NCT01355146

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
127

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Mar 2011

Longer than P75 for all trials

Geographic Reach
2 countries

22 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 15, 2011

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

May 16, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 17, 2011

Completed
6.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2017

Completed
Last Updated

March 17, 2021

Status Verified

March 1, 2021

Enrollment Period

6.6 years

First QC Date

May 16, 2011

Last Update Submit

March 15, 2021

Conditions

Fabry Disease

Keywords

Patients with proven Fabry's Disease

Outcome Measures

Primary Outcomes (1)

  • Patient satisfaction estimated on a 10-ary Likert scale

    comparison of baseline to 12 months value

Secondary Outcomes (1)

  • Number (per infusion) and severity of infusion-related side effects

    baseline compared to 12 months

Study Arms (1)

Fabry's Disease under Replagal

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Patients with proven Fabry's Disease foreseen for home treatment with Agalsidase alfa at German Fabry centers

You may qualify if:

  • 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
  • The patient / be lawful. Representative has consented in writing to participate in this study.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (22)

Landeskrankenhaus Bregenz

Bregenz, A-6900, Austria

Location

LKH-Universitätsklinikum Graz

Graz, A-8036, Austria

Location

Paracelsus Medizinische Privatuniversität Salzburg

Salzburg, A-5020, Austria

Location

Universitätsklinik für Innere Medizin III

Vienna, A-1090, Austria

Location

Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, A-1090, Austria

Location

Charité - Universitätsmedizin Berlin

Berlin, D-10117, Germany

Location

Klinik II für Innere Medizin

Cologne, 50937, Germany

Location

Med. Versorgungszentrum Dialyse-Centrum Cuxhaven

Cuxhaven, D-27476, Germany

Location

Alfried-Krupp-Krankenhaus Rüttenscheid

Essen, D-45131, Germany

Location

MVZ Immunologie am Krankenhaus Sachsenhausen

Frankfurt, 60594, Germany

Location

Zentrum für Kinder und Jugendmedizin

Freiburg im Breisgau, 79106, Germany

Location

Universitätsklinikum Gießen und Marburg

Giessen, 35392, Germany

Location

Facharzt für Allgemeinmedizin

Hagen, D-58089, Germany

Location

Facharzt für Innere Medizin/Kardiologie

Höxter, 37671, Germany

Location

Hans-Berger-Klinik für Neurologie

Jena, 07747, Germany

Location

Universitätsklinikum Mainz, Zentrum für Kinder- und Jugendmedizin

Mainz, 55131, Germany

Location

Private Practice, Dr. Glenn Sommer

Marienberg, D-09496, Germany

Location

KfH Kuratorium für Dialyse und Nierentransplantation e.V.

München, 80804, Germany

Location

Klinikum rechts der Isar

München, D-81675, Germany

Location

Landesklinikum St. Pölten, Neurologie

Pölten, A-3100, Germany

Location

Nephrologisches Zentrum Rendsburg

Rendsburg, 24768, Germany

Location

Universitätsklinikum Ulm

Ulm, D-89081, Germany

Location

Related Links

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 16, 2011

First Posted

May 17, 2011

Study Start

March 15, 2011

Primary Completion

September 30, 2017

Study Completion

September 30, 2017

Last Updated

March 17, 2021

Record last verified: 2021-03

Locations

DE(17)AU(5)