NCT03080480

Brief 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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
3

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Sep 2017

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 27, 2017

Completed
16 days until next milestone

First Posted

Study publicly available on registry

March 15, 2017

Completed
6 months until next milestone

Study Start

First participant enrolled

September 1, 2017

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 20, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 20, 2019

Completed
Last Updated

October 22, 2019

Status Verified

October 1, 2019

Enrollment Period

2.1 years

First QC Date

February 27, 2017

Last Update Submit

October 19, 2019

Conditions

Chronic Granulomatous Disease

Keywords

Chronic Granulomatous DiseaseSevere InfectionPPARγPioglitazonephagocytes

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • Incidence of Treatment-Emergent Adverse Events

    3 years

Study Arms (1)

Pioglitazone

EXPERIMENTAL

Treatment for chronic granulomatous disease patients with severe infection.

Drug: Pioglitazone

Interventions

PioglitazoneDRUG

Pioglitazone is PPARγ agonist that may be enhance ROS production and partially restore host phagocytes in CGD. pioglitazone is administered at a starting dose of 1 mg/kg and given the absence of adverse effects is progressively increased up to 3 mg/kg or 30 mg/daily.

Also known as: ACTOS
Pioglitazone

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age：1 months to 18 years
  • Chronic Granulomatous Disease
  • with severe infections

You may not qualify if:

  • \> 18 years of age
  • infections are treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Location

Related Publications (4)

  • 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. No abstract available.

    PMID: 27056268BACKGROUND

  • 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. No abstract available.

    PMID: 26386811BACKGROUND

  • 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.

    PMID: 25498313BACKGROUND

  • Hui X, Liu D, Wang W, Hou J, Ying W, Zhou Q, Yao H, Sun J, Wang X. Low-Dose Pioglitazone does not Increase ROS Production in Chronic Granulomatous Disease Patients with Severe Infection. J Clin Immunol. 2020 Jan;40(1):131-137. doi: 10.1007/s10875-019-00719-z. Epub 2019 Nov 19.

    PMID: 31745699DERIVED

MeSH Terms

Conditions

Granulomatous Disease, ChronicInfections

Interventions

Pioglitazone

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal DysfunctionLeukocyte DisordersHematologic DiseasesHemic and Lymphatic DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesImmunologic Deficiency SyndromesImmune System DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ThiazolidinedionesThiazolesSulfur CompoundsOrganic ChemicalsAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

February 27, 2017

First Posted

March 15, 2017

Study Start

September 1, 2017

Primary Completion

October 20, 2019

Study Completion

October 20, 2019

Last Updated

October 22, 2019

Record last verified: 2019-10

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)