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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 2
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 Duchenne muscular dystrophy, halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor

Eligibility Criteria

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

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

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 Type

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 4: HT-100 tablet, Dose 4

Cohort 5: HT-100 tablet, Dose 5

Arm Description

• Multiple dose administration: Dose 1

• Multiple dose administration: Dose 2

• Multiple dose administration: Dose 3

• Multiple dose administration: Dose 4

• Multiple dose administration: Dose 5

Outcomes

Primary Outcome Measures

Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
Target Safety profile by review of adverse events (AEs) Physical examination findings Clinical laboratory test results Other diagnostic testing

Secondary Outcome Measures

Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Pulmonary function Motor function Muscle composition Biochemical and imaging markers
Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Halofuginone plasma concentrations

Full Information

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

Unique Protocol Identification Number
NCT01978366
Brief Title
Open Label Extension Study of HT-100 in Patients With DMD
Official Title
An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Terminated
Why Stopped
Dosing stopped
Study Start Date
October 2013 (Actual)
Primary Completion Date
April 30, 2016 (Actual)
Study Completion Date
April 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
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).

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
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
• Multiple dose administration: Dose 1
Arm Title
Cohort 2: HT-100 tablet, Dose 2
Arm Type
Experimental
Arm Description
• Multiple dose administration: Dose 2
Arm Title
Cohort 3: HT-100 tablet, Dose 3
Arm Type
Experimental
Arm Description
• Multiple dose administration: Dose 3
Arm Title
Cohort 4: HT-100 tablet, Dose 4
Arm Type
Experimental
Arm Description
• Multiple dose administration: Dose 4
Arm Title
Cohort 5: HT-100 tablet, Dose 5
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 administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
Description
Target Safety profile by review of adverse events (AEs) Physical examination findings Clinical laboratory test results Other diagnostic testing
Time Frame
Months 2, 4, 6, 7
Secondary Outcome Measure Information:
Title
Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Description
Pulmonary function Motor function Muscle composition Biochemical and imaging markers
Time Frame
Months 4, 6, 7
Title
Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Description
Halofuginone plasma concentrations
Time Frame
Months 4, 6

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Diana M Escolar, MD
Organizational Affiliation
AkashiTherapeutics
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

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

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