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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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to month 60

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

~3570

0 / 7Eligibility criteria met