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Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

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ClinicalTrials.gov Identifier: NCT03582449
Recruitment Status : Recruiting
First Posted : July 11, 2018
Last Update Posted : July 11, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease
Hunter Syndrome

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Study Type : Observational
Estimated Enrollment : 25 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
Actual Study Start Date : January 19, 2018
Estimated Primary Completion Date : February 28, 2021
Estimated Study Completion Date : February 28, 2021


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



Primary Outcome Measures :
  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.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase will be evaluated for this study.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03582449


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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Mexico
Hospital Pediátrico de Sinaloa Recruiting
Culiacán Rosales, México, Mexico, 80200
Principal Investigator: Lyali Margarita Corrales, MD         
IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25) Recruiting
Monterrey, Nuevo León, Mexico, 64180
Principal Investigator: Luz Maria Sanchez, MD         
Hospital del Niño Tabasqueño Recruiting
Villahermosa, Tabasco, Mexico, 86100
Principal Investigator: Eduardo Gorian, MD         
Hospital Centenario Miguel Hidalgo Recruiting
Aguascalientes, Mexico, CP 20000
Contact: Jaime López Valdez, MD    52 449 9946720      
Principal Investigator: Jaime López Valdez, MD         
Hospital Infantil Federico Gomez Recruiting
Ciudad de México, Mexico, 06720
Principal Investigator: Magdalena Cerón Rodriguez, MD         
Hospital Universitario de Colima Recruiting
Colima, Mexico, 28019
Principal Investigator: Araceli Zamora, MD         
Sponsors and Collaborators
Shire
Investigators
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Study Director: Shire Study Physician Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03582449     History of Changes
Other Study ID Numbers: SHP ELA-701
First Posted: July 11, 2018    Key Record Dates
Last Update Posted: July 11, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Shire:
Hunter syndrome (Mucopolysaccharydosis II)

Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases