Propionic Acidemia Clinical Trial
— CAMPOfficial 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)
Verified date | April 2019 |
Source | King Abdullah International Medical Research Center |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
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 |
Country | Name | City | State |
---|---|---|---|
Saudi Arabia | King Abdullah Specialist Children Hospital, King Abdulaziz Medical City | Riyadh | |
Saudi Arabia | King Fahad Medical City | Riyadh |
Lead Sponsor | Collaborator |
---|---|
King Abdullah International Medical Research Center |
Saudi Arabia,
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 |
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