Unique ID issued by UMIN | UMIN000032119 |
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Receipt number | 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 10:30:08 |
An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients including children with Mucopolysaccharidosis and Mucolipidoses
An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients with Mucopolysaccharidosis and Mucolipidoses
An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients including children with Mucopolysaccharidosis and Mucolipidoses
An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients with Mucopolysaccharidosis and Mucolipidoses
Japan |
Mucopolysaccharidosis and Mucolipidoses
Endocrinology and Metabolism | Pediatrics |
Others
NO
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
Safety,Efficacy
Exploratory
Explanatory
Phase II
Relief of bone cartilage and joint symptoms
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
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
NO
NO
Institution is not considered as adjustment factor.
NO
No need to know
1
Treatment
Medicine |
<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.
3 | years-old | <= |
Not applicable |
Male and Female
Patients who have been diagnosed with MPS or Mucolipidosis and have contracture or restricted ROM
(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
6
1st name | |
Middle name | |
Last name | Yasuyuki Suzuki |
Gifu University
Medical Education Development Center
1-1 Yanagido, Gifu, 501-1194, JAPAN
058-230-6470
ysuz@gifu-u.ac.jp
1st name | |
Middle name | |
Last name | Kenji Tanaka |
ReqMed Company, Ltd.
Pharmaceutical and Business Development
Mitsui-Life Bldg. 1-7-23 Morino, Machida-City TOKYO
042-732-2207
http://www.reqmed.co.jp/
tanaka@reqmed.co.jp
ReqMed Company, Ltd.
Pharmaceutical and Business Development
Japan Agency for Medical Research and Development (AMED)
Japanese Governmental office
Japan
NO
2018 | Year | 04 | Month | 05 | Day |
Unpublished
No longer recruiting
2017 | Year | 11 | Month | 10 | Day |
2017 | Year | 12 | Month | 08 | Day |
2019 | Year | 01 | Month | 31 | Day |
2019 | Year | 02 | Month | 15 | Day |
2019 | Year | 02 | Month | 28 | Day |
2019 | Year | 03 | Month | 15 | Day |
2018 | Year | 04 | Month | 05 | Day |
2018 | Year | 05 | Month | 09 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034840
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