Fabry DiseasePhase 2NCT01363492
Safety Study of Replagal® Therapy in Children With Fabry Disease
A Phase 2 study testing Replagal (agalsidase alfa) in people with Fabry Disease, run by Shire at 5 sites in United States.
Trial summary
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT). Read more about this study
- Who is it for?
- Ages 7 to 17 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Replagal (agalsidase alfa)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesUnited States
- Study dates
- 12 May 2011to 17 Apr 2013
- Phase
- Phase 2
- 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 7 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients must meet all of the following criteria to be enrolled in this study.
- All patients must be diagnosed with Fabry disease by the following criteria:
- Male Patients: The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alfa-galactosidase A activity measured in serum, leukocytes, or fibroblasts or has a confirmed mutation of the alfa-galactosidase-A gene.
- Female Patients: The patient is a heterozygous female with Fabry disease as confirmed by a mutation of the alfa-galactosidase A gene.
- Note: If the diagnosis of Fabry disease is previously documented in the patient's medical record, screening tests do not need to be repeated.
- The patient is 7 to <18 years of age
Show all 9Show fewer
- The patient is ERT-naïve
- Adequate general health (as determined by the Investigators) to undergo the specified phlebotomy regimen and protocol-related procedures and no safety or medical contraindications for participation
- The minor child must assent to participate in the protocol and the parent(s) or legally authorized representative(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legally authorized representative(s)
Who cannot join
- Patients who meet any of the following criteria will be excluded from the study.
- Patient and/or the patient's parent(s) or legally authorized representative(s) are unable to understand the nature, scope, and possible consequences of the study
- Patient is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.
- Otherwise unsuitable for the study, in the opinion of the Investigator.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Baylor University Medical Center
Dallas, Texas, 75246
Emory Division of Medical Genetics
Decatur, Georgia, 30033
Duke University Medical Center
Durham, North Carolina, 27710
O & O Alpan LLC
Fairfax, Virginia, 22152
University of Utah Hospital
Salt Lake City, Utah, 84132
About this study
Official title: An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
Read the detailed description
In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure.
An agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process.
This study will evaluate the safety of Replagal AF, manufactured using the new bioreactor process at a dose of 0.2 mg/kg infused IV over 40 minutes, every other week (EOW) in children with Fabry disease who are 7 years to less than 18 years of age and who are naive to ERT.
- Conditions
- Fabry Disease
- Being tested
- Replagal (agalsidase alfa)
- Keywords
- agalsidase alfa, 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.
Replagal (agalsidase alfa)Biological
Also called agalsidase alfa
0.2 mg/kg administered over 40 minutes every other week (EOW)
| Study group | Role | What they receive |
|---|---|---|
| Replagal 0.2 mg/kg every other week (EOW) | Experimental | Not described Receives: Biological: Replagal (agalsidase alfa) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Serious Adverse Event (SAE)
Measured: Baseline to week 55
Number of Treatment Emergent Adverse Event (TEAE)
Measured: Baseline to week 55
Development of IgG Anti-Agalsidase Alfa Antibody
Reflects development of Anti-Agalsidase antibodies post baseline
Measured: Baseline to Week 55
Change From Baseline in Heart Rate Variability Parameter SDNN
Measured: Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter rMSSD
Measured: Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter pNN50
Measured: Baseline to week 55
Secondary outcomes (4)
Change From Baseline in LVMI
Measured: Baseline to week 55
Change From Baseline in MFS
Measured: Baseline to week 55
Change From Baseline in Plasma Gb3
Measured: Baseline to week 55
Change From Baseline in Urine Gb3
Measured: Baseline to week 55
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2011 (estimated)
- Study start12 May 2011
- Primary completion17 Apr 2013
- Study completion17 Apr 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 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
- NCT01363492
- Sponsor's protocol ID
- HGT-REP-084
- Status last verified
- May 2021
- First submitted
- 31 Mar 2011
- Last updated
- 9 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naive to enzyme replacement therapy. Drug Des Devel Ther. 2016 May 25;10:1771-81. doi: 10.2147/DDDT.S102761. eCollection 2016.
PubMed 27307708
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT01363492
- Acronym / sponsor ID
- HGT-REP-084
- Conditions
- Fabry Disease
- Keywords
- agalsidase alfa, Replagal, Enzyme Replacement Therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 12 May 2011 (actual)
- Primary completion
- 17 Apr 2013 (actual)
- Study completion
- 17 Apr 2013 (actual)
- First posted
- 1 Jun 2011 (estimated)
- Results
- Posted 20 May 2014
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 assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
Detailed description
In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure.
An agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process.
This study will evaluate the safety of Replagal AF, manufactured using the new bioreactor process at a dose of 0.2 mg/kg infused IV over 40 minutes, every other week (EOW) in children with Fabry disease who are 7 years to less than 18 years of age and who are naive to ERT.
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 every other week (EOW) | Experimental | Biological: Replagal (agalsidase alfa) |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Patients must meet all of the following criteria to be enrolled in this study.
- All patients must be diagnosed with Fabry disease by the following criteria:
- Note: If the diagnosis of Fabry disease is previously documented in the patient's medical record, screening tests do not need to be repeated.
- The patient is 7 to <18 years of age
- The patient is ERT-naïve
Key exclusion criteria (4 listed)
- Patients who meet any of the following criteria will be excluded from the study.
- Patient and/or the patient's parent(s) or legally authorized representative(s) are unable to understand the nature, scope, and possible consequences of the study
- Patient is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.
- 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 |
|---|---|---|
| Replagal (agalsidase alfa)Also: agalsidase alfa0.2 mg/kg administered over 40 minutes every other week (EOW) | Biological | Replagal 0.2 mg/kg every other week (EOW) |
Primary outcome
Number of Serious Adverse Event (SAE)
Time frame: Baseline to week 55
Number of Treatment Emergent Adverse Event (TEAE)
Time frame: Baseline to week 55
Development of IgG Anti-Agalsidase Alfa Antibody
Time frame: Baseline to Week 55
Reflects development of Anti-Agalsidase antibodies post baseline
Change From Baseline in Heart Rate Variability Parameter SDNN
Time frame: Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter rMSSD
Time frame: Baseline to week 55
Change From Baseline in Heart Rate Variability Parameter pNN50
Time frame: Baseline to week 55
4 secondary outcomes
Change From Baseline in LVMI
Time frame: Baseline to week 55
Change From Baseline in MFS
Time frame: Baseline to week 55
Change From Baseline in Plasma Gb3
Time frame: Baseline to week 55
Change From Baseline in Urine Gb3
Time frame: Baseline to week 55
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27307708Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naive to enzyme replacement therapy. Drug Des Devel Ther. 2016 May 25;10:1771-81. doi: 10.2147/DDDT.S102761. eCollection 2016.
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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
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Replagal (agalsidase alfa) in people with Fabry Disease, run by Shire at 5 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 9 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 9 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 5 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 20 May 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?
Number of Serious Adverse Event (SAE) (time frame: Baseline to week 55). The study lists 6 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 NCT01363492, 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 9 Jun 2021.