EUCTR2007-006345-40-DKActive, not recruitingNot Applicable
A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy
Shire Pharmaceutical Ireland Limited0 sitesStarted: January 14, 2008Last updated:
Trial Snapshot
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Sponsor
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •The patients from the phase I trial (EudraCT number: 2006-005341-11) must meet the following criteria to be enrolled in the study:
- •1. Subject’s legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
- •2. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
Exclusion Criteria
- •Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
- •1. Spasticity so severe to inhibit transportation
- •2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
- •3. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
- •4. Use of any investigational product other than rhASA within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
Investigators
Similar Trials
Active, not recruiting
Phase 1
A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients with high residual level of voluntary functioEUCTR2007-007165-20-DKShire Pharmaceuticals Ireland Limited6
Active, not recruiting
Not Applicable
A single center, open-label, non-randomized, uncontrolled, multiple-dose, dose escalation study of the safety, pharmacokinetics and efficacy of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy (MLD)ate infantile metachromatic leukodystrophy (MLD)MedDRA version: 9.1Level: LLTClassification code 10024381Term: LeukodystrophyEUCTR2006-005341-11-DKShire Pharmaceuticals Ireland Limited12
Active, not recruiting
Not Applicable
Continuation of cholic acid treatment for people with bile acid disorders who have previously taken part in clinical trials of cholic acid capsules.Patients with inborn errors of bile acid synthesis and metabolismMedDRA version: 14.0Level: PTClassification code 10070882Term: Inborn error in primary bile acid synthesisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004528-36-Outside-EU/EEAAsklepion Pharmaceuticals, LLC30
Completed
Not Applicable
Single center, open-label, non-randomized, non-placebo-controlled study to investigate the metabolism, excretion pattern, mass balance, safety, tolerability and pharmacokinetics after single administration of 200 mg [14C]rogaratinib (oral solution) in healthy male subjectsNL-OMON46572Bayer AG6
Completed
Not Applicable
Single center, open-label, non-randomized, non-placebo-controlled study to investigate the pharmacokinetics, metabolic disposition and mass balance after single administration of 5 mg [14C]BAY 1021189 (oral solution) in healthy male subjectsheart failureCardiovascular diseases10019280NL-OMON40889Bayer6