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UMIN ID:

Recruitment status No longer recruiting
Unique ID issued by UMIN UMIN000032119
Receipt No. R000034840
Scientific Title An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients including children with Mucopolysaccharidosis and Mucolipidoses
Date of disclosure of the study information 2018/04/05
Last modified on 2018/05/09

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Basic information
Public title An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients including children with Mucopolysaccharidosis and Mucolipidoses
Acronym An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients with Mucopolysaccharidosis and Mucolipidoses
Scientific Title An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients including children with Mucopolysaccharidosis and Mucolipidoses
Scientific Title:Acronym An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients with Mucopolysaccharidosis and Mucolipidoses
Region
Japan

Condition
Condition Mucopolysaccharidosis and Mucolipidoses
Classification by specialty
Endocrinology and Metabolism Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To evaluate the long-term safety and explore the efficacy of subcutaneous NaPPS in Patients with Mucopolysaccharidosis and Mucolipidoses for relief of bone cartilage and joint symptoms
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2 Explanatory
Developmental phase Phase II

Assessment
Primary outcomes Relief of bone cartilage and joint symptoms
Key secondary outcomes Efficacy endpoints
[1] Determination of GAGs (in blood and urine)
[2] bone metabolism biomarkers: ADAMTS-4, ADAMTS-5, COMP, MMP-3, type I collagen, type II collagen, type X collagen
[3] Joint mobility assessment: shoulder joints, elbow joints, wrist joints, hip joints, knee joints, foot joints, and trunk
[4] Activities of daily living (ADL) questionnaire: assessment of bodily movement, bodily movement associated with cognitive function, cognitive function, and other symptoms
[5] Pain assessment (Graphic Rating Scale(GRS))
[6] Inflammatory biomarkers: IL-1beta, MIF, MIP-1alpha, RANTES and TNF-alpha
[7] X-ray examinations of joints: shoulder joints, elbow joints, wrist joints, hip joints, knee joints, foot joints, and spine
[8] Spinal MRI scan
[9] Abdominal CT scan: hepatosplenomegaly
[10] Knee MRI scan
[11] Neurological examination: numbness
[12] Motor dysfunction test: 6-minute walking distance
[13] Ophthalmological and otological examinations: corneal opacity, retinal pigmentary degeneration, hearing, and middle otitis

Base
Study type Interventional

Study design
Basic design Single arm
Randomization Non-randomized
Randomization unit
Blinding Open -no one is blinded
Control Uncontrolled
Stratification NO
Dynamic allocation NO
Institution consideration Institution is not considered as adjustment factor.
Blocking NO
Concealment No need to know

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 <Dose>
The starting prescription dose is 1.0mg/kg
If the principal investigator (or sub-investigator) judged that the tolerability of the case where safety of 4 weeks was confirmed was not problematic, the prescription dose can be increased up to 1.5 mg/kg.
If the principal investigator (or sub-investigator) judged that the tolerability of the case was problematic, the prescription dose can be decreased up to 1.0 mg/kg.
<Administration period>
Let the continuous administration period for 13 weeks (administration period) and the washout period for 4 weeks (evaluation period) be 1course, repeat 2 courses.
Perform final evaluation at the end of the 2nd course evaluation period.
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
3 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria Patients who have been diagnosed with MPS or Mucolipidosis and have contracture or restricted ROM
Key exclusion criteria (1) Patients with any of the following conditions:
Hemorrhagic diathesis (gastrointestinal hemorrhage such as gastric and duodenal ulcers, and suspected cerebral hemorrhage)
Cardiovascular disease such as aneurysms or blood disease such as thrombocytopenia
Heparin-induced thrombocytopenia (HIT) Type II
Serious allergic disease or serious complications
Keloid diathesis
Taking oral immunosuppressants or corticosteroids
(2) Patients with any of the following medical histories:
Hypersensitivity to heparin or pentosan polysulfate
HIT Type II
Surgical operation within 6 months before the start of study treatment
Drug hypersensitivity or thrombocytopenia
Serious food allergy
Target sample size 6

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Yasuyuki Suzuki
Organization Gifu University
Division name Medical Education Development Center
Zip code
Address 1-1 Yanagido, Gifu, 501-1194, JAPAN
TEL 058-230-6470
Email ysuz@gifu-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Kenji Tanaka
Organization ReqMed Company, Ltd.
Division name Pharmaceutical and Business Development
Zip code
Address Mitsui-Life Bldg. 1-7-23 Morino, Machida-City TOKYO
TEL 042-732-2207
Homepage URL http://www.reqmed.co.jp/
Email tanaka@reqmed.co.jp

Sponsor
Institute ReqMed Company, Ltd.
Pharmaceutical and Business Development
Institute
Department

Funding Source
Organization Japan Agency for Medical Research and Development (AMED)
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization Japan

Other related organizations
Co-sponsor
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

Other administrative information
Date of disclosure of the study information
2018 Year 04 Month 05 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 No longer recruiting
Date of protocol fixation
2017 Year 11 Month 10 Day
Date of IRB
Anticipated trial start date
2017 Year 12 Month 08 Day
Last follow-up date
2019 Year 01 Month 31 Day
Date of closure to data entry
2019 Year 02 Month 15 Day
Date trial data considered complete
2019 Year 02 Month 28 Day
Date analysis concluded
2019 Year 03 Month 15 Day

Other
Other related information

Management information
Registered date
2018 Year 04 Month 05 Day
Last modified on
2018 Year 05 Month 09 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034840

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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