Granulomatous Disease, ChronicPhase 1/2CGDNCT00564759
Gene Therapy for Chronic Granulomatous Disease
A Phase 1/2 study testing retroviral SF71-gp91phox transduced CD34+ cells in people with Granulomatous Disease, Chronic, run by Johann Wolfgang Goethe University Hospital at 1 site in Germany.
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). Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Granulomatous Disease, Chronic
- What is being tested?
- retroviral SF71-gp91phox transduced CD34+ cells
- How many people
- 2 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2004to Dec 2008
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- < 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, 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
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.
- Conditions
- Granulomatous Disease, Chronic
- Being tested
- retroviral SF71-gp91phox transduced CD34+ cells
- Keywords
- CGD
- 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+ cellsDrug
autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells
What is being measured
The primary outcome is the main question the study is designed to answer.
safety, toxicity and feasibility
Measured: 2 years
Secondary outcomes (1)
Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2007 (estimated)
- Study startJan 2004
- Study completionDec 2008 (estimated)
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 Granulomatous Disease, ChronicStudies of the same condition, open and under way.
- 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
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johann Wolfgang Goethe University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Investigators
- Dieter Hoelzer, MD, PhD, Principal investigator, Goethe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00564759
- Sponsor's protocol ID
- 58/59
- Other IDs
- DeReG 31, KSG 31
- Acronym
- CGD
- Status last verified
- Nov 2007
- First submitted
- 26 Nov 2007
- Last updated
- 28 Nov 2007
- 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 28 Nov 2007. 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
- ClinicalTrials.gov ID
- NCT00564759
- Acronym / sponsor ID
- CGD / 58/59
- Conditions
- Granulomatous Disease, Chronic
- Keywords
- CGD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Johann Wolfgang Goethe University HospitalUniversity, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Study start
- Jan 2004
- Study completion
- Dec 2008 (estimated)
- First posted
- 28 Nov 2007 (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)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
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- < 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, 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 retroviral transduced (SF71-gp91phox) CD34+ cells | Drug | – |
Primary outcome
safety, toxicity and feasibility
Time frame: 2 years
1 secondary outcome
Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2007.
- Results publication: 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.
Dieter Hoelzer, MD, PhD principal investigator
Goethe University
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 Granulomatous Disease, Chronic, run by Johann Wolfgang Goethe University Hospital at 1 site in Germany.
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: status not confirmed, as of the sponsor's last update on 28 Nov 2007. 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 Germany. 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?
Results are not available in the source record.
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?
safety, toxicity and feasibility (time frame: 2 years). 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 NCT00564759, 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 28 Nov 2007.