with methylmalonic acidemia
Phase 3
Recruiting
- Conditions
- methylmalonic acidemia.E71.120
- Registration Number
- IRCT20211207053311N1
- Lead Sponsor
- Mashhad University of Medical Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 15
Inclusion Criteria
patients with isolated methylmalonic acidemia or methylmalonic acidemia with hemocyctinuria confirmed in MS/MS and urine organic acid or genetic
Exclusion Criteria
suspicious patients that have not certain diagnosis and poor control patients that have not cooperation
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Decrease more than 50 percent in urine methylmalonic acid considered positive and the patient is responsible to hydroxycobalamine. Timepoint: urine methylmalonic acid before starting the intervention and then 3 days after received hydroxycobalamine in stable patients and after 7 days in unstable patients. Method of measurement: evaluation of urine methylmalonic acid.
- Secondary Outcome Measures
Name Time Method