UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000009758 |
|---|---|
| Receipt number | R000011433 |
| Scientific Title | Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study |
| Date of disclosure of the study information | 2013/01/15 |
| Last modified on | 2019/01/16 15:51:57 |
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Basic information
Public title
Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study
Acronym
Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study
Scientific Title
Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study
Scientific Title:Acronym
Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study
Region
| Japan |
Condition
Condition
Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome)
Classification by specialty
| Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To determine the recommended dose of treosulfan when combined with low-dose irradiation, fludarabine, thymoglobulin for preparative regimen in patients with mucopolysaccharidosis
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Explanatory
Developmental phase
Phase I
Assessment
Primary outcomes
The incidence of severe RRT (Grade III/IV) on day 28 after HSCT with Treosulfan as a conditioning regimen.
Key secondary outcomes
Regimen-related toxicity, engraftment rate, survival rate at 28 days posttransplant, survival rate at 100 days posttransplant, chimeric study, GVHD, hepatic SOS, event-free survival and overall survival at 1 year posttransplant.
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Treosulfan 14 g/m2, intravenous,
day -6 -5 -4
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| Not applicable |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1. Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome)
2. Body weight; equal or more than 10 kg
3. ECOG performance status; 0 or 1
4. The transplant should be the first one for the patient. Retransplantation is acceptable under the following conditions; after over 6 months from previous transplantation, and has no effect of previous preconditioning regimen
5. One of the following stem cell donor is available
1) 6/6 or 5/6 (either class I or class II) HLA-A/B/DR serologically matched related bone marrow
2) 6/6 HLA-A/B/DR allelic matched or HLA-DR/DRB1 serologically/allelic mismatched unrelated bone marrow
3) Cord blood units with 6/6, 5/6, or 4/6 HLA serologically match and nucleated cell dose equal or more than 3.5 x 10e7/kg, and CD34+ cell dose equal or more than 1.0 x 10e5/kg
6. Major organ dysfunction (laboratory data)
1) Ejection fraction at rest (UCG); equal or more than 50%
2) Arterial oxygen saturation without oxygen supplementation; equal or greater than 93%
3) Serum creatinine < 1.3 mg/dl
4) Total bilirubin <1.6 mg/dl or AST(GOT) < 2 x normal of each institution
7. Patients without following active infections
1) Pathogen-proven bacterial infection requiring antimicrobial treatment
2) Imaging study (XP, CT, US, MRI) manifested infectious foci requiring antimicrobial treatment
3) Abscess formation or necrotizing infection
4) Viral infection necessitating systemic antiviral agents
5) Culture-positive or PCR-positive tuberculosis/non-tuberculous mycobacterial infection
6) Pneumocystis pneumonia
7) Meningitis, Encephalitis, Encephalopathy
8) Protozoan infection
9) Intraocular fungal infection
8. No previous history of hypersensitivity to the following drugs that are used for conditioning or prophylaxis of GVHD
Treosulfan (L-threitol-1,4-bis-methanesulfonate; dihydroxybusulfan)
Fludarabine
Antithymocyte globulin
Cyclosporine
Methotrexate
Tacrolimus
Key exclusion criteria
1. Down syndrome
2. HIV-positivity
Target sample size
12
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Shunichi Kato |
Organization
Tokai University School of Medicine
Division name
Department of Cell Transplantation and Regenerative Medicine
Zip code
Address
143, Shimokasuya, Isehara, Kanagawa, 259-1193, Japan
TEL
0463-93-1121
skato@is.icc.u-tokai.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Hiromasa Yabe |
Organization
Tokai University School of Medicine
Division name
Department of Cell Transplantation and Regenerative Medicine
Zip code
Address
143, Shimokasuya, Isehara, Kanagawa, 259-1193, Japan
TEL
0463-93-1121
Homepage URL
yabeh@is.icc.u-tokai.ac.jp
Sponsor or person
Institute
Tokai University School of Medicine
Institute
Department
Personal name
Funding Source
Organization
Government of Japan
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
1. School of Human Health Science Faculty of Medicine Kyoto University
2. Department of Pediatrics, Japanese Red cross Nagoya Daiichi Hospital
3. Department of Pediatrics, Nihon University
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
東海大学病院（神奈川県）
京都大学病院（京都府）
名古屋第一赤十字病院（愛知県）
日本大学病院（東京都）
Other administrative information
Date of disclosure of the study information
| 2013 | Year | 01 | Month | 15 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Terminated
Date of protocol fixation
| 2013 | Year | 06 | Month | 24 | Day |
Date of IRB
Anticipated trial start date
| 2013 | Year | 06 | Month | 24 | Day |
Last follow-up date
| 2014 | Year | 12 | Month | 23 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2013 | Year | 01 | Month | 11 | Day |
Last modified on
| 2019 | Year | 01 | Month | 16 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000011433
