MedTrace Logo

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2021-06-08

Study results available
· View outcomes & findings →

Summary

Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase). In the absence of this enzyme, there is an accumulation of the glycosaminoglycan, heparan sulfate, resulting in progressive neurodegeneration. Symptoms are usually first noted in the 1st or 2nd year of life, although definitive diagnosis is often delayed, with an average age of diagnosis of 4.5 years. The disease is characterized by developmental delays initially, followed by neurological developmental arrest, then regression. These developmental deficits are typically associated with severe behavioral disturbances. Patients have a significantly reduced lifespan, with few surviving beyond the 2nd or 3rd decade.

The purpose of this study is to evaluate the safety and efficacy of recombinant human heparan-N-sulfatase (rhHNS) in pediatric patients with Early Stage Mucopolysaccharidosis Type III A Disease.

Conditions

  • Sanfilippo Syndrome

Interventions

DRUG

Recombinant human heparan N-sulfatase [rhHNS]

Recombinant human heparan N-sulfatase \[rhHNS\]

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Months
Max Age
48 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-26
Primary Completion
2016-06-01
Completion
2016-06-01

Countries

  • United States
  • Argentina
  • France
  • Germany
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02060526 on ClinicalTrials.gov