NL-OMON36295
Completed
Phase 2
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
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- MPS IIIA
- Sponsor
- Shire
- Enrollment
- 6
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
Trial is onging in other countries
Investigators
Eligibility Criteria
Inclusion Criteria
- •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
Exclusion Criteria
- •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.
Outcomes
Primary Outcomes
Not specified
Similar Trials
Completed
Phase 2
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
Completed
Phase 2
A dose finding study to assess the safety and efficacy of K-877 in patients with statin-controlled LDL-C but abnormal lipid levelsabnormal cholesterol valuesdyslipidemia10013317NL-OMON40373Kowa Research Europe52
Completed
Phase 2
A Phase 2, Multicenter, Randomized, Double-Blind, Parallel, Placebo-Controlled Study of LY3074828 in Subjects with Moderate to Severe Ulcerative Colitisinflamatory bowel diseaseUlcerative colitis10017969NL-OMON46940Eli Lilly and Company7
Completed
Phase 2
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)Amyotrophic Lateral Sclerosism (ALS)Motor Neuron Disease10029317NL-OMON45879Cytokinetics, Inc.11
Completed
Phase 2
A Phase 2, Multi-Center, Double-Blind, Randomized, Placebo-Controlled Study of MGL-3196 in Patients With Heterozygous Familial HypercholesterolemiaNL-OMON45499Madrigal Pharmaceuticals, Inc.24