Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

The purpose of this non-interventional, observational study which is conducted in Mexico is to evaluate the safety profile of elaprase (idulsurfase) in participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase.

Study Overview

• Status: Completed
• Conditions: Hunter Syndrome

• Study Type: Observational
• Enrollment (Actual): 5

Contacts and Locations

Study Locations

• Mexico
  • Aguascalientes, Mexico, CP 20000
    • Hospital Centenario Miguel Hidalgo
  • Ciudad de México, Mexico, 06720
    • Hospital Infantil Federico Gomez
  • Colima, Mexico, 28019
    • Hospital Universitario de Colima
  • México
    • Culiacán Rosales, México, Mexico, 80200
      • Hospital Pediatrico de Sinaloa
  • Nuevo León
    • Monterrey, Nuevo León, Mexico, 64180
      • IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25)
  • Tabasco
    • Villahermosa, Tabasco, Mexico, 86100
      • Hospital del Niño Tabasqueño

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase will be evaluated for this study.

Description

Inclusion Criteria:

• Participants of both genders
• Participants to whom idursulfase (Elaprase) has been prescribed
• Participants with Hunter syndrome
• Participants accepting to participate
• Participants signing the informed consent and privacy notice for handling personal information in: the program Contigo Shire (training, diagnosis, family trees) and the intensive pharmacovigilance study for Elaprase. If the participant is unable to provide legal consent (example: under legal age), the participant's legal guardian must provide consent

Exclusion Criteria:

• Participants withdrawing their consent to continue participating in the non-intervention intensive pharmacovigilance study for Elaprase
• Known hypersensitivity or presence of any contraindication to Elaprase that cannot be controlled by pre-medication
• A physical condition that would not permit intravenous administration of enzyme replacement therapy (ERT)
• Participants enrolled in Shire sponsored Elaprase Registry or any other Shire sponsored Elaprase study

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Elaprase; Participants with Hunter syndrome (Mucopolysaccharydosis II) being treated with Elaprase will be evaluated for this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events During Administration of Elaprase	An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events during administration of elaprase will be reported.	During 12 months of study participation
Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events according to the system organ class and MedDRA preferred term will be reported.	During 12 months of study participation
Incidence of Adverse Events 2 months After Early Termination of Elaprase Treatment.	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that do not necessarily have a causal relationship with this treatment. Incidence of adverse events 2 months after early termination of Elaprase treatment will be reported.	2 months after early termination of Elaprase treatment
Change From Baseline in Height Over One Year Study Period	Change in height over one year study period will be assessed.	Baseline, 12 months
Change From Baseline in Weight Over One Year Study Period	Change in weight over one year study period will be assessed.	Baseline, 12 months

Collaborators and Investigators

• Sponsor: Shire
• Investigators: Study Director: Shire Study Physician, Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): January 19, 2018
• Primary Completion (Actual): May 10, 2020
• Study Completion (Actual): May 10, 2020

Study Registration Dates

• First Submitted: June 28, 2018
• First Submitted That Met QC Criteria: June 28, 2018
• First Posted (Actual): July 11, 2018

Study Record Updates

• Last Update Posted (Actual): September 15, 2023
• Last Update Submitted That Met QC Criteria: September 14, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Hunter syndrome (Mucopolysaccharydosis II)
• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Mucopolysaccharidosis II
• Other Study ID Numbers: SHP ELA-701

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual participants could be re-identified (due to the limited number of study participants)
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No