Ignite Creation Date:
2024-05-06 @ 4:50 PM
Last Modification Date:
2024-10-26 @ 2:17 PM
Study NCT ID:
NCT05104723
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-06-26
First Post:
2021-11-02
Brief Title:
Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase 12 Open-label Study to Evaluate the Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-10-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
Chronic granulomatous disease CGD is a disease of the immune system which is how the body fights germs People with CGD get infections easily and have other health problems Some medicines to treat CGD have a lot of side effects and do not always work Researchers want to see if a new drug can help
Objective
To see if tofacitinib is safe to use for treating chronic CGD
Eligibility
Adults aged 18 and older with CGD who have not had success with other treatments and who are enrolled on NIH study 93-I-0119
Design
Participants will be screened with the following
Physical exam
Medical history
Blood urine and stool tests
Pregnancy test if needed
An upper gastrointestinal endoscopy andor colonoscopy if needed for their symptoms Tissue samples will be collected
Skin assessment if needed
Participants will repeat some screening tests at visits
Participants will complete questionnaires about their general health and how CGD affects their daily life Photographs will be taken of their skin if needed They will have lung function tests if needed They will have a computed tomography CT scan of the chest abdomen and pelvis if needed A CT scan uses X-rays to create pictures of the inside of the body
Participants will gradually reduce the amount of some CGD medicines they take Then they will take tofacitinib as a pill twice a day or once a day for 3 months They will keep a drug diary They will have monthly study visits They will have a follow-up visit about 1 month after their last study drug visit
Participation will last for about 6 months
Chronic granulomatous disease CGD is a disease of the immune system which is how the body fights germs People with CGD get infections easily and have other health problems Some medicines to treat CGD have a lot of side effects and do not always work Researchers want to see if a new drug can help
Objective
To see if tofacitinib is safe to use for treating chronic CGD
Eligibility
Adults aged 18 and older with CGD who have not had success with other treatments and who are enrolled on NIH study 93-I-0119
Design
Participants will be screened with the following
Physical exam
Medical history
Blood urine and stool tests
Pregnancy test if needed
An upper gastrointestinal endoscopy andor colonoscopy if needed for their symptoms Tissue samples will be collected
Skin assessment if needed
Participants will repeat some screening tests at visits
Participants will complete questionnaires about their general health and how CGD affects their daily life Photographs will be taken of their skin if needed They will have lung function tests if needed They will have a computed tomography CT scan of the chest abdomen and pelvis if needed A CT scan uses X-rays to create pictures of the inside of the body
Participants will gradually reduce the amount of some CGD medicines they take Then they will take tofacitinib as a pill twice a day or once a day for 3 months They will keep a drug diary They will have monthly study visits They will have a follow-up visit about 1 month after their last study drug visit
Participation will last for about 6 months
Detailed Description:
Study Description
This is a phase 12 open-label trial to study the safety and to explore the biological efficacy of tofacitinib in patients with confirmed and symptomatic inflammatory complications gastrointestinal GI skin lung related to chronic granulomatous disease CGD After a 3-month regimen participants inflammatory complications will be objectively assessed
Primary Objective
To assess the safety of tofacitinib during the study period in patients with CGD
Secondary Objectives
1 To assess the overall clinical response for the specific inflammatory manifestations
2 To assess the biological effect of tofacitinib on interferon IFN-induced gene expression in CGD
Primary Endpoints
1 Rate of infection
2 Rate of treatment-related toxicities
3 Rate of adverse events AEs
4 Incidence of serious bacterial mycobacterial fungal or viral infections defined as infections that require medical assessment or hospitalization
Secondary Endpoints
CGD-related inflammatory bowel disease IBD
1 Change in modified Harvey-Bradshaw Index HBI
2 Change in histopathological endoscopy
Inflammatory lung disease
1 Change in forced expiratory volume FEV1
2 Change in diffusing capacity for carbon monoxide DLCO
3 Change in computed tomography CT radiography
4 Change in 6-minute walk
Skin disease
1 Change in presence of skin flares or ulcerations by objective photography evaluation
Gene expression
1Change in IFN gene module enrichment score derived from whole blood RNA expression data
This is a phase 12 open-label trial to study the safety and to explore the biological efficacy of tofacitinib in patients with confirmed and symptomatic inflammatory complications gastrointestinal GI skin lung related to chronic granulomatous disease CGD After a 3-month regimen participants inflammatory complications will be objectively assessed
Primary Objective
To assess the safety of tofacitinib during the study period in patients with CGD
Secondary Objectives
1 To assess the overall clinical response for the specific inflammatory manifestations
2 To assess the biological effect of tofacitinib on interferon IFN-induced gene expression in CGD
Primary Endpoints
1 Rate of infection
2 Rate of treatment-related toxicities
3 Rate of adverse events AEs
4 Incidence of serious bacterial mycobacterial fungal or viral infections defined as infections that require medical assessment or hospitalization
Secondary Endpoints
CGD-related inflammatory bowel disease IBD
1 Change in modified Harvey-Bradshaw Index HBI
2 Change in histopathological endoscopy
Inflammatory lung disease
1 Change in forced expiratory volume FEV1
2 Change in diffusing capacity for carbon monoxide DLCO
3 Change in computed tomography CT radiography
4 Change in 6-minute walk
Skin disease
1 Change in presence of skin flares or ulcerations by objective photography evaluation
Gene expression
1Change in IFN gene module enrichment score derived from whole blood RNA expression data
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 000064-I | None | None | None |