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| Name | Class |
|---|---|
| Target PharmaSolutions, Inc. | INDUSTRY |
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To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice.
Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Male and Female Adult and Pediatric Participants | Patients treated with Carbaglu for the treatment for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Carglumic Acid | Drug | Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Effects of Carbaglu® on plasma ammonia levels | Multiple plasma ammonia levels will be collected only during treatment with Carbaglu® according to prescribing information and routine medical practice in terms of visit frequency. | Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment. |
| Adverse Event frequency and severity | Any Carbaglu® related adverse events will be be collected and reported | Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment. |
| Measure | Description | Time Frame |
|---|---|---|
| Fetal Outcomes and Pregnancy Outcomes | Pregnancy risks including maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant (through the first year of life). | Collection of pregnancy information for patients who becomes pregnant while participating in the trial or at time of enrollment. Pregnancy reports and reports involving neonates and infants up to 1 year of age must be reported to RRD Pharmacovigilance. |
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Inclusion Criteria:
Provision of signed and dated informed consent/assent form
Prescribed and treated with Carbaglu®
Have an established diagnosis of PA or MMA defined as follows:
AND/OR
Exclusion Criteria:
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Patients with PA or MMA of any age and gender, including pregnant women, are eligible for enrollment in the study as long as they meet the eligibility criteria.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Anne Marie Cesario | Contact | 908-849-4907 | cesario.a@recordati.com |
| Name | Affiliation | Role |
|---|---|---|
| William Ludlum, MD | Recordati Rare Diseases Inc | Study Director |
| Nicholas Ah Mew, MD | Children's National Research Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Hospital | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
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| ID | Term |
|---|---|
| D022124 | Hyperammonemia |
| C537358 | Methylmalonic acidemia |
| D056693 | Propionic Acidemia |
| ID | Term |
|---|---|
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
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| ID | Term |
|---|---|
| C528449 | carglumic acid |
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|
| University of South Florida | Recruiting | Tampa | Florida | 33606 | United States |
|
| Ann & Robert H. Lurie Children's Hospital of Chicago | Recruiting | Chicago | Illinois | 60611 | United States |
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| Riley Children's Hospital | Recruiting | Indianapolis | Indiana | 46202 | United States |
|
| Icahn School of Medicine at Mt. Sinai | Recruiting | New York | New York | 10029 | United States |
|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |