Clinical trial NCT03582449: Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
Official title: Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hunter Syndrome
Keywords: Hunter syndrome (Mucopolysaccharydosis II)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2018-07-11
Enrollment: 5
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase will be evaluated for this study.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Adverse Events During Administration of Elaprase
timeFrame: During 12 months of study participation
measure: Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term
timeFrame: During 12 months of study participation
measure: Incidence of Adverse Events 2 months After Early Termination of Elaprase Treatment.
timeFrame: 2 months after early termination of Elaprase treatment
measure: Change From Baseline in Height Over One Year Study Period
timeFrame: Baseline, 12 months
measure: Change From Baseline in Weight Over One Year Study Period
timeFrame: Baseline, 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Culiacán Rosales
country: Mexico
facility: Hospital Pediátrico de Sinaloa
state: México
city: Monterrey
country: Mexico
facility: IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25)
state: Nuevo León
city: Villahermosa
country: Mexico
facility: Hospital del Niño Tabasqueño
state: Tabasco
city: Aguascalientes
country: Mexico
facility: Hospital Centenario Miguel Hidalgo
city: Ciudad de México
country: Mexico
facility: Hospital Infantil Federico Gomez
city: Colima
country: Mexico
facility: Hospital Universitario de Colima
Study officials:
affiliation: Shire
name: Shire Study Physician
role: STUDY_DIRECTOR
References: