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Clinical Trials/NCT00564759
NCT00564759UnknownPhase 1

Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease

Johann Wolfgang Goethe University Hospital2 sites in 1 country2 target enrollmentStarted: January 1, 2004Last updated:
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Conditions
Drugs

Trial Snapshot

Phase
Phase 1
Sponsor
Enrollment
2
Locations
2
Primary Endpoint
safety, toxicity and feasibility
Study OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Single Group
Masking
None

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
Male
Accepts Healthy Volunteers
No

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)

Outcomes

Primary Outcomes

safety, toxicity and feasibility

Time Frame: 2 years

Secondary Outcomes

  • Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit(2 years)

Investigators

Sponsor
Johann Wolfgang Goethe University Hospital
Sponsor Class
Other

Study Sites (2)

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