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

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
RECORDATI GROUP
Study ID
NCT05040178
Status
Recruiting
Apply to this trial

Conditions

  • Hyperammonemia
  • Methylmalonic Acidemia
  • Propionic Acidemia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Carglumic Acid — DRUG
    Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Study Details

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

Key Dates

Start date
Jun 30, 2022
Status verified
Apr 2025
Primary completion
Jun 30, 2032
Completion
Jun 30, 2032

Study Design

Enrollment
20 participants (estimated)

Arms

  • Arm: 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)

Primary Outcome Measure

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. ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Children's National HospitalWashington D.C.District of Columbia20010
Kara Simpson
[email protected]
(202) 545-2503
Nicholas Ah Mew, MD (PRINCIPAL_INVESTIGATOR)
University of South FloridaTampaFlorida33606
Farideh Oberheu
[email protected]
(813) 250-2292
Amarilis Sanchez-Valle, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Michael Sawin
[email protected]
(312) 227-2816
Joshua Baker, MD (PRINCIPAL_INVESTIGATOR)
Riley Children's HospitalIndianapolisIndiana46202
Susan Romie
[email protected]
(317) 278-6650
Melissa Lah, MD (PRINCIPAL_INVESTIGATOR)
Icahn School of Medicine at Mt. SinaiNew YorkNew York10029
Colleen Donnelly
[email protected]
(212) 241-5983
Margo Breilyn, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Washington D.C., DC

By research site
Children's National Hospital· Washington D.C., DCUniversity of South Florida· Tampa, FLAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILRiley Children's Hospital· Indianapolis, INIcahn School of Medicine at Mt. Sinai· New York, NY

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