Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Methylmalonic Acidemia Control Arm | No Intervention | patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12) | |
| Methylmalonic Acidemia Active arm | Experimental | patients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12) |
|
| Propionic Acidemia Control Arm | No Intervention | patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin) | |
| Propionic Acidemia Active arm | Experimental | patients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Carglumic Acid | Drug | Carglumic Acid 50 mg/kg/day (twice daily) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of emergency visits due to hyperammonemia within 24 months period | 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Time to first visit to the ER due to hyperammonemia from starting the treatment. | 24 months | |
| Plasma ammonia levels over the study treatment period. | 24 months | |
Not provided
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:
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:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Majid A. Alfadhel, MD | King Abdulaziz Medical City, National Guard / Riyadh-Saudi Arabia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| King Abdullah Specialist Children Hospital, King Abdulaziz Medical City | Riyadh | 11426 | Saudi Arabia | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31196016 | Derived | 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. |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D056693 | Propionic Acidemia |
| C537358 | Methylmalonic acidemia |
| ID | Term |
|---|---|
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C528449 | carglumic acid |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Number of days of hospitalization |
| 24 months |
| Acylcarnitine level for all patients | once on screening visit |
| Measuring urine organic acid levels for both diseases. | 24 months |
| Measuring Plasma aminoacids' levels for both diseases | 24 months |
| King Fahad Medical City |
| Riyadh |
| Saudi Arabia |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |