Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Pa… (NCT01155778) | Clinical Trial Compass
CompletedPhase 1/2
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Netherlands, United Kingdom12 participantsStarted 2010-06-01
Plain-language summary
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in lysosomes. MPS IIIA results from deficiency of the enzyme heparan N-sulfatase (sulfamidase). MPS IIIA symptoms arise on average at 7 months of age, with the average age of diagnosis at 4.5 years for the majority of patients. The central nervous system (CNS) is the most severely affected organ system in patients with MPS IIIA, evidenced by deficits in language development, motor skills, and intellectual development. In addition, there are abnormal behaviors including but not limited to aggression and excess motor activity/hyperactivity that contribute to disturbances in sleep.Overall, individuals with MPS IIIA have a marked developmental delay and significantly reduced lifespan of 15 years of age on average.
The purpose of this study is to determine the safety and tolerability of rhHNS via ascending doses administered via an a surgically implanted intrathecal drug delivery device (IDDD) intrathecal (IT) route once monthly (or every two weeks) for 6 months in patients with MPS IIIA.
Who can participate
Age range3 Years
SexALL
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Inclusion criteria
✓. a.) Patients had a documented deficiency in sulfamidase enzyme activity of ≤10% of the lower limit of the normal range as measured in fibroblasts or leukocytes.
✓. The patient was ≥3 years of age and had a developmental age ≥1 year.
✓. Patients must have been 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.
✓. The patient's parent(s) or legal guardian must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, the patient's parent(s), or legal guardian. The patients, patient's parents or legal guardian's consent and patient's assent as appropriate, must have been obtained.
Exclusion criteria
✕. The patient had significant non-MPS IIIA related CNS impairment or behavioral disturbances that would confound the scientific integrity or interpretation of study assessments, as determined by the investigator.
✕. The patient had MPS IIIA behavioral-related issues, as determined by the investigator, that would have precluded performance of study neurocognitive and developmental testing procedures
What they're measuring
1
Number of Treatment Emergent Serious Adverse Events (SAE)
Timeframe: Baseline to week 30 (follow-up)
2
Number of Treatment Emergent Adverse Events (TEAE)
Timeframe: Baseline to week 30 (follow-up)
3
Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group
Timeframe: Baseline, Week 26
4
Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group
Timeframe: Baseline, Week 26
5
Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26
✕. The patient was pregnant, breast feeding, or was a female patient of childbearing potential who would not or could not comply with the use of an acceptable method of birth control such as condoms, barrier method, oral contraception, etc.
✕. The patient was blind and/or deaf.
✕. The patient had any known or suspected hypersensitivity to anesthesia or was thought to have an unacceptably high risk for anesthesia due to airway compromise or other conditions.
✕. The patient or the patient's family had a history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns.
✕. The investigator may have chosen to exclude patients who have had complications resulting from prior lumbar punctures.