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OpenTrials
Completed

NCT Number: NCT03582449

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.

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Key information

Conditions

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

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Pediátrico de Sinaloa, Culiacán Rosales, México, Mexico

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Incidence of Adverse Events During Administration of Elaprase

    Time frame: 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.

  2. Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term

    Time frame: 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.

  3. Incidence of Adverse Events 2 months After Early Termination of Elaprase Treatment.

    Time frame: 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.

  4. Change From Baseline in Height Over One Year Study Period

    Time frame: Baseline, 12 months

    Change in height over one year study period will be assessed.

  5. Change From Baseline in Weight Over One Year Study Period

    Time frame: Baseline, 12 months

    Change in weight over one year study period will be assessed.

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Jul 11, 2018
Registry last updated
Sep 15, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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