Fabry's DiseasePhase 4SHORTENNCT06019728
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
A Phase 4 study testing AGALSIDASE BETA (GZ419828), Acetaminophen and Diphenhydramine in people with Fabry's Disease, run by Sanofi at 5 sites in United States.
Trial summary
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment… Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Fabry's Disease
- What is being tested?
- AGALSIDASE BETA (GZ419828), Acetaminophen and Diphenhydramine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 5 sitesUnited States
- Study dates
- 10 Nov 2023to 25 Oct 2024
- Phase
- Phase 4
- 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 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Women of childbearing potential must use a highly effective method of contraception through the study.
Who cannot join
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Emory University School of Medicine Site Number : 1005
Atlanta, Georgia, 30322-1007
Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002
Fairfax, Virginia, 22030
Infusion Associates Site Number : 1001
Grand Rapids, Michigan, 49525
Metropolitan Hospital Center Site Number : 1004
New York, New York, 10021
UCLA Medical Center Site Number : 1003
Santa Monica, California, 90404
About this study
Official title: A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
Read the detailed description
The total duration will be up to 7 months
- Conditions
- Fabry's Disease
- Being tested
- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast
- 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 BETA (GZ419828)Drug
Also called Fabrazyme
Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
AcetaminophenDrug
Tablet or solution; Oral
DiphenhydramineDrug
Tablet or solution; Oral
DexamethasoneDrug
Tablet or solution; Oral
MontelukastDrug
Tablet or chewable tablet or oral granules; Oral
LoratadineDrug
Tablet or chewable tablet; Oral
CetirizineDrug
Tablet or oral solution; Oral
FexofenadineDrug
Tablet or oral suspension; Oral
| Study group | Role | What they receive |
|---|---|---|
| agalsidase beta | Experimental | agalsidase beta 1 mg/kg infusion once every other week Receives: Drug: AGALSIDASE BETA (GZ419828); Drug: Acetaminophen; Drug: Diphenhydramine; Drug: Dexamethasone; Drug: Montelukast; Drug: Loratadine; Drug: Cetirizine; Drug: Fexofenadine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions
Percent reduction was with respect to the shortest tolerated infusion for the participant. The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second infusion associated reaction (IAR). The pre-study infusion duration was derived as the average of the duration of 3 most recent infusions the participant received prior to participating to this study. Percent reduction of infusion duration was calculated as: 100 x (average of pre-study infusion duration [minutes] - actual shortest infusion duration tolerated [minutes]) / average of pre-study infusion duration (minutes).
Measured: Up to 16 weeks
Shortest Actual Tolerated Infusion Duration
The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second IAR. IARs were events recorded as adverse event of special interest (AESI) and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.
Measured: Up to 16 weeks
Secondary outcomes (6)
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction
Number of participants who achieved the shortest planned duration of infusion time without experiencing any IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.
Measured: Up to 16 weeks
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction
Number of participants who achieved the shortest planned duration of infusion time without experiencing a second IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.
Measured: Up to 16 weeks
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction
Number of participants who tolerated the shortest planned duration of infusion time without experiencing any IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun. Participants with an initial planned target duration <90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration <90 minutes.
Measured: Up to 16 weeks
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction
Number of participants who tolerated the shortest planned duration of infusion time without experiencing a second IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun. Participants with an initial planned target duration <90 minutes were considered a responder if they tolerated at least 1 infusion during the study treatment period with actual duration <90 minutes.
Measured: Up to 16 weeks
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction
Number of participants who tolerated each planned infusion duration without experiencing any IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun. The planned duration of infusion time was 120 minutes, 90 minutes, 60 minutes, 40 minutes, and 20 minutes.
Measured: Up to 16 weeks
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction
Number of participants who tolerated each planned infusion duration without experiencing a second IAR is reported. ARs were events recorded as AESI and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun. The planned duration of infusion time was 120 minutes, 90 minutes, 60 minutes, 40 minutes, and 20 minutes.
Measured: Up to 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2023
- Study start10 Nov 2023
- Primary completion25 Oct 2024
- Study completion25 Oct 2024
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.
- Other trials for Fabry's DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06019728
- Sponsor's protocol ID
- LPS17726
- Other IDs
- U1111-1287-8570
- Acronym
- SHORTEN
- Status last verified
- Sep 2025
- First submitted
- 25 Aug 2023
- Last updated
- 21 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion
- ClinicalTrials.gov ID
- NCT06019728
- Acronym / sponsor ID
- SHORTEN / LPS17726
- Conditions
- Fabry's Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 10 Nov 2023 (actual)
- Primary completion
- 25 Oct 2024 (actual)
- Study completion
- 25 Oct 2024 (actual)
- First posted
- 31 Aug 2023 (actual)
- Results
- Posted 21 Oct 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
Detailed description
The total duration will be up to 7 months
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| agalsidase beta | Experimental | Drug: AGALSIDASE BETA (GZ419828), Drug: Acetaminophen, Drug: Diphenhydramine, Drug: Dexamethasone, Drug: Montelukast, Drug: Loratadine, Drug: Cetirizine, Drug: Fexofenadineagalsidase beta 1 mg/kg infusion once every other week |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
Key exclusion criteria (5 listed)
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
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 BETA (GZ419828)Also: FabrazymePharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion | Drug | agalsidase beta |
| AcetaminophenTablet or solution; Oral | Drug | agalsidase beta |
| DiphenhydramineTablet or solution; Oral | Drug | agalsidase beta |
| DexamethasoneTablet or solution; Oral | Drug | agalsidase beta |
| MontelukastTablet or chewable tablet or oral granules; Oral | Drug | agalsidase beta |
| LoratadineTablet or chewable tablet; Oral | Drug | agalsidase beta |
| CetirizineTablet or oral solution; Oral | Drug | agalsidase beta |
| FexofenadineTablet or oral suspension; Oral | Drug | agalsidase beta |
Primary outcome
Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions
Time frame: Up to 16 weeks
Percent reduction was with respect to the shortest tolerated infusion for the participant. The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second infusion associated reaction (IAR). The pre-study infusion duration was derived as the average of the duration of 3 most recent infusions the participant received prior to participating to this study. Percent reduction of infusion duration was calculated as: 100 x (average of pre-study infusion duration [minutes] - actual shortest infusion duration tolerated [minutes]) / average of pre-study infusion duration (minutes).
Shortest Actual Tolerated Infusion Duration
Time frame: Up to 16 weeks
The shortest infusion duration tolerated was the actual shortest duration (highest infusion rate) of a completed infusion tolerated by the participant without experiencing a second IAR. IARs were events recorded as adverse event of special interest (AESI) and were defined as AEs the Investigator considered related to Fabrazyme® infusion, that occurred on same day as infusion and only after infusion had begun.
6 secondary outcomes
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction
Time frame: Up to 16 weeks
Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction
Time frame: Up to 16 weeks
Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction
Time frame: Up to 16 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- LPS17726 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences & Operations study director
Sanofi
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing AGALSIDASE BETA (GZ419828), Acetaminophen and Diphenhydramine in people with Fabry's Disease, run by Sanofi 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 6 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: finished, as of the sponsor's last update on 21 Oct 2025. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 21 Oct 2025). 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?
Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions (time frame: Up to 16 weeks). 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?
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 21 Oct 2025.