The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05040178
Recruitment Status : Recruiting
First Posted : September 10, 2021
Last Update Posted : January 30, 2024
Sponsor:
Collaborator:
Target PharmaSolutions, Inc.
Information provided by (Responsible Party):
Recordati Rare Diseases

Tracking Information
First Submitted Date June 16, 2021
First Posted Date September 10, 2021
Last Update Posted Date January 30, 2024
Actual Study Start Date June 30, 2022
Estimated Primary Completion Date June 30, 2032   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 13, 2022)
  • Effects of Carbaglu® on plasma ammonia levels [ Time Frame: 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. ]
    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.
  • Adverse Event frequency and severity [ Time Frame: 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. ]
    Any Carbaglu® related adverse events will be be collected and reported
Original Primary Outcome Measures
 (submitted: September 9, 2021)		 Effects of Carbaglu® on plasma ammonia levels [ Time Frame: Plasma levels will be collected during treatment with Carbaglu® ]
Multiple plasma ammonia levels will be collected during treatment with Carbaglu®
Change History
Current Secondary Outcome Measures
 (submitted: June 13, 2022)		 Fetal Outcomes and Pregnancy Outcomes [ Time Frame: 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. ]
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).
Original Secondary Outcome Measures
 (submitted: September 9, 2021)		 Incidence of Carbaglu® Related Adverse Events [ Time Frame: Duration of treatment with Carbaglu® ]
Any Carbaglu® related adverse events will be be collected and reported
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Official Title A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
Brief Summary To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Detailed Description

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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population 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.
Condition
  • Hyperammonemia
  • Methylmalonic Acidemia
  • Propionic Acidemia
Intervention Drug: Carglumic Acid
Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Other Name: Carbaglu®
Study Groups/Cohorts 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)
Intervention: Drug: Carglumic Acid
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: June 13, 2022)		 20
Original Estimated Enrollment
 (submitted: September 9, 2021)		 10
Estimated Study Completion Date June 30, 2032
Estimated Primary Completion Date June 30, 2032   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  1. Provision of signed and dated informed consent/assent form
  2. Prescribed and treated with Carbaglu®

  3. Have an established diagnosis of PA or MMA defined as follows:

    • Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR
    • Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).

AND/OR

  • Confirmation by molecular genetic testing

Exclusion Criteria:

  • None
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Anne Marie Cesario 908-849-4907 cesario.a@recordati.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT05040178
Other Study ID Numbers CARBAGLU-RRDUS-PASS-0573
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Undecided
Current Responsible Party Recordati Rare Diseases
Original Responsible Party Same as current
Current Study Sponsor Recordati Rare Diseases
Original Study Sponsor Same as current
Collaborators Target PharmaSolutions, Inc.
Investigators
Study Director: William Ludlum, MD Recordati Rare Diseases Inc.
Principal Investigator: Nicholas Ah Mew, MD Children's National Research Institute
PRS Account Recordati Rare Diseases
Verification Date January 2024