CompletedPhase 3

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Saudi Arabia33 participantsStarted 2015-11

Plain-language 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.

Who can participate

Age range15 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female Children of 15 years old or less * Parent or legal guardian agree to participate and to sign the (Institutional Review Board) IRB approved Informed Consent Form (assent forms will be waived due to the mental disability of those children) * Not participating in any other clinical trial in the previous 30 days * PA or MMA confirmed using the following criteria: * PA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of propionyl Co-A carboxylase in leukocytes or cultured fibroblasts or by DNA molecular testing of PCCA (Propionyl CoA Carboxylase, Alpha Polypeptide) or PCCB (propionyl CoA carboxylase, beta polypeptide) gene * MMA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of methymalonyl Co-A mutase in culture fibroblasts or DNA molecular testing of mutgene. * Expected survival of at least 6 months, for the purpose of this study Survival expectance will be defined as patient not admitted to the Pediatric Intensive Care Unit (PICU) due to hyperammonemia more than 2 times per year or asymptomatic patients diagnosed by newborn screening program or stable chronic patients who are followed up at outpatient clinic. Exclusion Criteria: * Patients with other organic acidemia or any other cause of hyperammonemia * Patient receiving other investigational therapy for PA or MMA * Past history of hypersensitivity or drug allergy to Carbaglu® * Patient with PA or MMA and…

What they're measuring

Number of emergency visits due to hyperammonemia within 24 months period

Timeframe: 24 months

Trial details

NCT IDNCT02426775

SponsorKing Abdullah International Medical Research Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-03-19