Hunter SyndromeNCT03582449
Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
An observational study of Hunter Syndrome, run by Shire at 6 sites in Mexico.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hunter Syndrome
- How many people
- 5 participants
- Where
- 6 sitesMexico
- Study dates
- 19 Jan 2018to 10 May 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase will be evaluated for this study.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Mexico6 sites
Hospital Centenario Miguel Hidalgo
Aguascalientes, CP 20000
Hospital Universitario de Colima
Colima, 28019
Hospital Pediátrico de Sinaloa
Culiacán Rosales, México, 80200
Hospital Infantil Federico Gomez
Mexico City, 06720
IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25)
Monterrey, Nuevo León, 64180
Hospital del Niño Tabasqueño
Villahermosa, Tabasco, 86100
About this study
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.
- Conditions
- Hunter Syndrome
- Keywords
- Hunter syndrome (Mucopolysaccharydosis II)
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Elaprase | Participants with Hunter syndrome (Mucopolysaccharydosis II) being treated with Elaprase will be evaluated for this study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events During Administration of Elaprase
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.
Measured: During 12 months of study participation
Incidence of Adverse Events According to the System Organ Class and MedDRA Preferred Term
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.
Measured: During 12 months of study participation
Incidence of Adverse Events 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.
Measured: 2 months after early termination of Elaprase treatment
Change From Baseline in Height Over One Year Study Period
Change in height over one year study period will be assessed.
Measured: Baseline, 12 months
Change From Baseline in Weight Over One Year Study Period
Change in weight over one year study period will be assessed.
Measured: Baseline, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2018
- Study start19 Jan 2018
- Primary completion10 May 2020
- Study completion10 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hunter SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shire Study Physician, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03582449
- Sponsor's protocol ID
- SHP ELA-701
- Status last verified
- Sep 2023
- First submitted
- 28 Jun 2018
- Last updated
- 15 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual participants could be re-identified (due to the limited number of study participants)
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
- ClinicalTrials.gov ID
- NCT03582449
- Acronym / sponsor ID
- SHP ELA-701
- Conditions
- Hunter Syndrome
- Keywords
- Hunter syndrome (Mucopolysaccharydosis II)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 19 Jan 2018 (actual)
- Primary completion
- 10 May 2020 (actual)
- Study completion
- 10 May 2020 (actual)
- First posted
- 11 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elaprase | – | –Participants with Hunter syndrome (Mucopolysaccharydosis II) being treated with Elaprase will be evaluated for this study. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with hunter syndrome (mucopolysaccharydosis II) being treated with elaprase will be evaluated for this study.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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.
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.
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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2023.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual participants could be re-identified (due to the limited number of study participants)
Related trials for Hunter Syndrome
Enrolling by invitationPhase 2/3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Mucopolysaccharidosis II
Active, not recruitingPhase 3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
Mucopolysaccharidosis II
Active, not recruitingPhase 2/3
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Mucopolysaccharidosis Type II, Metabolic Diseases
Active, not recruitingPhase 2/3
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Mucopolysaccharidosis II
Active, not recruitingPhase 2
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Mucopolysaccharidosis II
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shire Study Physician study director
Shire
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hunter Syndrome, run by Shire at 6 sites in Mexico.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Mexico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events During Administration of Elaprase (time frame: During 12 months of study participation). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2023.