Chronic Granulomatous Disease Clinical Trial
Official title:
Efficacy and Safety of Pioglitazone Therapy for Chronic Granulomatous Disease Patients With Severe Infection.
Verified date | October 2019 |
Source | Children's Hospital of Fudan University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 3 |
Est. completion date | October 20, 2019 |
Est. primary completion date | October 20, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 18 Years |
Eligibility |
Inclusion Criteria: 1. Age:1 months to 18 years 2. Chronic Granulomatous Disease 3. with severe infections Exclusion Criteria: 1. > 18 years of age 2. infections are treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes) |
Country | Name | City | State |
---|---|---|---|
China | Children's Hospital of Fudan University | Shanghai | Shanghai |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital of Fudan University |
China,
Fernandez-Boyanapalli RF, Falcone EL, Zerbe CS, Marciano BE, Frasch SC, Henson PM, Holland SM, Bratton DL. Impaired efferocytosis in human chronic granulomatous disease is reversed by pioglitazone treatment. J Allergy Clin Immunol. 2015 Nov;136(5):1399-1401.e3. doi: 10.1016/j.jaci.2015.07.034. Epub 2015 Sep 18. — View Citation
Fernandez-Boyanapalli RF, Frasch SC, Thomas SM, Malcolm KC, Nicks M, Harbeck RJ, Jakubzick CV, Nemenoff R, Henson PM, Holland SM, Bratton DL. Pioglitazone restores phagocyte mitochondrial oxidants and bactericidal capacity in chronic granulomatous disease. J Allergy Clin Immunol. 2015 Feb;135(2):517-527.e12. doi: 10.1016/j.jaci.2014.10.034. Epub 2014 Dec 10. — View Citation
Migliavacca M, Assanelli A, Ferrua F, Cicalese MP, Biffi A, Frittoli M, Silvani P, Chidini G, Calderini E, Mandelli A, Camporesi A, Milani R, Farinelli G, Nicoletti R, Ciceri F, Aiuti A, Bernardo ME. Pioglitazone as a novel therapeutic approach in chronic granulomatous disease. J Allergy Clin Immunol. 2016 Jun;137(6):1913-1915.e2. doi: 10.1016/j.jaci.2016.01.033. Epub 2016 Apr 4. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | efficiency of Pioglitazone | Frequency of infections as indicator for the drug's benefit for the patients; Functional reconstitution of the NADPH oxidase in circulating cells of the peripheral blood (Stimulation Index by DHR analysis). | 3 years | |
Secondary | Incidence of Treatment-Emergent Adverse Events | Frequency and severity of metabolic disorders and unexpected toxic adverse events during and after using pioglitazone | 3 years |
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