Treatment of patients, who have Mucopolysaccharidosis Type I, receiving pentosan polysulfate subcutaneous injections weekly

• Conditions: Mucopolysaccharidosis 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]

• Registration Number: EUCTR2014-000350-11-DE
• Lead Sponsor: Multiplex Pharma Holdings LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03-10
• Last Update Posted: 16 November 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 6

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
F.1.3.1 Number of subjects for this age range

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.
11. For females: pregnancy or lactation.
12. For females of childbearing potential: not willing and able to use a contraceptive method, which the investigator considers reliable. Reliable methods for women are hormonal contraceptives, surgical intervention (e.g. tubal ligation), intrauterine device (IUD) and sexual abstinence. Women must use a reliable method from 6 weeks before the first administration of study medication until 3 weeks after the last administration of the study medication.
13. Participation in another clinical study involving a new chemical entity within 3 months and for studies involving biologicals 6 months prior to enrolment in this study.
14. The subject has received vaccination(s) within 1 month prior to enrollment, or is unwilling to postpone vaccinations for the duration of the study.
15. Subjects who are unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified