Trial Outcomes & Findings for Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase® (NCT NCT00937794)

Results Overview

Standardized tests were used to identify patients who were receiving treatment with Elaprase, had cognitive impairment, and were suitable to participate in the follow-on clinical study (HGT-HIT-045). Assessments included: 1-Cognition: The Differential Ability Scale, Second Edition (DAS-II) or the Bayley Scales of Infant Development, Third Edition (BSID-III); 2-Adaptive Behavior: The Scale of Independent Behavior-Revised (SIB-R); 3-Executive Function: The Behavior Rating Inventory of Executive Function-Preschool version (BRIEF-P) for children or the Behavior Rating Inventory of Executive Function (BRIEF) for children less than or ≥6 years of age, respectively; 4-Motor: The Peabody Developmental Motor Scales-2 (PDMS-2) or the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2) for children less than or ≥6 years of age, respectively.

Recruitment status

COMPLETED

Target enrollment

33 participants

Primary outcome timeframe

1 month

Results posted on

2021-06-14

Participant Flow

This screening study evaluated patients for disease and neurodevelopmental status to determine their potential eligibility for investigational study HGT-HIT-045 (NCT00920647), and informed decisions regarding study design, rate of enrollment, and estimated dates of completion for subsequent investigational studies in Hunter syndrome.

Participant milestones

Participant milestones
Measure	All Patients All patients who met the inclusion criteria and consented to participate in the study.
Overall Study STARTED	33
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=33 Participants All patients who met the inclusion criteria and consented to participate in the study.
Age, Categorical <=18 years	33 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	6.42 years STANDARD_DEVIATION 2.841 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	33 Participants n=5 Participants
Region of Enrollment United States	21 Participants n=5 Participants
Region of Enrollment United Kingdom	12 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 month

Population: All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.

Standardized tests were used to identify patients who were receiving treatment with Elaprase, had cognitive impairment, and were suitable to participate in the follow-on clinical study (HGT-HIT-045). Assessments included: 1-Cognition: The Differential Ability Scale, Second Edition (DAS-II) or the Bayley Scales of Infant Development, Third Edition (BSID-III); 2-Adaptive Behavior: The Scale of Independent Behavior-Revised (SIB-R); 3-Executive Function: The Behavior Rating Inventory of Executive Function-Preschool version (BRIEF-P) for children or the Behavior Rating Inventory of Executive Function (BRIEF) for children less than or ≥6 years of age, respectively; 4-Motor: The Peabody Developmental Motor Scales-2 (PDMS-2) or the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2) for children less than or ≥6 years of age, respectively.

Outcome measures

Outcome measures
Measure	All Patients n=33 Participants All patients who met the inclusion criteria and consented to participate in the study.
Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent	17 participants

PRIMARY outcome

Timeframe: 1 month

Population: All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.

The GCA sub-scale of the DAS, Second Edition (DAS-II) was used to obtain a general measure of cognitive ability.The maximum score is 120, with a higher score indicating greater cognitive ability. A score of 100 is considered an average score.

Outcome measures

Outcome measures
Measure	All Patients n=33 Participants All patients who met the inclusion criteria and consented to participate in the study.
Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)	5 participants

Adverse Events

All Patients

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	All Patients n=33 participants at risk All patients who met the inclusion criteria and consented to participate in the study.
Gastrointestinal disorders Vomiting	3.0% 1/33
General disorders Pyrexia	3.0% 1/33
Investigations Blood pressure systolic increased	3.0% 1/33

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.
Publication restrictions are in place

Restriction type: OTHER