NL-OMON36295已完成2 期
A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan-N-sulfatase (rhHNS) Intrathecal Administration via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA) - MPS IIIA ERT study
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 6
研究概览
简要总结
Trial is onging in other countries
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •1. a.)Patients have a documented deficiency in sulfamidase enzyme activity of *10% of the lower limit of the normal range as measured in fibroblasts or leukocytes (based on measurements by a laboratory that is acceptable to Shire HGT).
- •AND EITHER b or C
- •b.)Patients have a normal enzyme activity level of at least 1 other sulfatase (to rule out multiple sulfatase deficiency) as measured in fibroblasts or leukocytes (based on measurements by a laboratory that is acceptable to Shire HGT).
- •c.) Patients have 2 documented mutations (based on assessments by a laboratory that is acceptable to Shire HGT).
- •2. The patient is *3 years of age and has a developmental age above 1 year (developmental age will be determined by the screening neurocognitive and developmental tests).
- •3. Patients must be medically stable, in the opinion of the Investigator, to accommodate the protocol requirements, including travel, assessments, and IDDD surgery, without placing an undue burden on the patient/patient's family.
- •4. The patient*s parent(s) or legal guardian must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form
排除标准
- •1. The patient has significant non-MPS IIIA related central nervous system (CNS) impairment or behavioral disturbances that would confound the scientific integrity or interpretation of study assessments, as determined by the Investigator.
- •2. The patient has MPS IIIA behavioral-related issues, as determined by the Investigator that would preclude performance of study neurocognitive and developmental testing procedures.
- •3. The patient is pregnant, breast feeding, or is a female patient of childbearing potential who will not or cannot comply with the use of an acceptable method of birth control
- •4. The patient is blind and/or deaf
- •5. The patient has any known or suspected hypersensitivity to anesthesia or is thought to have an unacceptably high risk for anesthesia due to airway compromise or other conditions.
- •6. The patient or the patient's family has a history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns.
- •7. The Investigator may choose to exclude patients who have had complications resulting from prior lumbar punctures.
- •8. The patient has a CNS shunt.
- •9. The patient has skeletomuscular/spinal abnormalities or other contraindications for the surgical implantation of the IDDD.
- •10. The patient has a history of poorly controlled seizure disorder.
- •11. The patient is currently receiving psychotropic or other medications, which in the Investigator*s opinion, would be likely to substantially confound test results and the dose and regimen of which cannot be kept constant throughout the study.
- •12. The patient cannot sustain absence from aspirin, non-steroidal medications, or medications that affect blood clotting within 1 week prior to a relevant study-related procedure (eg, device implantation if applicable), or has ingested such medications within 1 week before any procedures in which any change in clotting activity would be deleterious.
- •13. The patient has received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within the 30 days prior to, or during the study, or is currently enrolled in another study that involves an investigational drug or device (screening through safety follow-up contact).
- •14. The patient has received a hematopoietic stem cell or bone marrow transplant.
- •15. The patient*s parent(s), or patient*s legal guardian(s) is/are unable to provide consent or the patient cannot provide assent,
研究者
相似试验
已完成
2 期
A phase I/II study evaluating the safety and activity of Pegylated recombinant human Arginase (BCT-100) in Relapsed/refractory cancers of Children and young adultsonbehandelbare en/of terugkerende vorm van kanker: leukemie, hoogradig glioom, neuroblastoom, sarcoomrelapsed or refractory cancer10027655NL-OMON49172Cancer Research UK Clinical Trials Unit4
已完成
2 期
A dose finding study to assess the safety and efficacy of K-877 in patients with statin-controlled LDL-C but abnormal lipid levelsdyslipidemiaabnormal cholesterol values10013317NL-OMON40373Kowa Research Europe52
已完成
2 期
A Phase 2, Multicenter, Randomized, Double-Blind, Parallel, Placebo-Controlled Study of LY3074828 in Subjects with Moderate to Severe Ulcerative Colitisinflamatory bowel disease10017969Ulcerative colitisNL-OMON46940Eli Lilly and Company7
已完成
2 期
A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability Of CK-2127107 In Patients with Amyotrophic Lateral Sclerosis (ALS)Motor Neuron DiseaseAmyotrophic Lateral Sclerosism (ALS)10029317NL-OMON45879Cytokinetics, Inc.11
已完成
2 期
A Phase 2, Multi-Center, Double-Blind, Randomized, Placebo-Controlled Study of MGL-3196 in Patients With Heterozygous Familial HypercholesterolemiaHeterozygous Familial HypercholesterolemiaNL-OMON45499Madrigal Pharmaceuticals, Inc.24