Gene Therapy for Chronic Granulomatous Disease | NCT00564759

Studies
Gene Therapy for Chronic Granulomatous Disease

NCT00564759

Sponsor

Johann Wolfgang Goethe University Hospital

Status

Unknown status

Last Update Posted

Nov 28, 2007Estimated

Enrollment

2Actual

Phase

Phase 1Phase 2

Conditions

Granulomatous Disease, Chronic

Interventions

retroviral SF71-gp91phox transduced CD34+ cells

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2007

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2004

Primary Completion Date

Not provided

Completion Date

Dec 2008Estimated

First Submitted Date

Nov 26, 2007

First Submission Date that Met QC Criteria

Nov 27, 2007

First Posted Date

Nov 28, 2007Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 27, 2007

Last Update Posted Date

Nov 28, 2007Estimated

Name	Class
German Federal Ministry of Education and Research	OTHER_GOV

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

2Actual

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
retroviral SF71-gp91phox transduced CD34+ cells	Drug	autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
safety, toxicity and feasibility		2 years

Secondary Outcomes

Measure	Description	Time Frame
Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit		2 years

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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)

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Dieter Hoelzer, MD, PhD	Goethe University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University Hospital, Hematology		Frankfurt		60596	Germany

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
16582916	Result	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.

ID	Term
D010585	Phagocyte Bactericidal Dysfunction
D007960	Leukocyte Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

ID	Type	Description	Link
DeReG 31
KSG 31