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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

Study Description
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Brief Summary:
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Condition or disease Intervention/treatment
Hyperammonemia Methylmalonic Acidemia Propionic Acidemia Drug: Carglumic Acid

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 Design
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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Other
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
Actual Study Start Date : June 30, 2022
Estimated Primary Completion Date : June 30, 2032
Estimated Study Completion Date : June 30, 2032

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
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)
 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®


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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


Secondary Outcome Measures :
  1. 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).


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05040178


Contacts
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Contact: Anne Marie Cesario 908-849-4907 cesario.a@recordati.com

Locations
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United States, District of Columbia
Children's National Hospital Recruiting
Washington, District of Columbia, United States, 20010
Contact: Kara Simpson    202-545-2503    ksimpson@childrensnational.org   
Principal Investigator: Nicholas Ah Mew, MD         
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33606
Contact: Farideh Oberheu    813-250-2292    faljalla@usf.edu   
Principal Investigator: Amarilis Sanchez-Valle, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Michael Sawin    312-227-2816    Msawin@luriechildrens.org   
Principal Investigator: Joshua Baker, MD         
United States, Indiana
Riley Children's Hospital Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan Romie    317-278-6650    sromie@iu.edu   
Principal Investigator: Melissa Lah, MD         
United States, New York
Icahn School of Medicine at Mt. Sinai Recruiting
New York, New York, United States, 10029
Contact: Colleen Donnelly    212-241-5983    Colleen.donnelly@mssm.edu   
Principal Investigator: Margo Breilyn, MD         
Sponsors and Collaborators
Recordati Rare Diseases
Target PharmaSolutions, Inc.
Investigators
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Study Director: William Ludlum, MD Recordati Rare Diseases Inc.
Principal Investigator: Nicholas Ah Mew, MD Children's National Research Institute
More Information
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Responsible Party: Recordati Rare Diseases
ClinicalTrials.gov Identifier: NCT05040178    
Obsolete Identifiers: NCT05842837
Other Study ID Numbers: CARBAGLU-RRDUS-PASS-0573
First Posted: September 10, 2021    Key Record Dates
Last Update Posted: January 30, 2024
Last Verified: January 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Recordati Rare Diseases:
PA & MMA
Additional relevant MeSH terms:
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Amino Acid Metabolism, Inborn Errors
Propionic Acidemia
Acidosis
Hyperammonemia
Acid-Base Imbalance
 Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Pathologic Processes