Fabry Diseasesifap2NCT00413595
Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation
An observational study of Fabry Disease and Cerebrovascular Accident, run by CENTOGENE GmbH Rostock at 20 sites in 7 countries.
Trial summary
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Fabry Disease, Cerebrovascular Accident
- How many people
- 100 participants
- Where
- 20 sitesin 7 countries
- Study dates
- Jul 2007to 1 Dec 2019
- 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 55 years
- All sexes
- No healthy volunteers
- Adult
Study population: Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack and genetic diagnosis (a-galactosidase defect) of Fabry disease.
Who may be able to join
- Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack
- Genetic diagnosis (a-galactosidase defect)of Fabry disease
- Written informed consent from patient
Who cannot join
- No proven Fabry disease
- Participating in an other clinical trial with any investigational new drug or medical device
- Contraindication to any of the diagnostic procedures like e.g. MRI investigation
- Patient has been pretreated with Enzyme Replacement Therapy at the date of informed consent of sifap2
Trial locations and contacts
20 sites in 7 countries.
20 sites in 7 countries
Germany14 sites
Department of Neurology, Klinikum Hohe Warte
Bayreuth, 95445
Charite Campus Benjamin Franklin, Dept. of Neurology
Berlin, D-12200
Department of Neurology, Allgemeines Krankenhaus Celle
Celle, 29223
Department of Neurology, Klinikum Chemnitz gGmbH
Chemnitz, 09131
Department of Neurology, Universitaetsklinikum Carl Gustav Carus
Dresden, 01307
Heinrich-Heine-University Duesseldorf, Dept. of Neurology
Düsseldorf, D-40225
University of Giessen-Marburg Dept. of Neurology
Giessen, D-35385
Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246
Department of Neurology, Universitaetsklinikum Jena
Jena, 07740
Department of Neurology, Universitaetsklinikum Leipzig
Leipzig, 04103
Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH
Mühlhausen, 99974
Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology
München, D-81377
Department of Neurology, University Tuebingen
Tübingen, 72076
University of Ulm, Department of Neurology
Ulm, D-89081
Croatia1 site
Department of Neurology, University Hospital Sestre Milosrdnice
Zagreb, 10000
France1 site
Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires
Lyon, F-69003
Georgia1 site
Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university
Tbilisi, 0179
Poland1 site
Institute of Psychiatry and Neurology, Dept. of Neurology
Warsaw, 02-957
Portugal1 site
Centro Hospitalar de Lisboa Central, Servico de Neurologia
Lisbon, 1150-199
About this study
Official title: Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation in Young Patients With Fabry Disease: An Epidemiological, International, Multicenter Prognosis Study
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. These fat molecules build up to dangerous levels, which start to damage the body, because they accumulate e.g. in the walls of the blood vessels. This accumulation in the blood vessels of the whole body may cause life-threatening malfunctions in the brain, inducing a stroke.
The purpose of this study is to investigate the stroke rehabilitation of Fabry patients during different therapeutic standard approaches for stroke and for Fabry disease (if any). During this study, stroke patients with Fabry disease will be monitored in greater detail to determine whether the differences in treatment are significant for patient recovery and on what they depend.
Read the detailed description
In a group of young stroke patients with diagnosed Fabry disease the stroke rehabilitation will be investigated during different prophylactic therapeutic approaches. In this study the investigator will not be given any instructions on stroke and Fabry therapy.
All patients with any etiology of stroke and a diagnosed Fabry disease submitted to the stroke unit of the participating centres which commit to work with the EUSI (European Stroke Initiative) recommendations for stroke management and diagnosis will be included into the study.
- Conditions
- Fabry Disease, Cerebrovascular Accident
- Being tested
- No intervention
- Keywords
- Cerebrovascular Accident, Cerebrovascular Accident, Acute, Fabry Disease, Fabry's Disease, Anderson-Fabry Disease, CVA (Cerebrovascular Accident), Stroke, acute, Cerebral Stroke
- Medical subject headings
- Fabry Disease, Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.
| Study group | Role | What they receive |
|---|---|---|
| Observation | Adult patients (18 - 55 years of age) with an acute cerebrovascular event of any etiology and the genetic diagnosis (a-galactosidase defect) of Fabry disease Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches
Measured: 54 months study duration
Secondary outcomes (8)
Quality of Life measured with the SF-36
Measured: 54 months study duration
Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis
Measured: 54 months study duration
Beck Depression Inventory II (BDI II)
Measured: 54 months study duration
Brief Pain Inventory (BPI)
Measured: 54 months study duration
Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)
Measured: 54 months study period
Habi test (only in Austrian and German centers)
Measured: 54 months study duration
Trail Making Test (TMT)
Measured: 54 months study duration
Functional neurological deficits measured by the Mini Mental State Examination (MMSE)
Measured: 54 months study duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2006 (estimated)
- Study startJul 2007
- Primary completion1 Dec 2019
- Study completion1 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CENTOGENE GmbH Rostock
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shire Human Genetic Therapies, Inc.
