Fabry's DiseasePREFABNCT02582294
Fabry's Disease and Pregnancy (PREFAB)
An observational study of Fabry's Disease and Pregnancy, run by Groupe Hospitalier Diaconesses Croix Saint-Simon at 1 site in France.
Trial summary
Fabry's disease is a progressive systemic disease X-linked which combines neurological (Fabry's pain crises), dermatologic (angiokeratomas), renal (renal failure), cardiovascular (hypertrophic cardiomyopathy, valvular disease, conduction disorder, coronary heart disease) and cerebral vascular (stroke) symptoms . Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Fabry's Disease, Pregnancy
- What is being tested?
- Questionnaire
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2015to 1 Jun 2018
- 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 to 45 years
- Women only
- No healthy volunteers
- Adult
Study population: Fabry's disease patients having had at least one childbirth
Who may be able to join
- Fabry's disease patients, aged 18 years or more, having had at least one childbirth
Who cannot join
- Patient under a legal protection procedure
- Patient denying to participate to the study
- Lack of affiliation to a social security system
Trial locations and contacts
1 site in 1 country.
France1 site
Groupe hospitalier Diaconesses Croix Saint Simon
Paris, Île-de-France Region, 75020
About this study
Official title: Description of Obstetrical Care, Mainly Anesthesic, in Patients With Fabry's Disease
Fabry's disease is a progressive systemic disease X-linked which combines neurological (Fabry's pain crises), dermatologic (angiokeratomas), renal (renal failure), cardiovascular (hypertrophic cardiomyopathy, valvular disease, conduction disorder, coronary heart disease) and cerebral vascular (stroke) symptoms .
It is a glycosphingolipid metabolism disorder due to deficient or absent activity of the alpha-galactosidase A, causing accumulation of globotriaosylceramide in the lysosomes.
Read more
The incidence is estimated being 1/40 000. Some patients suffering from Fabry's disease today are of childbearing age and their multidisciplinary care (by neurologists, obstetricians and anesthetists) raises several questions.
About the anesthetic, the question of epidural block is debated in patients with neurological diseases and recommendations are not unequivocal.
Indeed one of the problems of the management in those conditions is the potential worsening of the disease because of the anesthetic procedure. In addition, the possibility of an antiplatelet and / or an anticoagulant treatment in these patients may also contre-indicate an epidural block.
The rate of epidural block achieved in patients with Fabry's disease is not currently known. Moreover, only sparse data on pregnancy outcomes in these patients are reported.
- Conditions
- Fabry's Disease, Pregnancy
- Being tested
- Questionnaire
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnaireOther
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Fabry's disease patients having had an epidural block in childbirth.
Measured: The duration of the interview for this study with women with Fabry disease's will be one hour
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2015 (estimated)
- Study startOct 2015
- Primary completion1 Jun 2018
- Study completion1 Jun 2018
Why it stopped: Difficulty in patient recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry's DiseaseStudies 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
- Other
- Time perspective
- Retrospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Groupe Hospitalier Diaconesses Croix Saint-Simon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02582294
- Sponsor's protocol ID
- OLE_2015_4
- Acronym
- PREFAB
- Status last verified
- Feb 2019
- First submitted
- 15 Oct 2015
- Last updated
- 15 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Description of Obstetrical Care, Mainly Anesthesic, in Patients With Fabry's Disease
- ClinicalTrials.gov ID
- NCT02582294
- Acronym / sponsor ID
- PREFAB / OLE_2015_4
- Conditions
- Fabry's Disease, Pregnancy
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Difficulty in patient recruitment
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Groupe Hospitalier Diaconesses Croix Saint-SimonUniversity, hospital or other
- Study start
- Oct 2015 (actual)
- Primary completion
- 1 Jun 2018 (actual)
- Study completion
- 1 Jun 2018 (actual)
- First posted
- 21 Oct 2015 (estimated)
- 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.
Fabry's disease is a progressive systemic disease X-linked which combines neurological (Fabry's pain crises), dermatologic (angiokeratomas), renal (renal failure), cardiovascular (hypertrophic cardiomyopathy, valvular disease, conduction disorder, coronary heart disease) and cerebral vascular (stroke) symptoms .
It is a glycosphingolipid metabolism disorder due to deficient or absent activity of the alpha-galactosidase A, causing accumulation of globotriaosylceramide in the lysosomes.
The incidence is estimated being 1/40 000. Some patients suffering from Fabry's disease today are of childbearing age and their multidisciplinary care (by neurologists, obstetricians and anesthetists) raises several questions.
About the anesthetic, the question of epidural block is debated in patients with neurological diseases and recommendations are not unequivocal.
Indeed one of the problems of the management in those conditions is the potential worsening of the disease because of the anesthetic procedure. In addition, the possibility of an antiplatelet and / or an anticoagulant treatment in these patients may also contre-indicate an epidural block.
The rate of epidural block achieved in patients with Fabry's disease is not currently known. Moreover, only sparse data on pregnancy outcomes in these patients are reported.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Fabry's disease patients having had at least one childbirth
Key inclusion criteria (1 listed)
- Fabry's disease patients, aged 18 years or more, having had at least one childbirth
Key exclusion criteria (3 listed)
- Patient under a legal protection procedure
- Patient denying to participate to the study
- Lack of affiliation to a social security system
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Questionnaire | Other | – |
Primary outcome
Proportion of Fabry's disease patients having had an epidural block in childbirth.
Time frame: The duration of the interview for this study with women with Fabry disease's will be one hour
Results and safety
The study was stopped early (reason given: Difficulty in patient recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2019.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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's Disease and Pregnancy, run by Groupe Hospitalier Diaconesses Croix Saint-Simon at 1 site in France.
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 1 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: stopped early, as of the sponsor's last update on 15 Feb 2019. 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 France. 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 was stopped early (reason given: Difficulty in patient recruitment). Results are not available in the source record.
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?
Proportion of Fabry's disease patients having had an epidural block in childbirth. (time frame: The duration of the interview for this study with women with Fabry disease's will be one hour). 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 Feb 2019.