An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Dis… (NCT02350816) | Clinical Trial Compass
TerminatedPhase 2
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
Stopped: Completion of follow-up period
United States, France, Germany17 participantsStarted 2015-04-08
Plain-language 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.
Who can participate
Age range12 Months – 48 Months
SexALL
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Inclusion criteria
✓. Patient has completed through at least the Week 48 visit of Study HGT-SAN-093
✓. 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
✕. 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\*
✕. 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
✕. The patient has a known hypersensitivity to any of the components of HGT-1410
✕. 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
✕. 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
✕
What they're measuring
1
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug
Timeframe: From start of study drug administration up to follow-up (Week 276)
2
Number of Participants With Positive Anti-Recombinant Human Heparan N-Sulfatase (rhHNS) Antibody Status in Serum
Timeframe: Up to 120 weeks
3
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Cerebro Spinal Fluid (CSF)
Timeframe: Week 0 and 48
4
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Serum
Timeframe: Week 0, 48 and 96
5
Levels of Glycosaminoglycan (GAG) Concentration in Cerebro Spinal Fluid (CSF)
Timeframe: Up to Week 120
6
Levels of Glycosaminoglycan (GAG) Concentration in Urine