- Responsible party
- Sponsor
- Investigators
- Arndt Rolfs, Prof., MD, Principal investigator, University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00413595
- Sponsor's protocol ID
- II PV04/2006
- Acronym
- sifap2
- Status last verified
- Apr 2020
- First submitted
- 19 Dec 2006
- Last updated
- 12 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Rolfs A, Bottcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Lohr M, Harzer K, Strauss U, Pahnke J, Grossmann A, Benecke R. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet. 2005 Nov 19;366(9499):1794-6. doi: 10.1016/S0140-6736(05)67635-0.
PubMed 16298216
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation in Young Patients With Fabry Disease: An Epidemiological, International, Multicenter Prognosis Study
- ClinicalTrials.gov ID
- NCT00413595
- Acronym / sponsor ID
- sifap2 / II PV04/2006
- Conditions
- Fabry Disease, Cerebrovascular Accident
- Keywords
- Cerebrovascular Accident, Cerebrovascular Accident, Acute, Fabry Disease, Fabry's Disease, Anderson-Fabry Disease, CVA (Cerebrovascular Accident), Stroke, acute, Cerebral Stroke
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- CENTOGENE GmbH RostockIndustry (pharma, biotech, devices)
- Collaborators
- Shire Human Genetic Therapies, Inc.
- Study start
- Jul 2007 (actual)
- Primary completion
- 1 Dec 2019 (actual)
- Study completion
- 1 Dec 2019 (actual)
- First posted
- 20 Dec 2006 (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.
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. These fat molecules build up to dangerous levels, which start to damage the body, because they accumulate e.g. in the walls of the blood vessels. This accumulation in the blood vessels of the whole body may cause life-threatening malfunctions in the brain, inducing a stroke.
The purpose of this study is to investigate the stroke rehabilitation of Fabry patients during different therapeutic standard approaches for stroke and for Fabry disease (if any). During this study, stroke patients with Fabry disease will be monitored in greater detail to determine whether the differences in treatment are significant for patient recovery and on what they depend.
Detailed description
In a group of young stroke patients with diagnosed Fabry disease the stroke rehabilitation will be investigated during different prophylactic therapeutic approaches. In this study the investigator will not be given any instructions on stroke and Fabry therapy.
All patients with any etiology of stroke and a diagnosed Fabry disease submitted to the stroke unit of the participating centres which commit to work with the EUSI (European Stroke Initiative) recommendations for stroke management and diagnosis will be included into the study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observation | – | Other: No interventionAdult patients (18 - 55 years of age) with an acute cerebrovascular event of any etiology and the genetic diagnosis (a-galactosidase defect) of Fabry disease |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack and genetic diagnosis (a-galactosidase defect) of Fabry disease.
Key inclusion criteria (3 listed)
- Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack
- Genetic diagnosis (a-galactosidase defect)of Fabry disease
- Written informed consent from patient
Key exclusion criteria (4 listed)
- No proven Fabry disease
- Participating in an other clinical trial with any investigational new drug or medical device
- Contraindication to any of the diagnostic procedures like e.g. MRI investigation
- Patient has been pretreated with Enzyme Replacement Therapy at the date of informed consent of sifap2
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 |
|---|---|---|
| No interventionObservational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done. | Other | Observation |
Primary outcome
Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches
Time frame: 54 months study duration
8 secondary outcomes
Quality of Life measured with the SF-36
Time frame: 54 months study duration
Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis
Time frame: 54 months study duration
Beck Depression Inventory II (BDI II)
Time frame: 54 months study duration
Brief Pain Inventory (BPI)
Time frame: 54 months study duration
Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)
Time frame: 54 months study period
Habi test (only in Austrian and German centers)
Time frame: 54 months study duration
Trail Making Test (TMT)
Time frame: 54 months study duration
Functional neurological deficits measured by the Mini Mental State Examination (MMSE)
Time frame: 54 months study duration
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 12 Apr 2021.
- Background: PubMed 16298216Rolfs A, Bottcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Lohr M, Harzer K, Strauss U, Pahnke J, Grossmann A, Benecke R. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet. 2005 Nov 19;366(9499):1794-6. doi: 10.1016/S0140-6736(05)67635-0.
- Individual participant dataSharing planned: Undecided
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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.
Arndt Rolfs, Prof., MD principal investigator
University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration
20 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease and Cerebrovascular Accident, run by CENTOGENE GmbH Rostock at 20 sites in 7 countries.
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 3 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 12 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Germany, Austria, Croatia, France, Georgia and 2 more countries. 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?
Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches (time frame: 54 months study duration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00413595, which also lists 1 linked publication.
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 12 Apr 2021.