Trial Outcomes & Findings for Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease (NCT NCT01147042)
NCT ID: NCT01147042
Last Updated: 2017-09-20
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
2 participants
Primary outcome timeframe
21 weeks
Results posted on
2017-09-20
Participant Flow
Participant milestones
| Measure |
gp91 CGD With HIGH Baseline Superoxide
Patients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production
|
Autosomal Recessive CGD With p47
Patients with Autosomal Recessive Chronic Granulomatous Disease (CGD) with p47 phox mutation
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
0
|
|
Overall Study
COMPLETED
|
2
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
Baseline characteristics by cohort
| Measure |
gp91 CGD With Relatively High Baseline Superoxide
n=2 Participants
Subjects in this cohort have X-linked CGD resulting from a documented missense gene and superoxide production by cytochrome c reduction assay at baseline of greater than 2.5 nmol/106 cells per hour.
Following a washout period subjects have a subcutaneous injection of 50 mcg per meter squared administered once per week, Monday, for 4 weeks, twice per week, Monday and Thursday, for 4 weeks, then thrice per week, Monday, Wednesday, Friday, for 4 weeks for a total of 12 weeks of Interferon-gamma treatment.
|
Autosomal Recessive CGD With p47
Subjects in this cohort have autosomal recessive CGD resulting from a documented p47phox gene mutation.
Subjects will then be started on a 12 week course of IFN treatment. Subjects will have a subcutaneous injection of 50 mcg/m2 once per week (Monday) for 4 weeks, twice per week (Monday and Thursday) for 4 weeks, then thrice per week (Monday, Wednesday, Friday) for 4 weeks.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 21 weeksPopulation: Data were not collected.
Outcome measures
Outcome data not reported
Adverse Events
gp91 CGD With Relatively High Baseline Superoxide
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
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place