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

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ClinicalTrials.gov Identifier: NCT02525302
Recruitment Status : Terminated (Dosing stopped)
First Posted : August 17, 2015
Last Update Posted : March 12, 2019
Sponsor:
Information provided by (Responsible Party):
Akashi Therapeutics

Brief 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. 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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: HT-100 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02
Study Start Date : May 2015
Actual Primary Completion Date : December 30, 2016
Actual Study Completion Date : December 30, 2016


Arm Intervention/treatment
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 Name: 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 Name: 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 Name: 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 Name: 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 Name: halofuginone hydrobromide




Primary Outcome Measures :
  1. Number of adverse events by severity and relationship [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  2. Dose reduction or modification due to upper GI or other adverse events [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  3. Trial discontinuations due to upper GI or other AEs [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  4. 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

  5. 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.

  6. Electrocardiograms [ Time Frame: Every 6 months from enrollment for up to 3 years ]
    Number of subjects with clinically significant changes in QT interval

  7. 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

  8. 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


Secondary Outcome Measures :
  1. Cardiovascular Magnetic Resonance [ Time Frame: Every 6 months from enrollment for up to 3 years ]
    Circumferential strain and myocardial fibrotic areas

  2. 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.

  3. Motor function measure (MFM) scale [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  4. Performance of upper limb (PUL) scale [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  5. 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.

  6. Quantitative muscle testing (QMT) scores [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  7. Timed function tests (TFTs) [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  8. Motor Function Measure (MFM) [ Time Frame: 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) [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  10. 9-hole peg test [ Time Frame: Every 6 months from enrollment for up to 3 years ]
    Assessment of upper limb function and dexterity

  11. 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.

  12. Electrical impedance myography (EIM) score [ Time Frame: Every 6 months from enrollment for up to 3 years ]

Other Outcome Measures:
  1. Pharmacokinetics peak plasma concentration (Cmax) [ Time Frame: Pre-dose and 2-4 hour post-dose ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02525302


Locations
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United States, California
University of California, Davis Medical Center
Sacramento, California, United States, 95817
United States, Maryland
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Akashi Therapeutics
Investigators
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Study Director: Diana M Escolar, MD Askashi Therapeutics

Additional Information:
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Responsible Party: Akashi Therapeutics
ClinicalTrials.gov Identifier: NCT02525302     History of Changes
Other Study ID Numbers: HALO-DMD-03
First Posted: August 17, 2015    Key Record Dates
Last Update Posted: March 12, 2019
Last Verified: March 2019
Keywords provided by Akashi Therapeutics:
DMD
neuromuscular
Duchenne
Additional relevant MeSH terms:
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Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Halofuginone
Antineoplastic Agents
Coccidiostats
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors