Methylmalonic Aciduria and Homocystinuria,Cblc Type

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Methylmalonic Aciduria and Homocystinuria,Cblc Type trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT04880356

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less…

Italy100 participantsStarted 2021-03-01

Treatment: collection of data

All recent trials (3 shown)

By InvitationNot Applicable

NCT03655223

Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…

United States30,000 participantsStarted 2018-10-15

Treatment: Confirmatory Testing

CompletedPhase 2

NCT01793090

EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

The aim of the research is to investigate the safety and efficacy of EPI-743 treatment in patients with Cbl-C defect and related visual and neurological impairm…

Italy30 participantsStarted 2013-01

Treatment: Epi-743, Placebo supplementation