Open Label Extension Study of HT-100 in Patients With DMD

August 31, 2020 updated by: Processa Pharmaceuticals

An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01

This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

17

Phase

  • 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

Ages Eligible for Study

6 years to 20 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01
  • Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01
  • Ability to provide written informed consent
  • Ambulatory or non-ambulatory

Exclusion Criteria:

  • Recent, substantial change in use of cardiac medications or medications affecting muscle function
  • Clinically significant major disease, not related to DMD
  • Significantly compromised cardio-respiratory function
  • History of severe allergic or anaphylactic reactions
  • Prior treatment with another investigational product in past 6 months
  • Inability to undergo magnetic resonance imaging (MRI)
  • Current drug or alcohol abuse or prior treatment for 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
Intervention / Treatment
Experimental: Cohort 1: HT-100 tablet, Dose 1
• Multiple dose administration: Dose 1
Drug: HT-100
May be administered in either fed or fasted state
Other Names:
  • halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 2: HT-100 tablet, Dose 2
• Multiple dose administration: Dose 2
Drug: HT-100
May be administered in either fed or fasted state
Other Names:
  • halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 3: HT-100 tablet, Dose 3
• Multiple dose administration: Dose 3
Drug: HT-100
May be administered in either fed or fasted state
Other Names:
  • halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 4: HT-100 tablet, Dose 4
• Multiple dose administration: Dose 4
Drug: HT-100
May be administered in either fed or fasted state
Other Names:
  • halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 5: HT-100 tablet, Dose 5
• Multiple dose administration: Dose 5
Drug: HT-100
May be administered in either fed or fasted state
Other Names:
  • halofuginone hydrobromide delayed-release tablet

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
Time Frame: Months 2, 4, 6, 7
  • Target Safety profile by review of adverse events (AEs)
  • Physical examination findings
  • Clinical laboratory test results
  • Other diagnostic testing
Months 2, 4, 6, 7

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Time Frame: Months 4, 6, 7
  • Pulmonary function
  • Motor function
  • Muscle composition
  • Biochemical and imaging markers
Months 4, 6, 7
Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Time Frame: Months 4, 6
Halofuginone plasma concentrations
Months 4, 6

Collaborators and Investigators

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

Sponsor

Processa Pharmaceuticals

Investigators

  • Study Director: Diana M Escolar, MD, AkashiTherapeutics

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 (Actual)

October 1, 2013

Primary Completion (Actual)

April 30, 2016

Study Completion (Actual)

April 30, 2016

Study Registration Dates

First Submitted

October 31, 2013

First Submitted That Met QC Criteria

October 31, 2013

First Posted (Estimate)

November 7, 2013

Study Record Updates

Last Update Posted (Actual)

September 3, 2020

Last Update Submitted That Met QC Criteria

August 31, 2020

Last Verified

July 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HALO-DMD-02
  • HALO (Akashi Therapeutics)

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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