Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Propionic Acidemia Clinical Trial

— 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02426775
• Other study ID #: RC 13/116
• Status: Completed
• Phase: Phase 3
• Start date: November 2015
• Est. completion date: March 19, 2019

Study information

• Verified date: April 2019
• Source: King Abdullah International Medical Research Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 33
• Est. completion date: March 19, 2019
• Est. primary completion date: March 19, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Acidosis
• Amino Acid Metabolism, Inborn Errors
• Methylmalonic Acidemia
• Propionic Acidemia

Intervention

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

Locations

Country	Name	City	State
Saudi Arabia	King Abdullah Specialist Children Hospital, King Abdulaziz Medical City	Riyadh
Saudi Arabia	King Fahad Medical City	Riyadh

Sponsors (1)

Lead Sponsor	Collaborator
King Abdullah International Medical Research Center

Country where clinical trial is conducted

Saudi Arabia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of emergency visits due to hyperammonemia within 24 months period		24 months
Secondary	Time to first visit to the ER due to hyperammonemia from starting the treatment.		24 months
Secondary	Plasma ammonia levels over the study treatment period.		24 months
Secondary	Number of days of hospitalization		24 months
Secondary	Acylcarnitine level for all patients		once on screening visit
Secondary	Measuring urine organic acid levels for both diseases.		24 months
Secondary	Measuring Plasma aminoacids' levels for both diseases		24 months