Fabry DiseasePhase 3NCT01124643
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
A Phase 3 study testing Replagal in people with Fabry Disease, run by Shire at 9 sites in 7 countries.
Trial summary
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Replagal
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 9 sitesin 7 countries
- Study dates
- 13 Apr 2010to 8 Jul 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Complete all study requirements and assessments for Study TKT028 less than 30 days (+/- 7 days) prior to the first dose in this extension protocol
- Voluntarily signed an Institutional Review Board/Independent Ethics Committee- approved informed consent form after all relevant aspects of the study have been explained and discussed.
- Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028
- Female participants of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have a negative pregnancy test at the time of study entry and as required throughout participation in study
Who cannot join
- Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry.
- Is unable to comply with the protocol, (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator
- Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV) antibody, or human immunodeficiency virus (HIV) antibody
- Is pregnant or lactating
- Is morbidly obese, defined as body mass index (BMI) >39 kg/m2
- Has any safety or medical issues, as assessed by the Investigator, that contraindicate participation in the study (eg, has experienced an adverse reaction to treatment with Replagal or has a known hypersensitivity to any of the components of Replagal
Trial locations and contacts
9 sites in 7 countries.
9 sites in 7 countries
United States2 sites
O&O Alpan LLC
Fairfax, Virginia, 22030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Poland2 sites
Szpital Uniwersytecki w Krakowie
Krakow, 31-066
Instytut Kardiologii, I Klinika Choroby Wiencowej
Warsaw
United Kingdom1 site
Salford Royal NHS Foundation Trust
Salford, England, M6 8HD
About this study
Official title: An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
Read the detailed description
HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.
- Conditions
- Fabry Disease
- Being tested
- Replagal
- Keywords
- Agalsidase alfa, Alpha-Galactosidase, Replagal, Enzyme Replacement Therapy
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReplagalBiological
Also called algalsidase alfa, alpha-Galactosidase, DRX005B
0.2 mg/kg administered intravenously [IV] every other week
| Study group | Role | What they receive |
|---|---|---|
| Replagal 0.2 mg/kg EOW | Experimental | Intravenous, 0.2mg/kg EOW Receives: Biological: Replagal |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Measured: Baseline to 12 months
Safety Evaluations
Measured: Baseline to 12 months
Secondary outcomes (7)
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Measured: Baseline to 12 months
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Measured: Baseline to 12 months
Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
The MLHF-Q contains 21 questions with answers ranging from 0 (no) to 5 (very much). The final score ( 0 to 105) is the sum of the points for the 21 questions. A higher score indicates a worse quality of life.
Measured: Baseline to 12 months
Change From Baseline in New York Heart Association (NYHA) Functional Class
Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea. Class II: Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III: Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased.
Measured: Baseline to 12 months
Change From Baseline in Plasma Gb3
Measured: Baseline to 12 months
Change From Baseline in eGFR
Measured: Baseline to 12 months
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Measured: Baseline 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 2010 (estimated)
- Study start13 Apr 2010
- Primary completion8 Jul 2013
- Study completion8 Jul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 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
- NCT01124643
- Sponsor's protocol ID
- HGT-REP-060
- Other IDs
- 2009-015985-75, 2009
- Status last verified
- May 2021
- First submitted
- 23 Apr 2010
- Last updated
- 8 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.
PubMed 26185417
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT01124643
- Acronym / sponsor ID
- HGT-REP-060
- Conditions
- Fabry Disease
- Keywords
- Agalsidase alfa, Alpha-Galactosidase, Replagal, Enzyme Replacement Therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 13 Apr 2010 (actual)
- Primary completion
- 8 Jul 2013 (actual)
- Study completion
- 8 Jul 2013 (actual)
- First posted
- 17 May 2010 (estimated)
- Results
- Posted 22 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
Detailed description
HGT-REP-060 was designed to provide participants who participated in TKT028 an additional 52 weeks of treatment with Replagal at the standard dose of 0.2 milligram per kilogram (mg/kg) every other week (EOW) to assess the effect of continued Replagal therapy on long-term safety and clinical outcomes for this participant population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Replagal 0.2 mg/kg EOW | Experimental | Biological: ReplagalIntravenous, 0.2mg/kg EOW |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Complete all study requirements and assessments for Study TKT028 less than 30 days (+/- 7 days) prior to the first dose in this extension protocol
- Voluntarily signed an Institutional Review Board/Independent Ethics Committee- approved informed consent form after all relevant aspects of the study have been explained and discussed.
- Has received and tolerated at least 80% of the total planned Replagal infusions in Study TKT028
- Female participants of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have a negative pregnancy test at the time of study entry and as required throughout participation in study
Key exclusion criteria (6 listed)
- Has received treatment with any investigational drug (other than Replagal) or device within 30 days prior to study entry.
- Is unable to comply with the protocol, (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator
- Has a positive test for hepatitis B surface antigen (HBsAg), hepatitis C(HCV) antibody, or human immunodeficiency virus (HIV) antibody
- Is pregnant or lactating
- Is morbidly obese, defined as body mass index (BMI) >39 kg/m2
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 |
|---|---|---|
| ReplagalAlso: algalsidase alfa, alpha-Galactosidase, DRX005B0.2 mg/kg administered intravenously [IV] every other week | Biological | Replagal 0.2 mg/kg EOW |
Primary outcome
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI)
Time frame: Baseline to 12 months
Safety Evaluations
Time frame: Baseline to 12 months
7 secondary outcomes
Change From Baseline in Maximal Oxygen Consumption (VO2max) at Peak Exercise
Time frame: Baseline to 12 months
Change From Baseline in Distance Walked in 6- Minute Walk Test (6MWT)
Time frame: Baseline to 12 months
Change From Baseline in the Minnesota Living With Heart Failure Questionnaire (MLHF- Q)
Time frame: Baseline to 12 months
Change From Baseline in New York Heart Association (NYHA) Functional Class
Time frame: Baseline to 12 months
Change From Baseline in Plasma Gb3
Time frame: Baseline to 12 months
Change From Baseline in eGFR
Time frame: Baseline to 12 months
Change From Baseline in Albumin/Creatinine (A/Cr) Ratio
Time frame: Baseline to 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 26185417Golan L, Goker-Alpan O, Holida M, Kantola I, Klopotowski M, Kuusisto J, Linhart A, Musial J, Nicholls K, Gonzalez-Rodriguez D, Sharma R, Vujkovac B, Chang P, Wijatyk A. Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease. Drug Des Devel Ther. 2015 Jul 8;9:3435-44. doi: 10.2147/DDDT.S80928. eCollection 2015.
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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
9 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Replagal in people with Fabry Disease, run by Shire at 9 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 4 inclusion and 6 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 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Poland, Australia, Czechia, Finland 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Left Ventricular Mass Indexed to Height (LVMI) (time frame: Baseline to 12 months). The study lists 2 primary outcomes in all. 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 NCT01124643, 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 8 Jun 2021.