Trial Outcomes & Findings for Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia (NCT NCT01597440)

Last Updated: 2017-04-26

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

30 months

Results posted on

2017-04-26

Participant Flow

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	1	0
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Carbaglu n=1 Participants 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 n=1 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Age, Continuous	4 days STANDARD_DEVIATION 0 • n=5 Participants	—	4 days STANDARD_DEVIATION 0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	—	1 participants n=5 Participants
NH3	223 mcmol/L STANDARD_DEVIATION 0 • n=5 Participants	—	223 mcmol/L STANDARD_DEVIATION 0 • n=5 Participants

PRIMARY outcome

Timeframe: 30 months

Population: The study was closed prematurely. There is no analysis population.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Start of episode through 7 days or discharge (if earlier)

Safety is measured by tracking and detailing the number and type of adverse events and their severity based on the CTCAE.

Outcome measures

Outcome measures
Measure	Carbaglu n=1 Participants 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
Number of Participants With Adverse Events	1 Participants	—

Adverse Events

Carbaglu

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Carbaglu n=1 participants at risk 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
Nervous system disorders Lethargy	100.0% 1/1 • Number of events 1	— 0/0
Respiratory, thoracic and mediastinal disorders Hyperventilation	100.0% 1/1 • Number of events 1	— 0/0
Gastrointestinal disorders Vomiting	100.0% 1/1 • Number of events 1	— 0/0
Metabolism and nutrition disorders Ketosis	100.0% 1/1 • Number of events 1	— 0/0
Blood and lymphatic system disorders Acidosis	100.0% 1/1 • Number of events 1	— 0/0
Blood and lymphatic system disorders low white blood counts	100.0% 1/1 • Number of events 1	— 0/0
Blood and lymphatic system disorders anemia	100.0% 1/1 • Number of events 1	— 0/0

Additional Information

Dr. Mendel Tuchman

Children's National Health System

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place