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UMIN ID:

Recruitment status Terminated
Unique ID issued by UMIN UMIN000009758
Receipt No. 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

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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
Email 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
Email yabeh@is.icc.u-tokai.ac.jp

Sponsor
Institute Tokai University School of Medicine
Institute
Department

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
Email

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
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000011433

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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