NCT01338675

Brief Summary

In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2011

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

March 4, 2011

Completed
2 months until next milestone

First Posted

Study publicly available on registry

April 19, 2011

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

November 19, 2013

Status Verified

November 1, 2013

Enrollment Period

2.9 years

First QC Date

March 4, 2011

Last Update Submit

November 17, 2013

Conditions

Chronic Granulomatous Disease

Keywords

Chronic granulomatous diseaseHematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

  • Engraftment rate

    To evaluate engraftment rate after hematopoietic stem cell transplantation.

    1 month after transplantation

Secondary Outcomes (1)

  • Transplantation-related mortality and toxicities

    1, 3, 6 and 12 months after transplantation

Study Arms (1)

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

Drug: Busulfan

Interventions

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

Busulfan

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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%.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Seoul National University Hospital

Seoul, Daehangno, Jongno-gu, South Korea

RECRUITING

MeSH Terms

Conditions

Granulomatous Disease, Chronic

Interventions

Busulfan

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

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur Compounds

Study Officials

  • Hyoung Jin Kang, M.D., Ph.D

    Seoul National University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Hyoung Jin Kang, MD, PhD

CONTACT

82 2 2072 3304kanghj@snu.ac.kr

Ji Won Lee, MD

CONTACT

82 2 2072 0177agnesjw@hanmail.net

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

March 4, 2011

First Posted

April 19, 2011

Study Start

January 1, 2011

Primary Completion

December 1, 2013

Study Completion

December 1, 2013

Last Updated

November 19, 2013

Record last verified: 2013-11

Locations

KR(1)