Fabry DiseaseNCT01031173
Treatment Protocol of Replagal for Patients With Fabry Disease
A study testing agalsidase alfa in people with Fabry Disease, run by Shire at 25 sites in United States.
Trial summary
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week. The study will monitor the course of disease in males and females with Fabry disease who are naive to treatment or were previously treated with agalsidase… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fabry Disease
- What is being tested?
- agalsidase alfa
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 25 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of Fabry disease.
- Patient is willing and able to provide written informed consent, and assent if applicable.
- Females of childbearing potential must agree to use a method of birth control throughout the study and for at least 30 days after the final infusion. The method of contraception must be considered adequate and appropriate in the opinion of the investigator.
Who cannot join
- Hypersensitivity to Replagal, the active substance, or any of the excipients.
- The patient is pregnant or breast feeding.
- Concomitant use of agalsidase beta (Fabrazyme).
- Has received treatment with any investigation drug or device within the 30 days prior to study entry.
- Otherwise unsuitable for the study, in the opinion of the Investigator.
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
United States25 sites
Fullerton Genetics Center-Mission, St. Joseph's Hospital
Asheville, North Carolina, 28803
Tidewater Kidney Specialists
Chesapeake, Virginia, 23320
Carilion New River Valley Medical Center
Christiansburg, Virginia, 24073
University of Missouri Healthcare
Columbia, Missouri, 65212
University Research Foundation for Lysosomal Storage Disorders
Coral Springs, Florida, 33065
Baylor University Medical Center
Dallas, Texas, 75246
Emory University School of Medicine
Decatur, Georgia, 30033
Emory University
Decatur, Georgia, 30033
Denver Nephrologists, PC
Denver, Colorado, 80230
Infusion Associates
Grand Rapids, Michigan, 49525
University of Iowa Health Center
Iowa City, Iowa, 52242
University of California San Diego Medical Center
La Jolla, California, 92093-0830
Kaiser Medical Group Southern CA, Regional Metabolic Services
Los Angeles, California, 90027
North Shore Hematology/Oncology
Manhasset, New York, 11030
New York University School of Medicine
New York, New York, 10016
Children's Hospital & Research Center Oakland
Oakland, California, 94609
St. Joseph's Regional Medical Center
Paterson, New Jersey, 07503
Oregon Health and Science University
Portland, Oregon, 97239
UC Davis Children's Hospital
Sacramento, California, 95817
Central Coast Nephrology
Salinas, California, 93901
Baystate Medical Center
Springfield, Massachusetts, 01199
O & O Alpan LLC
Springfield, Virginia, 22152
Stuart Oncology Associates
Stuart, Florida, 34994
The Toledo Hospital
Toledo, Ohio, 43606
AKDHC Tucson Campbell
Tucson, Arizona, 85718
About this study
Official title: An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week. The study will monitor the course of disease in males and females with Fabry disease who are naive to treatment or were previously treated with agalsidase beta (Fabrazyme®).
Read the detailed description
This study will evaluate the safety and efficacy of Replagal in patients with Fabry disease who are either naive to treatment, who were previously treated with agalsidase beta, or who had previously received Replagal.
Patients diagnosed with Fabry disease who have not previously received treatment, who have received agalsidase beta, or who had previously received Replagal will be eligible to enroll in the study and will receive Replagal at a dose of 0.2 mg/kg body weight administered by an IV infusion over 40 minutes every other week.
This study will be conducted in the United States.
Study visits will occur in 3 phases:
- Screening/Baseline Phase: A Screening/Baseline period (Day -30 to Day -1) to determine eligibility and obtain baseline measurements. Patients who have previously received agalsidase beta will be tested for the presence of anti-agalsidase beta antibodies.
- Treatment Phase: A 12-month treatment phase during which all patients will receive Replagal administered IV every other week. Clinical assessments will occur at Months 1, 3, 6, 9, and 12. The study may be extended to continue giving patients access to treatment.
- End-of-Study Phase: An end-of-study contact either as an office visit or follow-up telephone call will occur one month after the last infusion.
- Conditions
- Fabry Disease
- Being tested
- agalsidase alfa
- Keywords
- α galactosidase A, glycosphingolipid storage disorder, agalsidase alfa, enzyme replacement therapy, Replagal, agalsidase beta, Fabrazyme
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
agalsidase alfaBiological
Also called Replagal
0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2009 (estimated)
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
- Expanded access
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
- NCT01031173
- Sponsor's protocol ID
- HGT-REP-059
- Status last verified
- May 2021
- First submitted
- 8 Dec 2009
- Last updated
- 24 May 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.
- ClinicalTrials.gov ID
- NCT01031173
- Acronym / sponsor ID
- HGT-REP-059
- Conditions
- Fabry Disease
- Keywords
- α galactosidase A, glycosphingolipid storage disorder, agalsidase alfa, enzyme replacement therapy, Replagal, agalsidase beta, Fabrazyme
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- First posted
- 14 Dec 2009 (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 study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week. The study will monitor the course of disease in males and females with Fabry disease who are naive to treatment or were previously treated with agalsidase beta (Fabrazyme®).
Detailed description
This study will evaluate the safety and efficacy of Replagal in patients with Fabry disease who are either naive to treatment, who were previously treated with agalsidase beta, or who had previously received Replagal.
Patients diagnosed with Fabry disease who have not previously received treatment, who have received agalsidase beta, or who had previously received Replagal will be eligible to enroll in the study and will receive Replagal at a dose of 0.2 mg/kg body weight administered by an IV infusion over 40 minutes every other week.
This study will be conducted in the United States.
Study visits will occur in 3 phases:
- Screening/Baseline Phase: A Screening/Baseline period (Day -30 to Day -1) to determine eligibility and obtain baseline measurements. Patients who have previously received agalsidase beta will be tested for the presence of anti-agalsidase beta antibodies.
- Treatment Phase: A 12-month treatment phase during which all patients will receive Replagal administered IV every other week. Clinical assessments will occur at Months 1, 3, 6, 9, and 12. The study may be extended to continue giving patients access to treatment.
- End-of-Study Phase: An end-of-study contact either as an office visit or follow-up telephone call will occur one month after the last infusion.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of Fabry disease.
- Patient is willing and able to provide written informed consent, and assent if applicable.
- Females of childbearing potential must agree to use a method of birth control throughout the study and for at least 30 days after the final infusion. The method of contraception must be considered adequate and appropriate in the opinion of the investigator.
Key exclusion criteria (5 listed)
- Hypersensitivity to Replagal, the active substance, or any of the excipients.
- The patient is pregnant or breast feeding.
- Concomitant use of agalsidase beta (Fabrazyme).
- Has received treatment with any investigation drug or device within the 30 days prior to study entry.
- Otherwise unsuitable for the study, in the opinion of the Investigator.
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 |
|---|---|---|
| agalsidase alfaAlso: Replagal0.2 mg/kg body weight, administered by an intravenous infusion over 40 minutes, every other week. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2021.
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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
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing agalsidase alfa in people with Fabry Disease, run by Shire at 25 sites in United States.
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 5 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: status not confirmed, as of the sponsor's last update on 24 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States. 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?
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.
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 24 May 2021.