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Gene Therapy

ASP2016 + Prednisolone for Friedreich Ataxia

Phase 1
Waitlist Available
Research Sponsored by Astellas Gene Therapies
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has a body mass index range of 17.0 to 30.0 kg/m2
Participant has both a clinical diagnosis of Friedreich ataxia (FA) and a documented history of genetic diagnosis of FA with either a guanine-adenine-adenine (GAA) trinucleotide repeat (TNR) expansion in intron 1 of both gene for frataxin (FXN) alleles or a GAA TNR expansion of intron 1 of one FXN allele and a pathogenic variant in the other FXN allele
Must not have
Participant has significant fibrosis on CMRI, defined as late gadolinium enhancement of > 15% left ventricular myocardial mass
Participant has a contraindication to endomyocardial biopsy or cardiac catheterization
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to month 60
Awards & highlights

Summary

"This trial is testing a new treatment called ASP2016 in adults with Friedreich Ataxia who have heart problems. ASP2016 is given as a slow injection into a vein, along with pred

Who is the study for?
Adults with Friedreich Ataxia and associated heart disease can join this trial. They must be able to tolerate the treatment and agree to overnight clinic stays, regular visits, or home nurse visits. The study excludes those who might have complications from gene therapy or cannot undergo necessary tests like biopsies.
What is being tested?
The trial is testing ASP2016, a new gene therapy given as an infusion for treating heart problems in Friedreich Ataxia patients. It's being tested alongside prednisolone tablets to check safety and tolerance at different doses.
What are the potential side effects?
Possible side effects include reactions to the infusion of ASP2016, immune system responses despite taking prednisolone, discomfort from frequent testing procedures such as biopsies and scans, and general risks associated with invasive cardiac procedures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My BMI is between 17.0 and 30.0.
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I have been diagnosed with Friedreich ataxia both clinically and genetically.
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My heart's pumping ability is moderately reduced.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart scan shows significant scarring.
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I cannot undergo certain heart tissue tests due to health risks.
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My FA symptoms started after I turned 25.
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I have high levels of specific antibodies against AAV8.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to month 60
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to month 60 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants with Serious Adverse Events (SAEs)
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Number of participants with electrocardiogram (ECG) abnormalities and/or AEs
+2 more
Secondary study objectives
Change from baseline of TAb to frataxin
Change from baseline of frataxin protein level in cardiac tissue
Change from baseline of left ventricular ejection fraction (LVEF)
+8 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: ASP2016Experimental Treatment2 Interventions
Participants will receive a single dose of ASP2016. Participants will also receive daily prednisolone beginning 1 day prior to ASP2016 dose and for at least 16 weeks after ASP2016 dose, in order to suppress the immune response to ASP2016.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisolone
2005
Completed Phase 4
~2720

Find a Location

Who is running the clinical trial?

Astellas Gene TherapiesLead Sponsor
6 Previous Clinical Trials
390 Total Patients Enrolled
Global Medical LeadStudy DirectorAstellas Pharma Global Development, Inc.
5 Previous Clinical Trials
825 Total Patients Enrolled
~9 spots leftby Jan 2031
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