NCT00564759

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.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
2

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2004

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2004

Completed
3.9 years until next milestone

First Submitted

Initial submission to the registry

November 26, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 28, 2007

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2008

Completed
Last Updated

November 28, 2007

Status Verified

November 1, 2007

First QC Date

November 26, 2007

Last Update Submit

November 27, 2007

Conditions

Granulomatous Disease, Chronic

Keywords

CGD

Outcome Measures

Primary Outcomes (1)

  • safety, toxicity and feasibility

    2 years

Secondary Outcomes (1)

  • Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit

    2 years

Interventions

retroviral SF71-gp91phox transduced CD34+ cellsDRUG

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

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Hospital, Hematology

Frankfurt, 60596, Germany

Location

Related Publications (1)

  • 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.

    PMID: 16582916RESULT

MeSH Terms

Conditions

Granulomatous Disease, Chronic

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

Study Officials

  • Dieter Hoelzer, MD, PhD

    Goethe University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

November 26, 2007

First Posted

November 28, 2007

Study Start

January 1, 2004

Study Completion

December 1, 2008

Last Updated

November 28, 2007

Record last verified: 2007-11

Locations

DE(1)