Chronic Granulomatous DiseasePhase 1/2NCT01338675
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
A Phase 1/2 study testing Busulfan in people with Chronic Granulomatous Disease, run by Seoul National University Hospital at 1 site in South Korea.
Trial summary
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- Busulfan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jan 2011to Dec 2013
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who are diagnosed as CGD.
- Patients who need hematopoietic stem cell transplantation with busulfan based conditioning regimen.
- Age: No limit.
- Performance status: ECOG 0-2.
- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
- Heart: a shortening fraction > 30% and ejection fraction > 45%.
Show all 11Show fewer
- Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.
- Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.
- Patients must lack any active viral infections or active fungal infection.
- Appropriate donor is available.
- Patients (or one of parents if patients age < 20) should sign informed consent.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Seoul National University Hospital
Seoul, Daehangno, Jongno-gu
Hyoung Jin Kang, MD, PhD, 82 2 2072 3304, kanghj@snu.ac.kr
Ji Won Lee, MD, 82 2 2072 0177, agnesjw@hanmail.net
About this study
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
Read the detailed description
Chronic granulomatous disease is one of the rare congenital immunodeficiency which can be cured by hematopoietic stem cell transplantation. Previous myeloablative conditioning regimen has problems related to the severe toxicities, and non-myeloablative conditioning regimen has the risk of graft failure. Recently, reduced-intensity myeloablative conditioning regimen with busulfan and fludarabine was used usually in leukemia patients.
Busulfan is a highly toxic drug with narrow therapeutic window. In this study we plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- Busulfan
- Keywords
- Chronic granulomatous disease, Hematopoietic Stem Cell Transplantation
- 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.
BusulfanDrug
First dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study
| Study group | Role | What they receive |
|---|---|---|
| Busulfan | Experimental | First dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study Receives: Drug: Busulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment rate
To evaluate engraftment rate after hematopoietic stem cell transplantation.
Measured: 1 month after transplantation
Secondary outcomes (1)
Transplantation-related mortality and toxicities
To evaluate 1-year event free survival after hematopoietic stem cell transplantation, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.
Measured: 1, 3, 6 and 12 months after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2011 (estimated)
- Study startJan 2011
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by Seoul National University HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seoul National University Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Hyoung Jin Kang, M.D., Ph.D, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01338675
- Sponsor's protocol ID
- SNUCH-SCT -1002
- Status last verified
- Nov 2013
- First submitted
- 4 Mar 2011
- Last updated
- 19 Nov 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
- ClinicalTrials.gov ID
- NCT01338675
- Acronym / sponsor ID
- SNUCH-SCT -1002
- Conditions
- Chronic Granulomatous Disease
- Keywords
- Chronic granulomatous disease, Hematopoietic Stem Cell Transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Seoul National University HospitalUniversity, hospital or other
- Study start
- Jan 2011
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 19 Apr 2011 (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.
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
Detailed description
Chronic granulomatous disease is one of the rare congenital immunodeficiency which can be cured by hematopoietic stem cell transplantation. Previous myeloablative conditioning regimen has problems related to the severe toxicities, and non-myeloablative conditioning regimen has the risk of graft failure. Recently, reduced-intensity myeloablative conditioning regimen with busulfan and fludarabine was used usually in leukemia patients.
Busulfan is a highly toxic drug with narrow therapeutic window. In this study we plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Busulfan | Experimental | Drug: BusulfanFirst dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Patients who are diagnosed as CGD.
- Patients who need hematopoietic stem cell transplantation with busulfan based conditioning regimen.
- Age: No limit.
- Performance status: ECOG 0-2.
- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| BusulfanFirst dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study | Drug | Busulfan |
Primary outcome
Engraftment rate
Time frame: 1 month after transplantation
To evaluate engraftment rate after hematopoietic stem cell transplantation.
1 secondary outcome
Transplantation-related mortality and toxicities
Time frame: 1, 3, 6 and 12 months after transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2013.
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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.
Hyoung Jin Kang, M.D., Ph.D principal investigator
Seoul National University Hospital
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 Busulfan in people with Chronic Granulomatous Disease, run by Seoul National University Hospital at 1 site in South Korea.
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 11 inclusion and 0 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 19 Nov 2013. 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 South Korea. 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?
Contact Hyoung Jin Kang, MD, PhD (82 2 2072 3304, kanghj@snu.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Engraftment rate (time frame: 1 month after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Nov 2013.