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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
HT-100
Sponsored by
Processa Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor

Eligibility Criteria

6 Years - 20 Years (Child, Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, Davis Medical Center
  • Kennedy Krieger Institute, Johns Hopkins School of Medicine
  • Washington University School of Medicine
  • Cincinnati Children's Hospital Medical Center
  • Nationwide Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Cohort 1: HT-100 tablet, Dose 1

Cohort 2: HT-100 tablet, Dose 2

Cohort 3: HT-100 tablet, Dose 3

Cohort 4a: HT-100 tablet, Dose 4

Cohort 4b: HT-100 tablet, Dose 5

Cohort 5: HT-100 tablet, Dose 6

Arm Description

Single dose administration: Dose 1 Multiple dose administration: Dose 1

Single dose administration: Dose 2 Multiple dose administration: Dose 2

Single dose administration: Dose 3 Multiple dose administration: Dose 3

Single dose administration: Dose 4 Multiple dose administration: Dose 4

* Multiple dose administration: Dose 5

* Multiple dose administration: Dose 5

Outcomes

Primary Outcome Measures

Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing

Secondary Outcome Measures

Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
Halofuginone plasma concentrations
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
Pharmacodynamic measures relevant to DMD pathology: Pulmonary function Motor function Muscle composition Biochemical and imaging markers

Full Information

First Posted
May 2, 2013
Last Updated
August 31, 2020
Sponsor
Processa Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01847573
Brief Title
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Terminated
Why Stopped
Dosing stopped
Study Start Date
May 2013 (undefined)
Primary Completion Date
March 30, 2016 (Actual)
Study Completion Date
March 30, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Processa Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
17 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1: HT-100 tablet, Dose 1
Arm Type
Experimental
Arm Description
Single dose administration: Dose 1 Multiple dose administration: Dose 1
Arm Title
Cohort 2: HT-100 tablet, Dose 2
Arm Type
Experimental
Arm Description
Single dose administration: Dose 2 Multiple dose administration: Dose 2
Arm Title
Cohort 3: HT-100 tablet, Dose 3
Arm Type
Experimental
Arm Description
Single dose administration: Dose 3 Multiple dose administration: Dose 3
Arm Title
Cohort 4a: HT-100 tablet, Dose 4
Arm Type
Experimental
Arm Description
Single dose administration: Dose 4 Multiple dose administration: Dose 4
Arm Title
Cohort 4b: HT-100 tablet, Dose 5
Arm Type
Experimental
Arm Description
* Multiple dose administration: Dose 5
Arm Title
Cohort 5: HT-100 tablet, Dose 6
Arm Type
Experimental
Arm Description
* Multiple dose administration: Dose 5
Intervention Type
Drug
Intervention Name(s)
HT-100
Other Intervention Name(s)
halofuginone hydrobromide delayed-release tablet
Intervention Description
May be administered in either fed or fasted state
Primary Outcome Measure Information:
Title
Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
Description
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
Time Frame
1 week
Secondary Outcome Measure Information:
Title
Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
Description
Halofuginone plasma concentrations
Time Frame
1 week
Title
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
Description
Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
Time Frame
4 weeks
Title
Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
Description
Pharmacodynamic measures relevant to DMD pathology: Pulmonary function Motor function Muscle composition Biochemical and imaging markers
Time Frame
4 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Diana M Escolar, MD
Organizational Affiliation
Akashi Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
University of California, Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Kennedy Krieger Institute, Johns Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.akashirx.com/
Description
Sponsor company website

Learn more about this trial

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

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