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Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia (CAMP)

Primary Purpose

Propionic Acidemia, Methylmalonic Acidemia

Status
Completed
Phase
Phase 3
Locations
Saudi Arabia
Study Type
Interventional
Intervention
Carglumic Acid
Sponsored by
King Abdullah International Medical Research Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Propionic Acidemia

Eligibility Criteria

undefined - 15 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • King Abdullah Specialist Children Hospital, King Abdulaziz Medical City
  • King Fahad Medical City

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

No Intervention

Experimental

No Intervention

Experimental

Arm Label

Methylmalonic Acidemia Control Arm

Methylmalonic Acidemia Active arm

Propionic Acidemia Control Arm

Propionic Acidemia Active arm

Arm Description

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

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

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

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

Outcomes

Primary Outcome Measures

Number of emergency visits due to hyperammonemia within 24 months period

Secondary Outcome Measures

Time to first visit to the ER due to hyperammonemia from starting the treatment.
Plasma ammonia levels over the study treatment period.
Number of days of hospitalization
Acylcarnitine level for all patients
Measuring urine organic acid levels for both diseases.
Measuring Plasma aminoacids' levels for both diseases

Full Information

First Posted
April 22, 2015
Last Updated
April 24, 2019
Sponsor
King Abdullah International Medical Research Center
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1. Study Identification

Unique Protocol Identification Number
NCT02426775
Brief Title
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Acronym
CAMP
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 Type
Interventional

2. Study Status

Record Verification Date
April 2019
Overall Recruitment Status
Completed
Study Start Date
November 2015 (undefined)
Primary Completion Date
March 19, 2019 (Actual)
Study Completion Date
March 19, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
King Abdullah International Medical Research Center

4. Oversight

Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Propionic Acidemia, Methylmalonic Acidemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
33 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Methylmalonic Acidemia Control Arm
Arm Type
No Intervention
Arm Description
patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12)
Arm Title
Methylmalonic Acidemia Active arm
Arm Type
Experimental
Arm Description
patients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12)
Arm Title
Propionic Acidemia Control Arm
Arm Type
No Intervention
Arm Description
patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin)
Arm Title
Propionic Acidemia Active arm
Arm Type
Experimental
Arm Description
patients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin)
Intervention Type
Drug
Intervention Name(s)
Carglumic Acid
Other Intervention Name(s)
Carbaglu
Intervention Description
Carglumic Acid 50 mg/kg/day (twice daily)
Primary Outcome Measure Information:
Title
Number of emergency visits due to hyperammonemia within 24 months period
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Time to first visit to the ER due to hyperammonemia from starting the treatment.
Time Frame
24 months
Title
Plasma ammonia levels over the study treatment period.
Time Frame
24 months
Title
Number of days of hospitalization
Time Frame
24 months
Title
Acylcarnitine level for all patients
Time Frame
once on screening visit
Title
Measuring urine organic acid levels for both diseases.
Time Frame
24 months
Title
Measuring Plasma aminoacids' levels for both diseases
Time Frame
24 months

10. Eligibility

Sex
All
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Majid A. Alfadhel, MD
Organizational Affiliation
King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia
Official's Role
Principal Investigator
Facility Information:
Facility Name
King Abdullah Specialist Children Hospital, King Abdulaziz Medical City
City
Riyadh
ZIP/Postal Code
11426
Country
Saudi Arabia
Facility Name
King Fahad Medical City
City
Riyadh
Country
Saudi Arabia

12. IPD Sharing Statement

Citations:
PubMed Identifier
31196016
Citation
Nashabat M, Obaid A, Al Mutairi F, Saleh M, Elamin M, Ahmed H, Ababneh F, Eyaid W, Alswaid A, Alohali L, Faqeih E, Aljeraisy M, Hussein MA, Alasmari A, Alfadhel M. Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial. BMC Pediatr. 2019 Jun 13;19(1):195. doi: 10.1186/s12887-019-1571-y.
Results Reference
derived

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Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

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