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Trial record 17 of 31 for:    "mucopolysaccharidosis type III"

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

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ClinicalTrials.gov Identifier: NCT02350816
Recruitment Status : Enrolling by invitation
First Posted : January 30, 2015
Last Update Posted : March 9, 2017
Sponsor:
Information provided by (Responsible Party):
Shire

Brief 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.

Condition or disease Intervention/treatment Phase
Sanfilippo Syndrome Mucopolysaccharidosis (MPS) Drug: HGT-1410 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : April 2015
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : October 2021


Arm Intervention/treatment
Active Comparator: HGT-1410 Q2W in Study HGT-SAN-093 randomized to HGT-1410 Q2W

Patients in Group 1 will continue HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 50, with a cumulative treatment period of up to 42 months (168 weeks) . HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).

HGT-SAN-093 = NCT02060526

Drug: HGT-1410
HGT-1410 administered according to Patient Group assignment.
Other Name: Recombinant Human Heparan N Sulfatase

Active Comparator: HGT-1410 Q4W in Study HGT-SAN-093 randomized to HGT-1410 Q4W
Patients in Group 2 will continue HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 52, with a cumulative treatment period of up to 42 months (168 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).
Drug: HGT-1410
HGT-1410 administered according to Patient Group assignment.
Other Name: Recombinant Human Heparan N Sulfatase

Active Comparator: no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q2W
Patients in Group 3A will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).
Drug: HGT-1410
HGT-1410 administered according to Patient Group assignment.
Other Name: Recombinant Human Heparan N Sulfatase

Active Comparator: no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q4W
Patients in Group 3B will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).
Drug: HGT-1410
HGT-1410 administered according to Patient Group assignment.
Other Name: Recombinant Human Heparan N Sulfatase




Primary Outcome Measures :
  1. Number of participants with serious adverse events (SAE) [ Time Frame: Baseline to week 124 ]
    Total Serious Adverse Events

  2. Number of participants with treatment emergent adverse events (TEAE) [ Time Frame: Baseline to week 124 ]
    Total Treatment-Emergent Adverse Events

  3. Anti-rhHNS Antibody Status in serum [ Time Frame: Baseline to week 120 ]
    AUC-Area Under Curve

  4. The concentration of rhHNS in CSF [ Time Frame: Baseline to week 120 ]
    AUC- Area Under Curve

  5. The concentration of rhHNS in serum [ Time Frame: Baseline to week 120 ]
    AUC- Area Under Curve

  6. The change from baseline in concentration of GAG in CSF [ Time Frame: Baseline to week 120 ]
    AUC- Area Under Curve

  7. The change from baseline in concentration of GAG in urine [ Time Frame: Baseline to week 120 ]
    AUC- Area Under Curve


Secondary Outcome Measures :
  1. The change from baseline in adaptive behavioral function, assessed by neurocognitive tests. [ Time Frame: Baseline to week 120 ]
    questionnaire

  2. The change from baseline in the DQ assessed by neurocognitive tests [ Time Frame: Baseline to week 120 ]
    questionnaire

  3. The change from baseline in total cortical grey matter volume [ Time Frame: Baseline to week 120 ]
    AUC- Area Under Curve



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months to 48 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
    4. The patient has a known or suspected local or general infection
    5. The patient is at risk of abnormal bleeding due to a medical condition or therapy
    6. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    7. The patient has a functioning CSF shunt device
    8. The patient has shown an intolerance to an implanted device
  7. The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator

    • All treated patients in Study HGT-SAN-093 will have their cognitive development assessed at the Week 48 Visit in Study HGT-SAN-093. If a decline from Baseline of 20 points or less in the BSID-III DQ score is observed, then the patient may proceed into the Study SHP-610-201 without further evaluation. If a decline from Baseline of more than 20 points in DQ score is observed, then an individual evaluation by the Investigator will occur to determine if the patient is a treatment failure. This individual evaluation will take into account the DQ scores, VABS-II score, physical status, and any other information available for that patient at that time. If the Investigator deems the patient to be a treatment failure, then the patient may not enter the Study SHP-610-201

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02350816


Locations
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27514
France
Chu Bicetre, Le Kremlin-Bicêtre
Paris, France, 94270
Germany
Universitätsklinikum Hamburg Eppendorf
Hamburg, Germany, 20246
Italy
Azienda Socio Sanitaria Territoriale - Asst di Monza
Monza, Italy, 20900
Netherlands
Academisch Medisch Centrum Amsterdam
Amsterdam, Netherlands, 22660
Spain
Hospital Universitario Vall D'hebron - Ppds
Barcelona, Spain, 08035
United Kingdom
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Shire
Investigators
Study Director: Study Physician Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02350816     History of Changes
Other Study ID Numbers: SHP610-201
2014-003960-20 ( EudraCT Number )
First Posted: January 30, 2015    Key Record Dates
Last Update Posted: March 9, 2017
Last Verified: March 2017

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases