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 including children with Mucopolysaccharidosis and Mucolipidoses - An Exploratory Clinical phase II study of Pentosan Polysulfate Sodium (NaPPS) in Patients with Mucopolysaccharidosis and Mucolipidoses

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000032119

Enrollment

Registered

2018-04-05

Start date

2017-12-08

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis and Mucolipidoses

Interventions

&lt

Dose&gt

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

Administration period&gt

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

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients who have been diagnosed with MPS or Mucolipidosis and have contracture or restricted ROM

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Relief of bone cartilage and joint symptoms	—

Secondary

Measure	Time frame
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	—

Measure

Time frame

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

—

Countries

Japan

Contacts

Public ContactKenji Tanaka

ReqMed Company, Ltd. Pharmaceutical and Business Development

tanaka@reqmed.co.jp042-732-2207

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026