HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

March 8, 2019 updated by: Akashi Therapeutics

HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02

This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with Duchenne Muscular Dystrophy (DMD).

Study Overview

Status

Status

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

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.

Detailed Description

As a follow-on study to the initial clinical studies of HT-100 in DMD (Protocols HALO-DMD-01 and HALO-DMD-02), this open-label study is designed to provide data on continuous long-term dosing. Subjects will be entered into the study without cessation of dosing, in a staggered fashion, into the same cohort assignment they had in the predecessor studies. Up to 30 subjects who have completed dosing in HALO-DMD-02 will be offered the opportunity to continue on the same dose regimen until market approval of HT-100 or termination of the study by the Sponsor. Reasons for termination could include, among others, safety concerns or lack of efficacy, based on analysis of combined data from all HT-100 studies. Safety data from subjects approaching the end the HALO-DMD-02 participation will be individually reviewed by the Medical Monitor and the subject's physician (Principal Investigator [PI]). If the Medical Monitor and the PI agree there are no clinically significant safety signals (absence of clinically significant laboratory or clinical abnormalities to date), the subject will be considered eligible and offered continuation of dosing. To avoid an interruption in dosing, subjects will immediately be screened for participation and enrolled upon completing the predecessor trial, HALO-DMD-02. Participation is in this study HALO-DMD-03 is optional. Safety and pharmacodynamics (PD) monitoring will continue throughout the subject's study participation. Dose reduction/modification might occur or individual subjects' participation in the trial may be discontinued if any Adverse Events (AEs) suggest that HT-100 is not sufficiently well tolerated.

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

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
10

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 2

Contacts and Locations

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

Study Locations

  • United States
    • California
      • Sacramento, California, United States, 95817
        • University of California, Davis Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute, Johns Hopkins School of Medicine
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital

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

6 years to 20 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. Completed both previous studies HALO-DMD-01 and HALO-DMD-02
  2. Ability to provide written informed consent
  3. Ability to understand and follow site and protocol instruction for the entire duration of the study

Exclusion Criteria:

Answering yes to any of the following make the subject NOT eligible to participate in the study.

  1. Clinically significant major disease not related to DMD that would make it not safe to be in the study or affect ability to follow the protocol
  2. History of severe allergic or anaphylactic reactions
  3. Recent report of drug/alcohol abuse

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: Non-Randomized
  • Interventional Model: Parallel 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: Cohort 1: HT-100 tablet, Dose 1
HT-100 multiple dose administration (dose 1).
Drug: HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.
Other Names:
  • halofuginone hydrobromide
Experimental: Cohort 1: HT-100 tablet, Dose 2
HT-100 multiple dose administration (dose 1).
Drug: HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.
Other Names:
  • halofuginone hydrobromide
Experimental: Cohort 1: HT-100 tablet, Dose 3
HT-100 multiple dose administration (dose 1).
Drug: HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.
Other Names:
  • halofuginone hydrobromide
Experimental: Cohort 1: HT-100 tablet, Dose 4
HT-100 multiple dose administration (dose 1).
Drug: HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.
Other Names:
  • halofuginone hydrobromide
Experimental: Cohort 1: HT-100 tablet, Dose 5
HT-100 multiple dose administration (dose 1).
Drug: HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.
Other Names:
  • halofuginone hydrobromide

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 adverse events by severity and relationship
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Dose reduction or modification due to upper GI or other adverse events
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Trial discontinuations due to upper GI or other AEs
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Vital signs (Number of subjects with clinically significant changes)
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes
Every 6 months from enrollment for up to 3 years
Laboratory values (Number of subjects with clinically significant changes)
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes.
Every 6 months from enrollment for up to 3 years
Electrocardiograms
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes in QT interval
Every 6 months from enrollment for up to 3 years
Echocardiograms
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes in left ventricular ejection fraction, end systolic and diastolic interventricular septal thickness, left ventricular posterior wall thickness
Every 6 months from enrollment for up to 3 years
Cardiovascular Magnetic Resonance
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant change in diagnostic interpretation
Every 6 months from enrollment for up to 3 years

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
Cardiovascular Magnetic Resonance
Time Frame: Every 6 months from enrollment for up to 3 years
Circumferential strain and myocardial fibrotic areas
Every 6 months from enrollment for up to 3 years
Pulmonary function testing (Number of subjects with clinically significant changes)
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes.
Every 6 months from enrollment for up to 3 years
Motor function measure (MFM) scale
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Performance of upper limb (PUL) scale
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes)
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes.
Every 6 months from enrollment for up to 3 years
Quantitative muscle testing (QMT) scores
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Timed function tests (TFTs)
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Motor Function Measure (MFM)
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL)
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years
9-hole peg test
Time Frame: Every 6 months from enrollment for up to 3 years
Assessment of upper limb function and dexterity
Every 6 months from enrollment for up to 3 years
Tip pinch and key pinch tests (Number of subjects with clinically significant changes)
Time Frame: Every 6 months from enrollment for up to 3 years
Number of subjects with clinically significant changes.
Every 6 months from enrollment for up to 3 years
Electrical impedance myography (EIM) score
Time Frame: Every 6 months from enrollment for up to 3 years
Every 6 months from enrollment for up to 3 years

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Time Frame
Pharmacokinetics peak plasma concentration (Cmax)
Time Frame: Pre-dose and 2-4 hour post-dose
Pre-dose and 2-4 hour post-dose

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.
Akashi Therapeutics

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: Diana M Escolar, MD, Askashi Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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

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.
May 1, 2015

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.
December 30, 2016

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.
December 30, 2016

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).
July 18, 2015

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.
August 14, 2015

First Posted (Estimate)

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.
August 17, 2015

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.
March 12, 2019

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.
March 8, 2019

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.
March 1, 2019

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.
  • HALO-DMD-03

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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