NCT00927134

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) children 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

87
On Track

Trial Health Score

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

Enrollment
2

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jun 2004

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

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

June 1, 2004

Completed
5.1 years until next milestone

First Submitted

Initial submission to the registry

June 22, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 24, 2009

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2011

Completed
Last Updated

September 27, 2011

Status Verified

September 1, 2011

Enrollment Period

6.5 years

First QC Date

June 22, 2009

Last Update Submit

September 26, 2011

Conditions

Chronic Granulomatous Disease

Keywords

Chronic Granulomatous Disease in children (=or< 18 years)

Outcome Measures

Primary Outcomes (1)

  • eradication of pre-existing therapy refractory bacterial and/or fungal infections

    6 months

Secondary Outcomes (1)

  • Reconstitution of ROS production by peripheral blood cells

    1 month

Interventions

retroviral SF71-gp91phox transduced CD34+ cellsGENETIC

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

Eligibility Criteria

Age1 Year - 18 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Children's Hospital

Zurich, CH-8032, Switzerland

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: 16582916BACKGROUND

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

  • Reinhard Seger, Prof Dr med

    University Children's Hospital, Zurich

    PRINCIPAL INVESTIGATOR

  • Janine Reichenbach, PD Dr med

    University Children's Hospital, Zurich

    STUDY CHAIR

  • Ulrich Siler, Dr rer nat

    University Children's Hospital, Zurich

    STUDY CHAIR

  • Manuel Grez, Dr rer nat

    Georg Speyer Research Institute, Frankfurt a.M.

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 22, 2009

First Posted

June 24, 2009

Study Start

June 1, 2004

Primary Completion

December 1, 2010

Study Completion

September 1, 2011

Last Updated

September 27, 2011

Record last verified: 2011-09

Locations

CH(1)