Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03582449

Enrollment

Registered

2018-07-11

Start date

2018-01-19

Completion date

2020-05-10

Last updated

2023-09-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hunter Syndrome

Keywords

Hunter syndrome (Mucopolysaccharydosis II)

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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.
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 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.
Change From Baseline in Height Over One Year Study Period	Baseline, 12 months	Change in height over one year study period will be assessed.
Change From Baseline in Weight Over One Year Study Period	Baseline, 12 months	Change in weight over one year study period will be assessed.

Countries

Mexico

Outcome results