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

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ClinicalTrials.gov Identifier: NCT01597440
Recruitment Status : Terminated (insufficient enrollment)
First Posted : May 14, 2012
Results First Posted : April 26, 2017
Last Update Posted : April 26, 2017
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

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
Enrollment 1
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Carbaglu Placebo
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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

Standard of Care therapy

Standard of Care: Standard of Care

Period Title: Overall Study
Started 1 0
Completed 1 0
Not Completed 0 0
Arm/Group Title Carbaglu Placebo Total
Hide Arm/Group Description

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

Standard of Care therapy

Standard of Care: Standard of Care

Total of all reporting groups
Overall Number of Baseline Participants 1 0 1
Hide Baseline Analysis Population Description
One participant, infant under 1 month old, with organic acidemia.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 0 participants 1 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, Continuous  
Mean (Standard Deviation)
Unit of measure:  Days
Number Analyzed 1 participants 0 participants 1 participants
4  (0) 4  (0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 0 participants 1 participants
Female
0
   0.0%
0
   0.0%
Male
1
 100.0%
1
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants 0 participants 1 participants
1 1
NH3  
Mean (Standard Deviation)
Unit of measure:  mcmol/L
Number Analyzed 1 participants 0 participants 1 participants
223  (0) 223  (0)
1.Primary Outcome
Title Neurodevelopment
Hide Description Neurodevelopmental outcome as measured by Cognitive Composite (Bayley III), Motor Composite (Bayley III) and Functional Status Scale and safety of NCG treatment as measured by adverse events and laboratory blood tests
Time Frame 30 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The study was closed prematurely. There is no analysis population.
Arm/Group Title Carbaglu Placebo
Hide Arm/Group Description:

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

Standard of Care therapy

Standard of Care: Standard of Care

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Number of Participants With Adverse Events
Hide Description Safety is measured by tracking and detailing the number and type of adverse events and their severity based on the CTCAE.
Time Frame Start of episode through 7 days or discharge (if earlier)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Carbaglu Placebo
Hide Arm/Group Description:

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

Standard of Care Therapy

Standard of Care: Standard of Care

Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: Participants
1
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Carbaglu Placebo
Hide Arm/Group Description

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

Standard of Care therapy

Standard of Care: Standard of Care

All-Cause Mortality
Carbaglu Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Carbaglu Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/1 (0.00%)      0/0    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Carbaglu Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/1 (100.00%)      0/0    
Blood and lymphatic system disorders     
Acidosis  [1]  1/1 (100.00%)  1 0/0  0
low white blood counts  [2]  1/1 (100.00%)  1 0/0  0
anemia  [3]  1/1 (100.00%)  1 0/0  0
Gastrointestinal disorders     
Vomiting  [4]  1/1 (100.00%)  1 0/0  0
Metabolism and nutrition disorders     
Ketosis  [5]  1/1 (100.00%)  1 0/0  0
Nervous system disorders     
Lethargy  [6]  1/1 (100.00%)  1 0/0  0
Respiratory, thoracic and mediastinal disorders     
Hyperventilation  [7]  1/1 (100.00%)  1 0/0  0
Indicates events were collected by systematic assessment
[1]
serum bicarbonate abnormal
[2]
Low overall white blood counts and low absolute neutrophil count
[3]
low hemoglobin and hematocrit
[4]
vomited early in the AM
[5]
large ketones in urine
[6]
Mildly lethargic
[7]
tachypnea, hyperpnea
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
Responsible Party: Mendel Tuchman, Children's Research Institute
ClinicalTrials.gov Identifier: NCT01597440     History of Changes
Other Study ID Numbers: NCGC 0007
First Submitted: May 10, 2012
First Posted: May 14, 2012
Results First Submitted: June 20, 2016
Results First Posted: April 26, 2017
Last Update Posted: April 26, 2017