Fabry DiseaseNCT05671770
Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)
An observational study of Fabry Disease, run by Sanofi at 1 site in United Arab Emirates.
Trial summary
Primary objective: To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the predictive algorithm (i.e., feasibility assessment eligibility criteria) Secondary objectives… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- How many people
- 15 participants
- Where
- 1 siteUnited Arab Emirates
- Study dates
- 28 Jul 2023to 14 Dec 2023
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with chronic kidney disease (Cohort 1) or hypertrophic cardiomyopathy (Cohort 2), who fulfill the criteria for being at high-risk for FD, per the predictive algorithm, and provide informed consent, will be eligible to participate in this study.
Who may be able to join
- Patients who were identified as high-risk for FD via the predictive algorithm per the feasibility assessment
- Patients who have signed an informed consent form
- Cohort 1 (chronic kidney disease)
- Adult male patient ≤60 years old or adult female patient of any age
- Having chronic kidney disease
- Having proteinuria
Show all 17Show fewer
- Having one or more of the following conditions:
- Neuralgia and neuritis, unspecified
- Disturbances in skin sensation
- Anhidrosis, and
- Hypertrophic cardiomyopathy.
- Cohort 2 (hypertrophic cardiomyopathy)
- Adult male patient ≤50 years old or adult female patient of any age
- Having hypertrophic cardiomyopathy
- Having one or more of the following conditions:
- transient cerebral ischemic attack,
- chronic kidney disease.
Who cannot join
- Patients with an established diagnosis of FD.
- Pregnant subject at the time of the study.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
1 site in 1 country.
United Arab Emirates1 site
Cleveland Clinic AbuDhabi
Abu Dhabi, 11111
About this study
Official title: Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE
Primary objective:
To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the predictive algorithm (i.e., feasibility assessment eligibility criteria)
Secondary objectives:
- To estimate the prevalence of FD among patients at high-risk for FD (i.e., among enrolled patients)
- To characterize the patient profile, overall and in Cohorts 1 and 2
- To describe the most common characteristics among positive FD patients and negative FD patients
Read the detailed description
Patients at high-risk for FD will be consecutively enrolled over approximately 6 months
- Conditions
- Fabry Disease
- Medical subject headings
- Fabry Disease
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 |
|---|---|---|
| Cohort 1 | Patients with chronic kidney disease | |
| Cohort 2 | Patients with hypertrophic cardiomyopathy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and proportion of patients at high-risk for FD in the CCAD EMR database
The prevalence of patients at high-risk for FD in the CCAD EMR database, according to the predictive algorithm, from May 2016 to May 2022, will be estimated. The percentage (and 95% confidence interval [CI]) of patients at high-risk for FD will be calculated, overall and in Cohorts 1 and 2.
Measured: Up to 6 months
Secondary outcomes (3)
Number and proportion of patients with FD (overall and among patients in Cohorts 1 and 2)
The prevalence of patients diagnosed with FD among patients at high-risk for FD (i.e., among enrolled patients) will be estimated. The percentage (and 95% CI) of patients diagnosed with FD will be calculated, overall and in Cohorts 1 and 2.
Measured: Up to 6 months
To describe the following patient characteristics, overall and in Cohorts 1 and 2:
The following patient characteristics will be summarized by descriptive statistics, overall and in Cohorts 1 and 2: demographics, physical examinations, medical history, comorbidities, concomitant medications, clinical symptoms, and FD diagnostic test results including test outcome for FD (positive/negative).
Measured: Up to 6 months
To describe the following patient characteristics, among positive FD patients and negative FD cases:
The following patient characteristics will be summarized by descriptive statistics, among positive FD patients and negative FD patients: demographics, physical examinations, medical history, comorbidities, concomitant medications, and clinical symptoms.
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2023
- Study start28 Jul 2023
- Primary completion14 Dec 2023
- Study completion14 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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
- Retrospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05671770
- Sponsor's protocol ID
- OBS17546
- Status last verified
- Mar 2024
- First submitted
- 20 Dec 2022
- Last updated
- 6 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE
- ClinicalTrials.gov ID
- NCT05671770
- Acronym / sponsor ID
- OBS17546
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 28 Jul 2023 (actual)
- Primary completion
- 14 Dec 2023 (actual)
- Study completion
- 14 Dec 2023 (actual)
- First posted
- 5 Jan 2023 (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.
Primary objective:
To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the predictive algorithm (i.e., feasibility assessment eligibility criteria)
Secondary objectives:
- To estimate the prevalence of FD among patients at high-risk for FD (i.e., among enrolled patients)
- To characterize the patient profile, overall and in Cohorts 1 and 2
- To describe the most common characteristics among positive FD patients and negative FD patients
Detailed description
Patients at high-risk for FD will be consecutively enrolled over approximately 6 months
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | –Patients with chronic kidney disease |
| Cohort 2 | – | –Patients with hypertrophic cardiomyopathy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with chronic kidney disease (Cohort 1) or hypertrophic cardiomyopathy (Cohort 2), who fulfill the criteria for being at high-risk for FD, per the predictive algorithm, and provide informed consent, will be eligible to participate in this study.
Key inclusion criteria (17 listed)
- Patients who were identified as high-risk for FD via the predictive algorithm per the feasibility assessment
- Patients who have signed an informed consent form
- Cohort 1 (chronic kidney disease)
- Adult male patient ≤60 years old or adult female patient of any age
- Having chronic kidney disease
Key exclusion criteria (3 listed)
- Patients with an established diagnosis of FD.
- Pregnant subject at the time of the study.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
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
Number and proportion of patients at high-risk for FD in the CCAD EMR database
Time frame: Up to 6 months
The prevalence of patients at high-risk for FD in the CCAD EMR database, according to the predictive algorithm, from May 2016 to May 2022, will be estimated. The percentage (and 95% confidence interval [CI]) of patients at high-risk for FD will be calculated, overall and in Cohorts 1 and 2.
3 secondary outcomes
Number and proportion of patients with FD (overall and among patients in Cohorts 1 and 2)
Time frame: Up to 6 months
To describe the following patient characteristics, overall and in Cohorts 1 and 2:
Time frame: Up to 6 months
To describe the following patient characteristics, among positive FD patients and negative FD cases:
Time frame: Up to 6 months
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 6 Mar 2024.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences & Operations study director
Sanofi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Sanofi at 1 site in United Arab Emirates.
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 17 inclusion and 3 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 6 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Arab Emirates. 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?
Number and proportion of patients at high-risk for FD in the CCAD EMR database (time frame: Up to 6 months). 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 6 Mar 2024.