Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

August 31, 2020 updated by: Processa Pharmaceuticals

A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy

The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

17

Phase

  • Phase 2
  • 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 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 (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Main Inclusion Criteria:

  • Ambulatory or non-ambulatory
  • Diagnosis of DMD with confirmation of minimal to no dystrophin
  • Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)

Main Exclusion Criteria:

  • Recent, substantial change in use of cardiac medications or medications affecting muscle function
  • Inability to undergo magnetic resonance imaging (MRI)
  • Significantly compromised cardio-respiratory function
  • Prior treatment with another investigational product in past 6 months

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
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Cohort 1: HT-100 tablet, Dose 1
  • Single dose administration: 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
  • Single dose administration: 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
  • Single dose administration: 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 4a: HT-100 tablet, Dose 4
  • Single dose administration: 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 4b: 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
EXPERIMENTAL: Cohort 5: HT-100 tablet, Dose 6
* 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 administering single and multiple ascending doses of HT-100 in DMD boys
Time Frame: 1 week
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
1 week

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
Time Frame: 1 week
Halofuginone plasma concentrations
1 week
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
Time Frame: 4 weeks
Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
4 weeks
Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
Time Frame: 4 weeks

Pharmacodynamic measures relevant to DMD pathology:

  • Pulmonary function
  • Motor function
  • Muscle composition
  • Biochemical and imaging markers
4 weeks

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, Akashi 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

May 1, 2013

Primary Completion (ACTUAL)

March 30, 2016

Study Completion (ACTUAL)

March 30, 2016

Study Registration Dates

First Submitted

May 2, 2013

First Submitted That Met QC Criteria

May 6, 2013

First Posted (ESTIMATE)

May 7, 2013

Study Record Updates

Last Update Posted (ACTUAL)

September 3, 2020

Last Update Submitted That Met QC Criteria

August 31, 2020

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HALO-DMD-01
  • 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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