Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

PHASE2TerminatedINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

September 30, 2012

Primary Completion Date

February 28, 2015

Study Completion Date

February 28, 2015

Conditions
Propionic AcidemiaMethylmalonic Acidemia
Interventions
DRUG

N-carbamylglutamate

"Active NCG \& Standard of Care Chemical Composition: N-carbamyl-L-glutamic acid (NCG) The daily dose will be 100 mg/kg/ day. The doses are to be divided into 2 equal doses and administered orally or enterally by nasogastric or gastrostomy tube (standard of care will prevail when choosing the mode of drug administration).~The tablets must be dispersed in a minimum of 2.5-10 ml of water and ingested immediately or administered by fast push through a syringe via a nasogastric or gastrostomy tube. The suspension has a slightly acidic taste.~This drug will be administered for 7 days after admission or until discharge (whichever is sooner)."

OTHER

Standard of Care

Standard of Care

Trial Locations (7)

19104

The Children's Hospital of Philadelphia, Philadelphia

20010

Children's National Medical Center, Washington D.C.

44106

University Hospitals of Cleveland/Rainbow Babies and Children's Hospital, Cleveland

80045

The Children's Hospital of Colorado, Aurora

90095

University of California Los Angeles, Los Angeles

94304

Lucile Packard Children's Hospital at Stanford, Palo Alto

02115

Children's Hospital Boston, Boston

All Listed Sponsors
collaborator

Children's National Research Institute

OTHER

collaborator

Boston Children's Hospital

OTHER

collaborator

University Hospitals Cleveland Medical Center

OTHER

collaborator

University of California, Los Angeles

OTHER

collaborator

Children's Hospital of Philadelphia

OTHER

collaborator

Lucile Packard Children's Hospital

OTHER

collaborator

University of Colorado, Denver

OTHER

lead

Mendel Tuchman

OTHER