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

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ClinicalTrials.gov Identifier: NCT02060526
Recruitment Status : Completed
First Posted : February 12, 2014
Results First Posted : March 3, 2017
Last Update Posted : May 15, 2017
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Sanfilippo Syndrome
Intervention: Drug: Recombinant human heparan N-sulfatase [rhHNS]

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 24 participants were screened, of them 21 participants were enrolled in the study.

Reporting Groups
  Description
No HGT-1410 Participants received no treatment (HGT-1410).
HGT-1410 45 mg Q2W Participants received HGT-1410 45 milligram (mg) intrathecally once every two weeks (Q2W) using surgically implanted intrathecal drug delivery device (IDDD) or lumbar puncture (LP) for 48 weeks.
HGT-1410 45 mg Q4W Participants received HGT-1410 45 mg intrathecally once every four weeks (Q4W) using surgically implanted IDDD or LP for 48 weeks.

Participant Flow:   Overall Study
    No HGT-1410   HGT-1410 45 mg Q2W   HGT-1410 45 mg Q4W
STARTED   7   7   7 
COMPLETED   7   7   7 
NOT COMPLETED   0   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat (ITT) population included all randomized participants.

Reporting Groups
  Description
No HGT-1410 Participants received no treatment (HGT-1410).
HGT-1410 45 mg Q2W Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
HGT-1410 45 mg Q4W Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
Total Total of all reporting groups

Baseline Measures
   No HGT-1410   HGT-1410 45 mg Q2W   HGT-1410 45 mg Q4W   Total 
Overall Participants Analyzed 
[Units: Participants]
 7   7   7   21 
Age 
[Units: Months]
Mean (Standard Deviation)
 32.42  (9.548)   29.64  (9.989)   33.53  (9.336)   31.87  (9.287) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
       
Female      3  42.9%      5  71.4%      4  57.1%      12  57.1% 
Male      4  57.1%      2  28.6%      3  42.9%      9  42.9% 


  Outcome Measures

1.  Primary:   Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III)   [ Time Frame: Baseline (Week 0) up to Week 48 ]

2.  Secondary:   Number of Participants With Serious Adverse Events (SAE)   [ Time Frame: Baseline (Week 0) up to Week 52 ]

3.  Secondary:   Number of Participants With Treatment Emergent Adverse Events (TEAEs)   [ Time Frame: Baseline (Week 0) up to Week 52 ]

4.  Secondary:   Number of Participants With Positive Anti-recombinant Human Heparan-N-Sulfatase (rhHNS) Antibody in Serum at Week 48   [ Time Frame: Baseline (Week 0) up to Week 48 ]

5.  Secondary:   Change From Baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) Development Quotient (DQ) Score at Week 48   [ Time Frame: Baseline (Week 0), Week 48 ]

6.  Secondary:   Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48   [ Time Frame: Baseline (Week 0), Week 48 ]

7.  Secondary:   Change From Baseline in Total Cortical Grey Matter Volume at Week 48   [ Time Frame: Baseline (Week 0), Week 48 ]

8.  Secondary:   Change From Baseline in Concentration of Glycosaminoglycan (GAG) in Cerebrospinal Fluid (CSF) at Week 48   [ Time Frame: Baseline (Week 0), Week 48 ]

9.  Secondary:   Change From Baseline in Concentration of GAG in Urine at Week 48   [ Time Frame: Baseline (Week 0), Week 48 ]

10.  Secondary:   Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)   [ Time Frame: Pre-dose, 4, 48 hours on Week 0 and Week 48 ]

11.  Secondary:   Maximum Observed Drug Concentration (Cmax) of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Serum   [ Time Frame: Predose, 0.5 h, 1 h, 2 h, 4 h, 8 h, 12 h, 24 h, and 48 h post-dose on Week 0 and Week 48 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Physician
Organization: Shire
phone: ClinicalTransparency@shire.com



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02060526     History of Changes
Other Study ID Numbers: HGT-SAN-093
2013-003450-24 ( EudraCT Number )
First Submitted: February 10, 2014
First Posted: February 12, 2014
Results First Submitted: January 11, 2017
Results First Posted: March 3, 2017
Last Update Posted: May 15, 2017