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Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease

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ClinicalTrials.gov Identifier: NCT01147042
Recruitment Status : Terminated (Early termination due to only 2 subjects completing trial.)
First Posted : June 22, 2010
Results First Posted : September 20, 2017
Last Update Posted : September 20, 2017
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions IFN-Gamma Therapy
CGD Gene Mutation
CGD Response to IFNg
CGD - Chronic Granulomatous Disease
Immunodeficiency Disease
Intervention Drug: IFN-gamma
Enrollment 2
Recruitment Details  
Pre-assignment Details  
Arm/Group Title gp91 CGD With HIGH Baseline Superoxide Autosomal Recessive CGD With p47
Hide Arm/Group Description Patients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production Patients with Autosomal Recessive Chronic Granulomatous Disease (CGD) with p47 phox mutation
Period Title: Overall Study
Started 2 0
Completed 2 0
Not Completed 0 0
Arm/Group Title gp91 CGD With Relatively High Baseline Superoxide Autosomal Recessive CGD With p47 Total
Hide Arm/Group Description

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.

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 of all reporting groups
Overall Number of Baseline Participants 2 0 2
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 0 participants 2 participants
<=18 years 1 0 1
Between 18 and 65 years 1 0 1
>=65 years 0 0 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 0 participants 2 participants
Female
0
   0.0%
0
0
   0.0%
Male
2
 100.0%
0
2
 100.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 2 participants 0 participants 2 participants
2
 100.0%
0
2
 100.0%
1.Primary Outcome
Title Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils
Hide Description [Not Specified]
Time Frame 21 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected.
Arm/Group Title gp91 CGD With HIGH Baseline Superoxide Autosomal Recessive CGD With p47
Hide Arm/Group Description:
Patients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production
Patients with Autosomal Recessive Chronic Granulomatous Disease (CGD) with p47 phox mutation
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title gp91 CGD With Relatively High Baseline Superoxide
Hide Arm/Group Description Patients with X-linked Chronic Granulomatous Disease (CGD) with a missense gp91phox mutation and relatively high baseline superoxide production
All-Cause Mortality
gp91 CGD With Relatively High Baseline Superoxide
Affected / at Risk (%)
Total   0/2 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
gp91 CGD With Relatively High Baseline Superoxide
Affected / at Risk (%)
Total   0/2 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
gp91 CGD With Relatively High Baseline Superoxide
Affected / at Risk (%)
Total   0/2 (0.00%) 
Early termination due to only 2 subjects completing trial.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. John Gallin
Organization: NIH CC
Phone: 301-827-5428
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) )
ClinicalTrials.gov Identifier: NCT01147042     History of Changes
Other Study ID Numbers: 100123
10-I-0123 ( Other Identifier: National Institutes of Health )
First Submitted: June 17, 2010
First Posted: June 22, 2010
Results First Submitted: July 20, 2017
Results First Posted: September 20, 2017
Last Update Posted: September 20, 2017