EUCTR2006-005341-11-DK进行中（未招募）不适用
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)
Shire Pharmaceuticals Ireland Limited0 个研究点目标入组 12 人开始时间: 2006年11月8日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •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 patient must have a confirmed diagnosis of MLD as defined by:
- •-ASA activity < 10 nmol/h/mg in leukocytes
- •-Presence of elevated sulfatide in urine
- •3. The patient must have a confirmed nerve conduction velocity < 2 standard deviations (from the appropriate age level)
- •4. The patient must have a residual level of voluntary function (as judged by the investigator), including presence of residual cognitive function (attention, executive and visual functions) as well as the presence of residual voluntary motor function in one upper or lower limb as a minimum.
- •5. The patient must have an age at the time of screening = 1 year and < 6 years
- •6. The patient must have had onset of symptoms before the age of 4 years
- •7. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
- •8. The patients’ medical record must document that the legal guardian(s) has had independent counselling or a consultation regarding stem cell transplantation in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative
- •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
排除标准
- •1. Presence of a gross motor function measure (GMFM < 10)
- •2. Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing)
- •3. Spasticity so severe to inhibit transportation
- •4. Known multiple sulfatase deficiency
- •5. Presence of major congenital abnormality
- •6. Presence of known chromosomal abnormality and syndromes affecting psychomotor development
- •7. History of stem cell transplantation
- •8. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition
- •9. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
- •10. Use of any investigational product within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations.
- •11. Received ERT with rhASA from any source
- •12. Planned or anticipated initiation of antispastic treatment after trial initiation
研究者
相似试验
进行中（未招募）
1 期
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
进行中（未招募）
不适用
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 leukodystrophyEUCTR2007-006345-40-DKShire Pharmaceutical Ireland Limited
进行中（未招募）
不适用
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
已完成
不适用
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
已完成
不适用
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