Fabry DiseaseNCT01355146
Home Therapy With Replagal in Fabry Disease
An observational study of Fabry Disease, run by Shire at 22 sites in 2 countries.
Trial summary
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Morbus Fabry disease receiving their Enzyme Replacement Therapy with Replagal (Agalsidase alfa) at home compared to receiving the infusions at the clinic or at doctor's practice. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Fabry Disease
- How many people
- 127 participants
- Where
- 22 sitesin 2 countries
- Study dates
- 15 Mar 2011to 30 Sep 2017
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers
Who may be able to join
- Male or female patient with a confirmed diagnosis of Fabry disease
- Age> 4 years
- Patient is under Replagal since at least 12 weeks ® therapy
- The patient is compliant, the previous Replagal ® infusions were performed approximately every 2 weeks in the center close to home or at the hospital / general practitioner at
- Patient has been well tolerated Replagal ® therapy, and there were in the last 12 weeks before inclusion in this study no significant infusion reactions to
- The patient has been selected before inclusion in this study for a home infusion therapy and has consented to (or their legal representative)
- The patient / be lawful. Representative has consented in writing to participate in this study.
Who cannot join
- Patient/legal representative does not give consent to participation in this study
- Patient/legal representative declines Replagal® home therapy
- The patient is participating in a clinical trial with a medicinal product
Trial locations and contacts
22 sites in 2 countries.
22 sites in 2 countries
Germany17 sites
Charité - Universitätsmedizin Berlin
Berlin, D-10117
Klinik II für Innere Medizin
Cologne, 50937
Med. Versorgungszentrum Dialyse-Centrum Cuxhaven
Cuxhaven, D-27476
Alfried-Krupp-Krankenhaus Rüttenscheid
Essen, D-45131
MVZ Immunologie am Krankenhaus Sachsenhausen
Frankfurt, 60594
Zentrum für Kinder und Jugendmedizin
Freiburg im Breisgau, 79106
Universitätsklinikum Gießen und Marburg
Giessen, 35392
Facharzt für Allgemeinmedizin
Hagen, D-58089
Facharzt für Innere Medizin/Kardiologie
Höxter, 37671
Hans-Berger-Klinik für Neurologie
Jena, 07747
Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin
Mainz, 55131
Private Practice, Dr. Glenn Sommer
Marienberg, D-09496
KfH Kuratorium für Dialyse und Nierentransplantation e.V.
München, 80804
Klinikum rechts der Isar
München, D-81675
Landesklinikum St. Pölten, Neurologie
Pölten, A-3100
Nephrologisches Zentrum Rendsburg
Rendsburg, 24768
Universitätsklinikum Ulm
Ulm, D-89081
Austria5 sites
Landeskrankenhaus Bregenz
Bregenz, A-6900
LKH-Universitätsklinikum Graz
Graz, A-8036
Paracelsus Medizinische Privatuniversität Salzburg
Salzburg, A-5020
Universitätsklinik für Innere Medizin III
Vienna, A-1090
Universitätsklinik für Kinder- und Jugendheilkunde
Vienna, A-1090
About this study
Official title: Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Morbus Fabry disease receiving their Enzyme Replacement Therapy with Replagal (Agalsidase alfa) at home compared to receiving the infusions at the clinic or at doctor's practice.
- Conditions
- Fabry Disease
- Keywords
- Patients with proven Fabry's 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 |
|---|---|---|
| Fabry's Disease under Replagal | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient satisfaction estimated on a 10-ary Likert scale
Measured: comparison of baseline to 12 months value
Secondary outcomes (1)
Number (per infusion) and severity of infusion-related side effects
Measured: baseline compared to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2011 (estimated)
- Study start15 Mar 2011
- Primary completion30 Sep 2017
- Study completion30 Sep 2017
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.
- 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
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01355146
- Sponsor's protocol ID
- Shire/CS02
- Status last verified
- Mar 2021
- First submitted
- 16 May 2011
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non Interventional Study of Replagal® Home Therapy in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT01355146
- Acronym / sponsor ID
- Shire/CS02
- Conditions
- Fabry Disease
- Keywords
- Patients with proven Fabry's Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 127 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 15 Mar 2011 (actual)
- Primary completion
- 30 Sep 2017 (actual)
- Study completion
- 30 Sep 2017 (actual)
- First posted
- 17 May 2011 (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.
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Morbus Fabry disease receiving their Enzyme Replacement Therapy with Replagal (Agalsidase alfa) at home compared to receiving the infusions at the clinic or at doctor's practice.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fabry's Disease under Replagal | – | – |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers
Key inclusion criteria (7 listed)
- Male or female patient with a confirmed diagnosis of Fabry disease
- Age> 4 years
- Patient is under Replagal since at least 12 weeks ® therapy
- The patient is compliant, the previous Replagal ® infusions were performed approximately every 2 weeks in the center close to home or at the hospital / general practitioner at
- Patient has been well tolerated Replagal ® therapy, and there were in the last 12 weeks before inclusion in this study no significant infusion reactions to
Key exclusion criteria (3 listed)
- Patient/legal representative does not give consent to participation in this study
- Patient/legal representative declines Replagal® home therapy
- The patient is participating in a clinical trial with a medicinal product
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
Patient satisfaction estimated on a 10-ary Likert scale
Time frame: comparison of baseline to 12 months value
1 secondary outcome
Number (per infusion) and severity of infusion-related side effects
Time frame: baseline compared to 12 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 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
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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.
Study Director study director
Takeda
22 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Shire at 22 sites in 2 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 7 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 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in Germany, Austria. 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?
Patient satisfaction estimated on a 10-ary Likert scale (time frame: comparison of baseline to 12 months value). 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 17 Mar 2021; AzTrials last refreshed its copy on 6 Nov 2026.