Hunter Outcome Survey (HOS)

study id #: NCT03292887

condition: Hunter Syndrome

status: Recruiting

purpose:

The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.

results: https://clinicaltrials.gov/ct2/show/results/NCT03292887

last updated: March 05, 2022

start date: August 1, 2005

estimated completion: September 1, 2022

last updated: June 18, 2021

size / enrollment: 2000

primary outcomes:

• Number of Participants With Infusion-related Reactions (IRRs)
  An Infusion-related reaction (IRR) is an adverse event (AE) that occurs during or within 24 hours of an infusion and with evidence of a causal relationship with Elaprase.
• Baseline to year 17
  Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
  An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or adverse drug reaction (ADR) that meets one or more of the following criteria/outcomes is classified as serious whether considered to be related to the pharmaceutical product or not: death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.
• Baseline to year 17
  Number of Participants With Positive Antibody Response
  Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies.
• Baseline to year 17
  Change in Urinary Glycosaminoglycan (GAG) Levels
  Change in urinary GAG levels from the start of ERT is reported.
• Baseline to year 17
  Change in Height
  Change in height from the start of ERT will be reported.
• Baseline to year 17
  Change in Weight
  Change in weight from the start of ERT will be reported.
• Baseline to year 17
  Change in Head Circumference and Corresponding Calculated Z-scores
  Change in head circumference with the corresponding Z-scores from the start of ERT will be reported.
• Baseline to year 17
  Change in Distance Walked in the 6-minute Walk Test
  Change in distance walked in 6-minute walk test from the start of ERT is reported.
• Baseline to year 17
  Left Ventricular Mass Index (LVMI)
  Change in LVMI will be assessed as calculated by echocardiography.
• Baseline to year 17
  Change in Forced Expiratory Volume in 1 Second (FEV1)
  Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1).
• Baseline to year 17
  Change in Forced Vital Capacity (FVC)
  Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).
• Baseline to year 17
  Change in Liver and Spleen Size
  Change in liver and spleen size as estimated by palpation will be reported.
• Baseline to year 17
  Prevalence of Cardiac and Pulmonary-related Hospitalizations
  Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
• Baseline to year 17
  Age at the Time of Death
  Age at the time of death will be reported.
• Baseline to year 17
  Cause of Death
  Causes of death will be reported
• Baseline to year 17
  

secondary outcomes:

• Natural History of Untreated Participants With Hunter Syndrome
  Baseline to year 17
• Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
  Baseline to year 17
• Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
  Baseline to year 17
• Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
  Baseline to year 17
• Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
  Baseline to year 17
• Dosing Regimens of Elaprase for Reason for Missed Infusions.
  Baseline to year 17
• Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)
  Baseline to year 17

inclusion criteria:

Inclusion Criteria:

Diagnosis of Hunter syndrome (biochemically and/or genetically)

Signed and dated written informed consent, as per either a or b below:

Prospective Participants: Signed and dated written informed consent from the participant or, for participants aged less than (<) 18 years (<16 years in Scotland), parent and/or participant's legally authorized representative (LAR), and assent of the minor where applicable.

informed consent must be obtained from LARs for cognitively impaired participants, where applicable.

OR

Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .

exclusion criteria: Criteria:

Participants enrolled in an interventional clinical trial are not eligible. Participants may re-enroll once they have completed or withdrawn from the other clinical study.
Participants receiving treatment for Hunter syndrome with an ERT product other than Elaprase are not eligible. Participants may enroll or re-enroll once they have stopped treatment with another ERT.

sponsor: Shire

contacts: Takeda Contact, +1 866 842 5335, ClinicalTransparency@takeda.com

investigators: Study Director,Shire

trial center locations: United States

• United States, Massachusetts
  Shire
  Central Contact, ClinicalTransparency@shire.com