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Enzyme Replacement Therapy

Shorter Infusion of Fabrazyme for Fabry Disease (SHORTEN Trial)

Phase 4
Recruiting
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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.
- 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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Drug Has Already Been Approved
No Placebo-Only Group
Pivotal Trial

Summary

This trial will look into how to make Fabrazyme treatments safer and more manageable for those with Fabry disease.

Who is the study for?
This trial is for individuals with Fabry Disease, including those new to enzyme replacement therapy (ERT-naïve) and others previously treated with Fabrazyme. Participants must be between 2-65 years old and meet specific weight criteria. Women who can have children must use effective birth control. People cannot join if they've had infusion reactions to Fabrazyme in their last three treatments.
What is being tested?
The study tests whether increasing the rate of infusing Fabrazyme, a treatment for Fabry Disease, while reducing its volume is safe and tolerable. It aims to make this lifelong treatment less burdensome by shortening the time patients spend receiving it.
What are the potential side effects?
Possible side effects may include allergic reactions or intolerance related to faster infusion rates of Fabrazyme. Pre-medications like acetaminophen, montelukast, diphenhydramine, and dexamethasone are used which could cause drowsiness or other mild symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a woman over 30kg, treated with Fabrazyme for 3+ months without reactions to the last 3 doses.
Select...
I am between 2 and 65 years old and have a confirmed diagnosis of FD.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: agalsidase betaExperimental Treatment5 Interventions
agalsidase beta 1 mg/kg infusion once every other week
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Dexamethasone
2007
Completed Phase 4
~2650
Diphenhydramine
2002
Completed Phase 4
~1170
Montelukast
2008
Completed Phase 4
~15460
Acetaminophen
2017
Completed Phase 4
~2030

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor
2,209 Previous Clinical Trials
4,039,748 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
8 Previous Clinical Trials
4,240 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
876 Previous Clinical Trials
2,021,736 Total Patients Enrolled
~8 spots leftby Oct 2025
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