Chronic Granulomatous DiseasePhase 1/2XCGDinChildrenNCT00927134
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
A Phase 1/2 study testing retroviral SF71-gp91phox transduced CD34+ cells in people with Chronic Granulomatous Disease, run by University of Zurich at 1 site in Switzerland.
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) children with chronic granulomatous disease (CGD). Read more about this study
- Who is it for?
- Ages 1 year to 18 years, men only
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- retroviral SF71-gp91phox transduced CD34+ cells
- How many people
- 2 participants
- Where
- 1 siteSwitzerland
- Study dates
- Jun 2004to Sep 2011
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- > 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
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.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- retroviral SF71-gp91phox transduced CD34+ cells
- Keywords
- Chronic Granulomatous Disease in children (=or< 18 years)
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
retroviral SF71-gp91phox transduced CD34+ cellsGenetic
autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells
What is being measured
The primary outcome is the main question the study is designed to answer.
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Measured: 6 months
Secondary outcomes (1)
Reconstitution of ROS production by peripheral blood cells
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2009 (estimated)
- Study startJun 2004
- Primary completionDec 2010
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by University of ZurichThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Zurich
- Sponsor type
- University, hospital or other
- Collaborators
- Goethe University
- Responsible party
- Sponsor
- Investigators
- Reinhard Seger, Prof Dr med, Principal investigator, University Children's Hospital, ZurichJanine Reichenbach, PD Dr med, Study chair, University Children's Hospital, ZurichUlrich Siler, Dr rer nat, Study chair, University Children's Hospital, ZurichManuel Grez, Dr rer nat, Study chair, Georg Speyer Research Institute, Frankfurt a.M.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00927134
- Sponsor's protocol ID
- PedsZürich_GT05
- Acronym
- XCGDinChildren
- Status last verified
- Sep 2011
- First submitted
- 22 Jun 2009
- Last updated
- 27 Sep 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 16582916
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
- ClinicalTrials.gov ID
- NCT00927134
- Acronym / sponsor ID
- XCGDinChildren / PedsZürich_GT05
- Conditions
- Chronic Granulomatous Disease
- Keywords
- Chronic Granulomatous Disease in children (=or< 18 years)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of ZurichUniversity, hospital or other
- Collaborators
- Goethe University
- Study start
- Jun 2004
- Primary completion
- Dec 2010 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 24 Jun 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- > 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| retroviral SF71-gp91phox transduced CD34+ cellsautologous ex-vivo transduced (SF71-gp91phox)CD34+ cells | Genetic | – |
Primary outcome
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Time frame: 6 months
1 secondary outcome
Reconstitution of ROS production by peripheral blood cells
Time frame: 1 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2011.
- Background: PubMed 16582916Ott 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Reinhard Seger, Prof Dr med principal investigator
University Children's Hospital, Zurich
Janine Reichenbach, PD Dr med study chair
University Children's Hospital, Zurich
Ulrich Siler, Dr rer nat study chair
University Children's Hospital, Zurich
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing retroviral SF71-gp91phox transduced CD34+ cells in people with Chronic Granulomatous Disease, run by University of Zurich at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Sep 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
eradication of pre-existing therapy refractory bacterial and/or fungal infections (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00927134, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Sep 2011.