UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000032119
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

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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 or person

Institute

ReqMed Company, Ltd.
Pharmaceutical and Business Development

Institute

Department

Personal name



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

Value
https://center6.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