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CAMP: Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Sponsor
King Abdullah International Medical Research Center (Other)
Overall Status
Completed
CT.gov ID
NCT02426775
Collaborator
(none)
33
Enrollment
2
Locations
4
Arms
40.5
Duration (Months)
16.5
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Carglumic Acid
 Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
33 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official 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)
Study Start Date :
Nov 1, 2015
Actual Primary Completion Date :
Mar 19, 2019
Actual Study Completion Date :
Mar 19, 2019

Arms and Interventions

Arm Intervention/Treatment
No Intervention: Methylmalonic Acidemia Control Arm

patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12)
Experimental: Methylmalonic Acidemia Active arm

patients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12)

Drug: Carglumic Acid
Carglumic Acid 50 mg/kg/day (twice daily)
Other Names:
  • Carbaglu

    		•
    No Intervention: Propionic Acidemia Control Arm

    patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin)
    Experimental: Propionic Acidemia Active arm

    patients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin)

    Drug: Carglumic Acid
    Carglumic Acid 50 mg/kg/day (twice daily)
    Other Names:
  • Carbaglu

    		•

    Outcome Measures

    Primary Outcome Measures

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

    Secondary Outcome Measures

    1. Time to first visit to the ER due to hyperammonemia from starting the treatment. [24 months]

    2. Plasma ammonia levels over the study treatment period. [24 months]

    3. Number of days of hospitalization [24 months]

    4. Acylcarnitine level for all patients [once on screening visit]

    5. Measuring urine organic acid levels for both diseases. [24 months]

    6. Measuring Plasma aminoacids' levels for both diseases [24 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 15 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 King Abdullah Specialist Children Hospital, King Abdulaziz Medical City Riyadh Saudi Arabia 11426
    2 King Fahad Medical City Riyadh Saudi Arabia

    Sponsors and Collaborators

    • King Abdullah International Medical Research Center

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Dr. Majid Alfadhel, Head Genetics Division, Department of Pediatrics. Assistant Professor, King Saud bin Abdulaziz University for Health Sciences (KSAU-HS), King Abdullah International Medical Research Center
    ClinicalTrials.gov Identifier:
    NCT02426775
    Other Study ID Numbers:
    • RC 13/116
    First Posted:
    Apr 27, 2015
    Last Update Posted:
    Apr 25, 2019
    Last Verified:
    Apr 1, 2019
    Additional relevant MeSH terms:
    Amino Acid Metabolism, Inborn Errors
    Propionic Acidemia
    Acidosis

    Study Results

    No Results Posted as of Apr 25, 2019