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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 baseline to month 4

Awards & highlights

SHORTEN 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.Check my eligibility

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.See study design

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.

SHORTEN Trial 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.

SHORTEN Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 4

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 4

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 4 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Reduction of infusion duration from initial 120 minutes

Reduction of infusion duration from pretrial average of recent 3 infusions

Shortest infusion duration each participant tolerates

Secondary outcome measures

Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR

Number of participants achieving infusion duration without experiencing any IAR

Number of participants achieving the shortest planned duration of infusion time

+2 more

SHORTEN 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

Montelukast

2008

Completed Phase 4

~15460

Acetaminophen

2017

Completed Phase 4

~2030

Dexamethasone

2007

Completed Phase 4

~2590

Diphenhydramine

2002

Completed Phase 4

~1170

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor

2,164 Previous Clinical Trials

3,514,962 Total Patients Enrolled

13 Trials studying Fabry Disease

2,241 Patients Enrolled for Fabry Disease

Clinical Sciences & OperationsStudy DirectorSanofi

862 Previous Clinical Trials

2,019,820 Total Patients Enrolled

6 Trials studying Fabry Disease

170 Patients Enrolled for Fabry Disease

Clinical Sciences & OperationsStudy DirectorSanofi

8 Previous Clinical Trials

4,912 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are geriatric patients being included in this research?

"This research project will accept patients older than 2 years and younger than 65 years of age."

Answered by AI

Are there any slots available for participants to join the current trial?

"Confirmed. The trial's data on clinicaltrials.gov confirms that it is actively recruiting participants, which began on November 10th 2023 and was last refreshed by the 29th of November 2023. This study requires 18 patients to be enrolled at two medical centres."

Answered by AI

What is the current participation rate in this medical experiment?

"Affirmative. Clinicaltrials.gov showcases this clinical trial as actively recruiting participants, with the original posting dated November 10th 2023. This research venture is searching for 18 individuals from 2 medical centres to collaborate in their study."

Answered by AI

What are the potential adverse effects of agalsidase beta on patients?

"Agalsidase beta is deemed to have reached a level of safety that merits a score of 3, given the fact this treatment has attained FDA approval as part of Phase 4 clinical trials."

Answered by AI

Is there an opportunity for me to participate in this experiment?

"This clinical trial requires 18 individuals, aged 2-65, with Fabry Disease. Additionally, in order to be eligible participants must have been receiving infusions of Fabrazyme for at least 3 months without Incidence Adverse Reactions (IARs) during the last 3 treatments and their body weight must meet pre-determined criteria depending on gender and prior treatment history. For women of childbearing potential an effective contraception method throughout the course of the study is also mandatory."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

2023. "A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06019728.

All > Fabry Disease