This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

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

This study has been terminated.
(insufficient enrollment)
Sponsor:
Collaborators:
Children's Research Institute
Boston Children’s Hospital
University Hospitals Cleveland Medical Center
University of California, Los Angeles
Children's Hospital of Philadelphia
Lucile Packard Children's Hospital
University of Colorado, Denver
Information provided by (Responsible Party):
Mendel Tuchman, Children's Research Institute
ClinicalTrials.gov Identifier:
NCT01597440
First received: May 10, 2012
Last updated: March 15, 2017
Last verified: March 2017
Results First Received: June 20, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Propionic Acidemia
Methylmalonic Acidemia
Interventions: Drug: N-carbamylglutamate
Other: Standard of Care

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Carbaglu

Active NCG & Standard of Care

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).

Standard of Care: Standard of Care

Placebo

Standard of Care therapy

Standard of Care: Standard of Care


Participant Flow:   Overall Study
    Carbaglu   Placebo
STARTED   1   0 
COMPLETED   1   0 
NOT COMPLETED   0   0 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
One participant, infant under 1 month old, with organic acidemia.

Reporting Groups
  Description
Carbaglu

Active NCG & Standard of Care

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).

Standard of Care: Standard of Care

Placebo

Standard of Care therapy

Standard of Care: Standard of Care

Total Total of all reporting groups

Baseline Measures
   Carbaglu   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 1   0   1 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      1 100.0%         1 100.0% 
Between 18 and 65 years      0   0.0%         0   0.0% 
>=65 years      0   0.0%         0   0.0% 
Age 
[Units: Days]
Mean (Standard Deviation)
 4  (0)      4  (0) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      0   0.0%         0   0.0% 
Male      1 100.0%         1 100.0% 
Region of Enrollment 
[Units: Participants]
     
United States   1      1 
NH3 
[Units: mcmol/L]
Mean (Standard Deviation)
 223  (0)      223  (0) 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Neurodevelopment   [ Time Frame: 30 months ]

2.  Secondary:   Number of Participants With Adverse Events   [ Time Frame: Start of episode through 7 days or discharge (if earlier) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Mendel Tuchman
Organization: Children's National Health System
e-mail: mtuchman@childrensnational.org



Responsible Party: Mendel Tuchman, Children's Research Institute
ClinicalTrials.gov Identifier: NCT01597440     History of Changes
Other Study ID Numbers: NCGC 0007
Study First Received: May 10, 2012
Results First Received: June 20, 2016
Last Updated: March 15, 2017