Pioglitazone Therapy for Chronic Granulomatous Disease

Status Terminated

Clinical Trial Summary

The purpose of this proposed research is to investigate the efficacy and safety of the therapy with pioglitazone for chronic granulomatous disease (CGD) patients severe infection.

Clinical Trial Description

Chronic granulomatous disease (CGD) is a rare genetic disease caused by defects in genes encoding the subunits of the nicotinamide adenine dinucleotide（NADPH）phosphate oxidase complex. In normal phagocytes peroxisome proliferator-activated receptor gamma (PPARγ) activation links NADPH oxidase activity with enhanced mitochondrial reactive oxygen species (ROS) production. There is deficient mitochondrial ROS production in CGD，due to the lack of this upstream signaling by the NADPH oxidase and PPARγ. These patients are susceptible to bacterial and fungal infections, as well as extensive tissue granuloma formation. X-chromosome-linked CGD (X-CGD) is most frequently. And it generally produces a severe phenotype, with a mortality rate of 3% to 5% per year despite state-of-the-art prophylaxis and intensive multimodal treatment.

At present the most curative treatment for patients with X-CGD is hematopoietic stem cell transplantion (HSCT). But for many patients without an HLA-matched donor and active infections/inflammatory complications still require novel approches.

PPARγ agonist such as pioglitazone, approved for type 2 diabetes, was reported to bypass the need for the NADPH oxidase for enhanced mtROS production and partially restored host defense in CGD. What's more, some animal models and several clinical cases have proved its efficacy. The investigators propose to study the efficacy and safety of the therapy with pioglitazone for children with severe infection of CGD, and its long-term effects.

Through this study the investigators hope to confirm the benefits of pioglitazone in the treatment of this rare disease especially for those patients without a prompt suitable matched donor or for whom the critical disease conditions force to postpone HSCT. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03080480
Study type	Interventional
Source	Children's Hospital of Fudan University
Contact
Status	Terminated
Phase	Phase 1/Phase 2
Start date	September 1, 2017
Completion date	October 20, 2019

View Details

See also
Status	Clinical Trial	Phase
Withdrawn	`NCT03278912` - Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations
Recruiting	`NCT01652092` - Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies	N/A
Completed	`NCT04136028` - IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease	Early Phase 1
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Terminated	`NCT00394316` - Gene Therapy for Chronic Granulomatous Disease	Early Phase 1
Recruiting	`NCT03910452` - Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide	Early Phase 1
Terminated	`NCT02282904` - Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide	Phase 1/Phase 2
Terminated	`NCT02926963` - Generation of Powerful Biological Tools for Understanding the Pathophysiology of Chronic Granulomatous Disease.	N/A
Completed	`NCT00006417` - Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease	Phase 2
Completed	`NCT00368446` - Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT06162936` - Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease
Enrolling by invitation	`NCT06253507` - pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)	Phase 1/Phase 2
Terminated	`NCT05915897` - Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)
Terminated	`NCT00325078` - Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease	Phase 1/Phase 2
Completed	`NCT00001317` - A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood	Phase 4
Completed	`NCT03548818` - Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease
Recruiting	`NCT01821781` - Immune Disorder HSCT Protocol	Phase 2
Completed	`NCT00578643` - Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease	Phase 2
Terminated	`NCT00006054` - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies	N/A