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An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. | Clinical Trials | Clareo Health

TERMINATEDPHASE2

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Mucopolysaccharidosis Type IIIA Disease

NCT02350816•Shire

View on ClinicalTrials.gov

Summary

This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy of the study drug.

Eligibility

Age

1 - 4 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Patients must meet all of the following criteria to be considered eligible for enrollment:
•1. Patient has completed through at least the Week 48 visit of Study HGT-SAN-093
•2. The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board- (IRB-)/ Independent Ethics Committee- (IEC-) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained

Exclusion Criteria

•Patients will be excluded from the study if any of the following criteria are met:
•1. The patient, if randomized to treatment in Study HGT-SAN-093, has experienced a decline of more than 20 points in the BSID-III cognitive DQ score between Baseline and the Week 48 visit in Study HGT-SAN-093, AND, upon individual evaluation by the Investigator, has been deemed a treatment failure\*
•2. The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with HGT-1410, including but not limited to clinically relevant intracranial hypertension, severe infusion-related reactions after treatment with HGT-1410, uncontrollable seizure disorder
•3. The patient has a known hypersensitivity to any of the components of HGT-1410
•4. The patient is enrolled in another clinical study, other than HGT-SAN-093, that involves clinical investigations or use of any investigational product (drug or \[intrathecal/spinal\] device) within 30 days prior to study enrollment or at any time during the study
•5. The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions
•6. The patient has a condition that is contraindicated as described in the SOPH-A-PORT® Mini S IDDD Instructions for Use, including:
•1. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT ® Mini S device
•2. The patient's body size is too small to support the size of the SOPH-A-PORT ® Mini S Access Port, as judged by the Investigator
•3. The patient's drug therapy requires substances known to be incompatible with the materials of construction
+7 more criteria

Locations (8)

University of Minnesota

Minneapolis, Minnesota, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Chu Bicetre, Le Kremlin-Bicêtre

Paris, France

Universitätsklinikum Hamburg Eppendorf

Hamburg, Germany

Azienda Socio Sanitaria Territoriale - Asst di Monza

Monza, Italy

Academisch Medisch Centrum Amsterdam

Amsterdam, Netherlands

Hospital Universitario Vall D'hebron - Ppds

Barcelona, Spain

Great Ormond Street Hospital

London, United Kingdom

Key Dates

Start Date

April 8, 2015

Primary Completion Date

April 12, 2019

Completion Date

April 12, 2019

Last Updated

June 11, 2021

Enrollment

ACTUAL participants

Conditions

Sanfilippo SyndromeMucopolysaccharidosis (MPS)

Interventions

HGT-1410

DRUG

Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041

Sanfilippo SyndromeMucopolysaccharidosis III

View study

TERMINATEDPHASE1/PHASE2

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734

MPS IIIASanfilippo Syndrome+2 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 11, 2021Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Inclusion Criteria

Exclusion Criteria

Study of Cannabidiol in Sanfilippo Syndrome

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB