Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

NCT03582449 | Completed

• 1 other identifier: SHP ELA-701
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Hunter Syndrome

Keywords

Hunter syndrome (Mucopolysaccharydosis II)

Outcome Measures

Primary Outcomes (5)

• 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

Study Arms (1)

Elaprase

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Shire: lead

Study Sites (6)

Hospital Pediátrico de Sinaloa

Culiacán Rosales, México, 80200, Mexico

Location

IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25)

Monterrey, Nuevo León, 64180, Mexico

Location

Hospital del Niño Tabasqueño

Villahermosa, Tabasco, 86100, Mexico

Location

Hospital Centenario Miguel Hidalgo

Aguascalientes, CP 20000, Mexico

Location

Hospital Universitario de Colima

Colima, 28019, Mexico

Location

Hospital Infantil Federico Gomez

Mexico City, 06720, Mexico

Location

Related Links

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

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Shire Study Physician

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2018
• First Posted: July 11, 2018
• Study Start: January 19, 2018
• Primary Completion: May 10, 2020
• Study Completion: May 10, 2020
• Last Updated: September 15, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

ME (6)