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NCT Number: NCT02426775

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

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.

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Key information

Conditions

Propionic Acidemia Amino Acid Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Methylmalonic Acidemia Nutritional and Metabolic Diseases

Age range

Up to 15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

King Abdullah Specialist Children Hospital, King Abdulaziz Medical City, Riyadh, Saudi Arabia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 other inherited genetic conditions or congenital anomalies

Treatment and study plan

Carglumic Acid

Drug

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

Other names: Carbaglu

Primary outcomes

  1. Number of emergency visits due to hyperammonemia within 24 months period

    Time frame: 24 months

Secondary outcomes

  1. Time to first visit to the ER due to hyperammonemia from starting the treatment.

    Time frame: 24 months

  2. Plasma ammonia levels over the study treatment period.

    Time frame: 24 months

  3. Number of days of hospitalization

    Time frame: 24 months

  4. Acylcarnitine level for all patients

    Time frame: once on screening visit

  5. Measuring urine organic acid levels for both diseases.

    Time frame: 24 months

  6. Measuring Plasma aminoacids' levels for both diseases

    Time frame: 24 months

Sponsors and collaborators

Lead sponsor

King Abdullah International Medical Research Center

Other

Registry information

Official study title

Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)

Acronym: CAMP

Important dates

Study start
2015
Primary completion
2019
Study completion
2019
First posted
Apr 27, 2015
Registry last updated
Apr 25, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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