Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Completed

• Conditions: Hunter Syndrome

• Registration Number: NCT03582449
• Lead Sponsor: Shire

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-01-19
• Study First Submitted: 2018-06-28
• Study First Submitted QC: 2018-06-28
• Study First Posted: 2018-07-11
• Primary Completion: 2020-05-10
• Study Completion: 2020-05-10
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-14
• Last Update Posted: 2023-09-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Weight Over One Year Study Period	Baseline, 12 months	Change in weight over one year study period will be assessed.
Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term	During 12 months of study participation	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.
Incidence of Adverse Events During Administration of Elaprase	During 12 months of study participation	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.
Change From Baseline in Height Over One Year Study Period	Baseline, 12 months	Change in height over one year study period will be assessed.
Incidence of Adverse Events 2 months After Early Termination of Elaprase Treatment.	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.

Trial Locations

Locations (6)

Hospital Pediátrico de Sinaloa; 🇲🇽 Culiacán Rosales, México, Mexico

Hospital Centenario Miguel Hidalgo; 🇲🇽 Aguascalientes, Mexico

IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25); 🇲🇽 Monterrey, Nuevo León, Mexico

Hospital del Niño Tabasqueño; 🇲🇽 Villahermosa, Tabasco, Mexico

Hospital Infantil Federico Gomez; 🇲🇽 Ciudad de México, Mexico

Hospital Universitario de Colima; 🇲🇽 Colima, Mexico