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Trial record 2 of 11 for:    Methylmalonic Acidemia

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia (CAMP)

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ClinicalTrials.gov Identifier: NCT02426775
Recruitment Status : Completed
First Posted : April 27, 2015
Last Update Posted : April 25, 2019
Sponsor:
Information provided by (Responsible Party):
Dr. Majid Alfadhel, King Abdullah International Medical Research Center

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
Propionic Acidemia Methylmalonic Acidemia Drug: Carglumic Acid Phase 3

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Study Type : Interventional  (Clinical Trial)
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 : November 2015
Actual Primary Completion Date : March 19, 2019
Actual Study Completion Date : March 19, 2019


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 Name: 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 Name: Carbaglu




Primary Outcome Measures :
  1. Number of emergency visits due to hyperammonemia within 24 months period [ Time Frame: 24 months ]

Secondary Outcome Measures :
  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 ]


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Ages Eligible for Study:   up to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02426775


Locations
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Saudi Arabia
King Abdullah Specialist Children Hospital, King Abdulaziz Medical City
Riyadh, Saudi Arabia, 11426
King Fahad Medical City
Riyadh, Saudi Arabia
Sponsors and Collaborators
King Abdullah International Medical Research Center
Investigators
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Principal Investigator: Majid A. Alfadhel, MD King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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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     History of Changes
Other Study ID Numbers: RC 13/116
First Posted: April 27, 2015    Key Record Dates
Last Update Posted: April 25, 2019
Last Verified: April 2019
Additional relevant MeSH terms:
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Propionic Acidemia
Amino Acid Metabolism, Inborn Errors
Acidosis
Acid-Base Imbalance
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn