Gene Therapy for Chronic Granulomatous Disease

Granulomatous Disease, Chronic Clinical Trial

— CGD  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00564759
• Other study ID #: 58/59
• Secondary ID: DeReG 31KSG 31
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• First received: November 26, 2007
• Last updated: November 27, 2007
• Start date: January 2004
• Est. completion date: December 2008

Study information

• Verified date: November 2007
• Source: Johann Wolfgang Goethe University Hospitals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 2
• Est. completion date: December 2008
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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)

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Chronic Disease
• Granuloma
• Granulomatous Disease, Chronic

Intervention

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

Locations

Country	Name	City	State
Germany	University Hospital, Hematology	Frankfurt

Sponsors (2)

Lead Sponsor	Collaborator
Johann Wolfgang Goethe University Hospitals	German Federal Ministry of Education and Research

Country where clinical trial is conducted

Germany, 

References & Publications (1)

Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kühlcke 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, Lüthi U, Hassan M, Thrasher AJ, Hoelzer D, vo — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety, toxicity and feasibility		2 years
Secondary	Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit		2 years