An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy.

Multiplex Pharma Holdings LLC0 sites6 target enrollmentMarch 10, 2014

ConditionsMucopolysaccharidosis type I (alpha-L-Iduronidase deficiency)MedDRA version: 17.0Level: LLTClassification code 10056888Term: Mucopolysaccharidosis ISSystem Organ Class: 100000004850 MedDRA version: 17.0Level: LLTClassification code 10056887Term: Mucopolysaccharidosis IH/SSystem Organ Class: 100000004850 MedDRA version: 17.0Level: LLTClassification code 10028094Term: Mucopolysaccharidosis IHSystem Organ Class: 100000004850 Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

DrugsPentosan Polysulfat SP 54® injection solution

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Mucopolysaccharidosis type I (alpha-L-Iduronidase deficiency)
Sponsor: Multiplex Pharma Holdings LLC
Enrollment: 6
Status: Active, not recruiting
Last Updated: 10 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

March 10, 2014

End Date

TBD

Last Updated

10 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Multiplex Pharma Holdings LLC

Eligibility Criteria

Inclusion Criteria

•1\. Male or female.
•2\. Age \=18 years.
•3\.Diagnosed as having Mucopolysaccharidosis Type I (alpha\-L\-Iduronidase deficiency) and have been treated with enzyme replacement therapy (ERT) for a minimum of 24 months.
•4\. Joint pain and/or reduced joint range of motion at time of enrolment according to the investigator.
•5\. Written informed consent upon enrolment.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1\.2 Adults (18\-64 years) yes
•F.1\.2\.1 Number of subjects for this age range 6
•F.1\.3 Elderly (\>\=65 years) no

Exclusion Criteria

•1\. History of hypersensitivity to pentosan polysulfate or any of the excipients.
•2\. Known allergic reaction to heparin or pentosan polysulfate e.g. heparin induced thrombocytopenia.
•3\. Clinically relevant concomitant severe condition (with the exception of signs and symptoms relating to MPSI) e.g. gastrointestinal (including gastrointestinal ulcerations, polyps, or diverticula), renal, hepatic (ALT and/or AST \>10 times upper limit of reference range), pancreatic, pulmonary, cardiovascular (including endocarditis lenta), aneurysm, cerebral hemorrhage, hematological (including thrombocytopenia, hemophilia, hemorrhagic diathesis), endocrinological, suspected tumors with risk of bleeding, neurological, or psychiatric conditions in the opinion of the investigator.
•4\. Planned invasive procedure other than blood sampling.
•5\. History of or planned bone marrow transplant.
•6\. Clinically relevant abnormal laboratory test results including liver enzymes, thrombocytopenia, prolonged clotting time.
•7\. Intake of anti\-inflammatory agents e.g. corticosteroids, non\-steroidal anti\-inflammatory drugs (NSAIDs) other than ibuprofen or paracetamol within 10 half lives (t½) of the drug prior to first dosing and throughout the study.
•8\. Intake of anti\-thrombotic agents e.g. heparin, tissue plasminogen activator (t\-PA), streptokinase, aspirin within 10 half lives (t½) of the drug prior to first dosing and throughout the study. Coumarin anticoagulants are permitted.
•9\. Known history or presence of alcohol or substance abuse.
•10\. Known hepatitis B or C or human immunodeficiency virus (HIV) or syphilis seropositivity. Note: testing for hepatitis and HIV will be performed as part of the screening procedures.