A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

November 24, 2025 updated by: Astellas Gene Therapies

A Multicenter, Open-Label, Dose Escalation, Phase 1b Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia Cardiomyopathy

Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene.

At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia.

In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems.

The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016.

Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion.

For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise.

After the 12-month visit, people will visit the clinic every few months for up to a few years.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California - Los Angeles
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas - Houston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.
  • Participant has a resting LVEF ≥ 40% and < 55% as measured at screening by ECHO.
  • Participant has a body mass index range of 17.0 to 30.0 kg/m2.
  • Participant agrees not to begin omaveloxolone treatment during the 52-week period after receiving study intervention.
  • Participants on omaveloxolone, who opt to discontinue omaveloxolone, may enroll if they stop omaveloxolone for 3 weeks and pass study screening, including LFTs.
  • Participants on omaveloxolone, who opt to stay on omaveloxolone will need to have been on it for a minimum of 3 months, with LFTs that pass diagnostic assessments exclusion criteria at screening and prior to dosing with ASP2016. Prior elevation(s) in AST/ALT associated with omaveloxolone use must be discussed with the sponsor medical monitor. If there is a liver function test (LFT) elevation after treatment, participant agrees to stop omaveloxolone treatment until 52 weeks.
  • Participants on omaveloxolone will need to discontinue strong or moderate cytochrome P450 3A4 (CYP3A4) inducers and inhibitors.
  • Woman of Child Bearing Potential (WOCBP) on omaveloxolone must use a nonhormonal, highly effective methods of contraception (e.g., nonhormonal intrauterine device system), as omaveloxolone may interfere with hormonal methods of contraception.

Exclusion Criteria:

  • Participant has late-onset FA, defined as symptom onset after the age of 25 years.
  • Participant is unable to complete cardiopulmonary exercise testing (CPET) procedure.
  • Participant has a contraindication to endomyocardial biopsy or cardiac catheterization.
  • Participant has a contraindication to cardiac magnetic resonance imaging (CMRI) with contrast, including hypersensitivity to gadolinium contrast agent, cardiac pacemaker or implantable cardiac defibrillator.
  • Participant has an elevated titer of anti-AAV8 total antibodies, as determined by central testing.
  • Participant has significant fibrosis on CMRI, defined as late gadolinium enhancement of > 15% left ventricular myocardial mass.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ASP2016
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.
Drug: Prednisolone
Oral
Genetic: ASP2016
Intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time Frame: Up to month 60

A TEAE is defined as an AE observed after starting administration of the study intervention.

An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

Note: an AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of study intervention. This includes events related to the comparator, if applicable, and events related to the (study) procedures.
Up to month 60
Number of Participants with Serious Adverse Events (SAEs)
Time Frame: Up to month 60
An SAE is defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, and other medically important events.
Up to month 60
Number of participants with laboratory value abnormalities and/or AEs
Time Frame: Up to month 60
Number of participants with potentially clinically significant laboratory values.
Up to month 60
Number of participants with electrocardiogram (ECG) abnormalities and/or AEs
Time Frame: Up to month 60
Number of participants with potentially clinically significant ECG values.
Up to month 60
Number of participants with physical exam value abnormalities and/or AEs
Time Frame: Up to month 60
Number of participants with potentially clinically significant physical exam values.
Up to month 60

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change from baseline of frataxin protein level in cardiac tissue
Time Frame: Baseline, week 24 and week 52
Frataxin protein level in cardiac tissue will be recorded from endomyocardial biopsies collected by cardiac catheterization.
Baseline, week 24 and week 52
Change from baseline of peak rate of oxygen consumption (VO2peak)
Time Frame: Baseline, week 24 and week 52
VO2peak will be measured by upper extremity cardiopulmonary exercise testing (CPET).
Baseline, week 24 and week 52
Change from baseline of ventilatory anaerobic threshold (AT)
Time Frame: Baseline, week 24 and week 52
AT will be measured by upper extremity CPET.
Baseline, week 24 and week 52
Change from baseline of ventilation (VE)/volume of exhaled carbon dioxide (VCO2) slope
Time Frame: Baseline, week 24 and week 52
VE/VCO2 slope will be measured by upper extremity CPET.
Baseline, week 24 and week 52
Change from baseline of left ventricular ejection fraction (LVEF)
Time Frame: Baseline, week 24 and week 52
LVEF will be measured by echocardiogram (ECHO).
Baseline, week 24 and week 52
Change from baseline of left ventricular mass index (LVMI)
Time Frame: Baseline, week 24 and week 52
LVMI will be measured by ECHO.
Baseline, week 24 and week 52
Change from baseline of longitudinal cardiac strain
Time Frame: Baseline, week 24 and week 52
Longitudinal cardiac strain will be measured by ECHO.
Baseline, week 24 and week 52
Change from baseline of vector copy number (VCN)
Time Frame: Baseline, week 24 and week 52
VCN will be recorded from endomyocardial biopsies collected by cardiac catheterization.
Baseline, week 24 and week 52
Change from baseline of total antibody (TAb) to adeno-associated virus 8 (AAV8)
Time Frame: Baseline and up to week 52
TAb to AAV8 will be recorded from serum samples collected.
Baseline and up to week 52
Change from baseline of neutralizing antibody (NAb) to AAV8
Time Frame: Baseline and up to week 52
NAb to AAV8 will be recorded from serum samples collected.
Baseline and up to week 52
Change from baseline of TAb to frataxin
Time Frame: Baseline and up to week 52
TAb to frataxin will be recorded from serum samples collected.
Baseline and up to week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Astellas Gene Therapies

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Global Medical Lead, Astellas Pharma Global Development, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 8, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 22, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 22, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 26, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 26, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 3, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 28, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 24, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 2016-CL-0101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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