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Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

NCT02426775 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2019-04-25

No results posted yet for this study

Summary

A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients who had received Carbaglu® for 1 to 15 days.

There is no current evidence supporting the use of carglumic acid for the chronic management of patients with OA. The investigators are proposing a randomized multicentre prospective clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined with standard chronic therapy in patients with PA and MMA compared to standard chronic therapy alone.

Conditions

Interventions

DRUG

Carglumic Acid

Carglumic Acid 50 mg/kg/day (twice daily)

Sponsors & Collaborators

  • King Abdullah International Medical Research Center

    lead OTHER

Principal Investigators

  • Majid A. Alfadhel, MD · King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-11-30
Primary Completion
2019-03-19
Completion
2019-03-19

Countries

  • Saudi Arabia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02426775 on ClinicalTrials.gov