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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
17
HGT-1410 administered according to Patient Group assignment.
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
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug
An AE was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. TEAEs was defined as all AEs from the time of initial IDDD implantation (or first dose if earlier) in either Study NCT02060526 (HGT-SAN-093) or Study NCT02350816 (SHP-610-210) to the data cutoff date (28 Jun 2017), or 30 days after the date of the last dose or 2 weeks after the date of device explant (whichever was later) if early termination occurred. Treatment-emergent AEs were summarized by type (serious, life-threatening), severity (mild, moderate, severe) and degree of relationship to investigational product (Intrathecal Drug Delivery Device (IDDD), device surgical procedure, or intraThecal administration of HGT-1410).
Time frame: From start of study drug administration up to follow-up (Week 276)
Number of Participants With Positive Anti-Recombinant Human Heparan N-Sulfatase (rhHNS) Antibody Status in Serum
Number of participants with positive anti-rhHNS antibody status in serum were reported.
Time frame: Up to 120 weeks
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Cerebro Spinal Fluid (CSF)
No sufficient pharmacokinetic (PK) samples were collected and analyzed due to early termination of the study.
Time frame: Week 0 and 48
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Serum
No sufficient PK samples were collected and analyzed due to early termination of the study.
Time frame: Week 0, 48 and 96
Levels of Glycosaminoglycan (GAG) Concentration in Cerebro Spinal Fluid (CSF)
Levels of GAG concentration in CSF was reported. Last measurable data was presented for respective participant up to their last observed time point.
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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
Time frame: Up to Week 120
Levels of Glycosaminoglycan (GAG) Concentration in Urine
Levels of GAG concentration in Urine were reported. Last measurable data was presented for respective participant up to their last available time point.
Time frame: Up to Week 120
Change From Baseline Vineland Adaptive Behavior Scales Second Edition (VABS-II)
VABS-II measured adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. It was an instrument that supports the diagnosis of intellectual and developmental disabilities in participants. This test measured 5 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite ((a composite of the other four domains). Scoring was 'Usually' = 2, 'Sometimes'/Partially' = 1 or 'Never' = 0. The raw scores was converted to domain standard scores (mean 100, SD 15). Higher scores indicate undesirable behavior. Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Time frame: Baseline, Week 120
Change From Baseline in the Developmental Quotient (DQ) Assessed by Neurocognitive Tests
The development quotient (DQ) was to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range: 0, 100). Higher scores are indicative of decreased development. Neurocognitive tests included Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III), Kaufman Assessment Battery for Children, Second Edition (KABC-II), Vineland Adaptive Behavior Scales, Second Edition (VABS-II). Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Time frame: Baseline, Week 120
Change From Baseline in Total Cortical Grey Matter Volume
The total cortical grey matter volume was assessed by volumetric magnetic resonance imaging (MRI) of the brain. Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Time frame: Baseline, Week 120