Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02034630 |
Recruitment Status : Unknown
Verified April 2018 by Seoul National University Hospital.
Recruitment status was: Recruiting
First Posted : January 13, 2014
Last Update Posted : April 8, 2019
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Genetic Rare Disease | Drug: Busulfan | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 5 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease |
Study Start Date : | January 2014 |
Estimated Primary Completion Date : | April 2019 |
Estimated Study Completion Date : | December 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: Busulfan
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
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 |
- To evaluate engraftment rate [ Time Frame: 1 year ]To evaluate engraftment rate
- To evaluate treatment related mortality [ Time Frame: 1 year ]To evaluate treatment related mortality
- To evaluate survival rate [ Time Frame: 1, 3, 6 and 12 months after transplantation ]To evaluate survival rate
- To evaluate toxicities associated with hematopoietic stem cell transplantation [ Time Frame: 1, 3, 6 and 12 months after transplantation ]To evaluate toxicities associated with hematopoietic stem cell transplantation
- To evaluate acute GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ]To evaluate acute GVHD
- To evaluate chronic GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ]To evaluate chronic GVHD

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | up to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients who are diagnosed as genetic rare disease.
- Patients who need hematopoietic stem cell transplantation
- Age: up to 21 years
- 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. 1. Heart: a shortening fraction > 30% and ejection fraction > 45%. 2. Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal. 3. 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: Matched in 6/6 of A, B, DR loci.
- Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion Criteria:
- Pregnant or nursing women.
- Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- Psychiatric disorder that would preclude compliance.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02034630
Contact: Hyoung Jin Kang, MD, PhD | 82 2 2072 3304 | kanghj@snu.ac.kr | |
Contact: Ji Won Lee, MD | 82 2 2072 4192 | agnesjw@hanmail.net |
Korea, Republic of | |
Seoul National University Hospital | Recruiting |
Seoul, Daehangno, Jongno-gu, Korea, Republic of | |
Contact: Hyoung Jin Kang, MD, PhD 82 2 2072 3304 kanghj@snu.ac.kr | |
Contact: Ji Won Lee, MD 82 2 2072 3452 agnesjw@hanmail.net |
Principal Investigator: | Hyoung Jin Kang, M.D., Ph.D | Seoul National University Hospital |
Responsible Party: | Seoul National University Hospital |
ClinicalTrials.gov Identifier: | NCT02034630 |
Other Study ID Numbers: |
SNUCH_GRDBuFlu |
First Posted: | January 13, 2014 Key Record Dates |
Last Update Posted: | April 8, 2019 |
Last Verified: | April 2018 |
hematopoietic stem cell transplantation |
Rare Diseases Disease Attributes Pathologic Processes Busulfan Alkylating Agents Molecular Mechanisms of Pharmacological Action |
Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antineoplastic Agents, Alkylating Antineoplastic Agents Myeloablative Agonists |