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Duchenne Muscular DystrophyPhase 2NCT02525302

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

A Phase 2 study testing HT-100 in people with Duchenne Muscular Dystrophy, run by Akashi Therapeutics at 5 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 6 to 20 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
HT-100
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
5 sitesUnited States
Study dates
May 2015to 30 Dec 2016
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 20 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Who may be able to join

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

Who cannot join

  • Answering yes to any of the following make the subject NOT eligible to participate in the study.
  • 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
  • History of severe allergic or anaphylactic reactions
  • Recent report of drug/alcohol abuse

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Kennedy Krieger Institute, Johns Hopkins School of Medicine

    Baltimore, Maryland, 21205

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • University of California, Davis Medical Center

    Sacramento, California, 95817

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: 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).

Read the 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.

Being tested
HT-100
Keywords
DMD, neuromuscular, Duchenne
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HT-100Drug

    Also called halofuginone hydrobromide

    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.

Study groups in NCT02525302
Study groupRoleWhat they receive
Cohort 1: HT-100 tablet, Dose 1ExperimentalHT-100 multiple dose administration (dose 1).
Receives: Drug: HT-100
Cohort 1: HT-100 tablet, Dose 2ExperimentalHT-100 multiple dose administration (dose 1).
Receives: Drug: HT-100
Cohort 1: HT-100 tablet, Dose 3ExperimentalHT-100 multiple dose administration (dose 1).
Receives: Drug: HT-100
Cohort 1: HT-100 tablet, Dose 4ExperimentalHT-100 multiple dose administration (dose 1).
Receives: Drug: HT-100
Cohort 1: HT-100 tablet, Dose 5ExperimentalHT-100 multiple dose administration (dose 1).
Receives: Drug: HT-100

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of adverse events by severity and relationship

    Measured: Every 6 months from enrollment for up to 3 years

  2. Dose reduction or modification due to upper GI or other adverse events

    Measured: Every 6 months from enrollment for up to 3 years

  3. Trial discontinuations due to upper GI or other AEs

    Measured: Every 6 months from enrollment for up to 3 years

  4. Vital signs (Number of subjects with clinically significant changes)

    Number of subjects with clinically significant changes

    Measured: Every 6 months from enrollment for up to 3 years

  5. Laboratory values (Number of subjects with clinically significant changes)

    Number of subjects with clinically significant changes.

    Measured: Every 6 months from enrollment for up to 3 years

  6. Electrocardiograms

    Number of subjects with clinically significant changes in QT interval

    Measured: Every 6 months from enrollment for up to 3 years

  7. Echocardiograms

    Number of subjects with clinically significant changes in left ventricular ejection fraction, end systolic and diastolic interventricular septal thickness, left ventricular posterior wall thickness

    Measured: Every 6 months from enrollment for up to 3 years

  8. Cardiovascular Magnetic Resonance

    Number of subjects with clinically significant change in diagnostic interpretation

    Measured: Every 6 months from enrollment for up to 3 years

Secondary outcomes (12)

  1. Cardiovascular Magnetic Resonance

    Circumferential strain and myocardial fibrotic areas

    Measured: Every 6 months from enrollment for up to 3 years

  2. Pulmonary function testing (Number of subjects with clinically significant changes)

    Number of subjects with clinically significant changes.

    Measured: Every 6 months from enrollment for up to 3 years

  3. Motor function measure (MFM) scale

    Measured: Every 6 months from enrollment for up to 3 years

  4. Performance of upper limb (PUL) scale

    Measured: Every 6 months from enrollment for up to 3 years

  5. Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes)

    Number of subjects with clinically significant changes.

    Measured: Every 6 months from enrollment for up to 3 years

  6. Quantitative muscle testing (QMT) scores

    Measured: Every 6 months from enrollment for up to 3 years

  7. Timed function tests (TFTs)

    Measured: Every 6 months from enrollment for up to 3 years

  8. Motor Function Measure (MFM)

    Measured: Every 6 months from enrollment for up to 3 years

  9. Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL)

    Measured: Every 6 months from enrollment for up to 3 years

  10. 9-hole peg test

    Assessment of upper limb function and dexterity

    Measured: Every 6 months from enrollment for up to 3 years

  11. Tip pinch and key pinch tests (Number of subjects with clinically significant changes)

    Number of subjects with clinically significant changes.

    Measured: Every 6 months from enrollment for up to 3 years

  12. Electrical impedance myography (EIM) score

    Measured: Every 6 months from enrollment for up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Aug 2015 (estimated)
  2. Study startMay 2015
  3. Primary completion30 Dec 2016
  4. Study completion30 Dec 2016

Why it stopped: Dosing stopped

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02525302
Sponsor's protocol ID
HALO-DMD-03
Status last verified
Mar 2019
First submitted
18 Jul 2015
Last updated
12 Mar 2019
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing HT-100 in people with Duchenne Muscular Dystrophy, run by Akashi Therapeutics at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 12 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Dosing stopped). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of adverse events by severity and relationship (time frame: Every 6 months from enrollment for up to 3 years). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02525302.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2019.

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