Trial Outcomes & Findings for Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis (NCT NCT00028262)

Last Updated: 2016-10-27

Results Overview

The GRODs in peripheral white blood cells from all patients before and during treatment were analyzed by transmission electron microscopy (TEM) at 30000xmagnification. Two investigators working independently of each other identified and counted the GRODs and the results were averaged.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

10 participants

Primary outcome timeframe

10 years

Results posted on

2016-10-27

Participant Flow

Participant milestones

Participant milestones
Measure	Drug Cystagon and N-acetylcysteine
Overall Study STARTED	10
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Drug Cystagon and N-acetylcysteine n=10 Participants
Age, Categorical <=18 years	10 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 10 years

Population: Out of 10 enrolled subjects, one subject did not complete study and was lost to follow up.

Outcome measures

Outcome measures
Measure	Drug Cystagon and N-acetylcysteine n=9 Participants
Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells.	-0.3317 Average number of GRODs Interval -0.3943 to -0.2691

Adverse Events

Drug Cystagon and N-acetylcysteine

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Anil Mukherjee

National Institute of Child Health and Human Development

Phone: +1 301 496 7213

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place