Gene Therapy for Chronic Granulomatous Disease

NCT ID: NCT00564759

Last Updated: 2007-11-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-01-31

Study Completion Date

2008-12-31

Brief Summary

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The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan)patients with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

Detailed Description

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Conditions

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Granulomatous Disease, Chronic

Keywords

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CGD

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Blinding Strategy

NONE

Interventions

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retroviral SF71-gp91phox transduced CD34+ cells

autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* x-linked Chronic Granulomatous Disease
* history of life-threatening severe infections
* no HLA-matched related or non-related donor
* therapy resistent life threatening infections/organ dysfunction
* no other treatment options e.g. BMT

Exclusion Criteria

* \< 18 years of age
* HIV infection
* life expectancy \> 2 years
* infections treatable by conventional therapy (antibiotics, allogeneic granulocytes)
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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German Federal Ministry of Education and Research

OTHER_GOV

Sponsor Role collaborator

Johann Wolfgang Goethe University Hospital

OTHER

Sponsor Role lead

Principal Investigators

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Dieter Hoelzer, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Goethe University

Locations

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University Hospital, Hematology

Frankfurt, , Germany

Site Status

Countries

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Germany

References

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Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. doi: 10.1038/nm1393. Epub 2006 Apr 2.

Reference Type RESULT
PMID: 16582916 (View on PubMed)

Other Identifiers

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DeReG 31

Identifier Type: -

Identifier Source: secondary_id

KSG 31

Identifier Type: -

Identifier Source: secondary_id

58/59

Identifier Type: -

Identifier Source: org_study_id