Hunter Syndrome
Conditions
Brief summary
This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in a clinical study with an investigational agent.
Interventions
BEHAVIORALNeurobehavioral testing
If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to assess his neurodevelopmental status using a standardized battery of neurobehavioral testing.
OTHERVisual and auditory assessments
If the patient is found to be eligible after completion of a telephone interview, he will undergo further testing to evaluate his visual and auditory function.
Sponsors
Shire
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
32 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
1. The patient is male and is ≥3 and \<18 years of age 2. The patient is currently receiving weekly IV infusions of Elaprase. 3. The patient, patient's parent(s), or legally authorized guardian(s) has voluntarily signed an Institutional Review Board / Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and subject's assent, as relevant, must be obtained.
Exclusion criteria
1. The patient has a CNS shunt. 2. The patient has received a hematopoietic stem cell transplant. 3. The patient is currently enrolled in a clinical trial. 4. The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent | 1 month | 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. |
| Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS) | 1 month | 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. |
Countries
United Kingdom, United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| All Patients All patients who met the inclusion criteria and consented to participate in the study. | 33 |
| Total | 33 |
Baseline characteristics
| Characteristic | All Patients |
|---|---|
| Age, Categorical <=18 years | 33 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 6.42 years STANDARD_DEVIATION 2.841 |
| Region of Enrollment United Kingdom | 12 Participants |
| Region of Enrollment United States | 21 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 33 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 33 |
| serious Total, serious adverse events | 0 / 33 |
Outcome results
Primary
Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent
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.
Time frame: 1 month
Population: All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| All Patients | Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent | 17 participants |
Primary
Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)
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.
Time frame: 1 month
Population: All enrolled patients, defined as patients who met the inclusion criteria and consented to participate in the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| All Patients | 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 |