NCT01978366

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).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2013

Geographic Reach
1 country

5 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2013

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

October 31, 2013

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 7, 2013

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 30, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 30, 2016

Completed
Last Updated

September 3, 2020

Status Verified

July 1, 2019

Enrollment Period

2.6 years

First QC Date

October 31, 2013

Last Update Submit

August 31, 2020

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophyhalofuginone hydrobromideanti-fibroticanti-inflammatorymuscle regenerationprotein synthesis inhibitor

Outcome Measures

Primary Outcomes (1)

  • 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

    Months 2, 4, 6, 7

Secondary Outcomes (2)

  • Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.

    Months 4, 6, 7

  • Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.

    Months 4, 6

Study Arms (5)

Cohort 1: HT-100 tablet, Dose 1

EXPERIMENTAL

• Multiple dose administration: Dose 1

Drug: HT-100

Cohort 2: HT-100 tablet, Dose 2

EXPERIMENTAL

• Multiple dose administration: Dose 2

Drug: HT-100

Cohort 3: HT-100 tablet, Dose 3

EXPERIMENTAL

• Multiple dose administration: Dose 3

Drug: HT-100

Cohort 4: HT-100 tablet, Dose 4

EXPERIMENTAL

• Multiple dose administration: Dose 4

Drug: HT-100

Cohort 5: HT-100 tablet, Dose 5

EXPERIMENTAL

• Multiple dose administration: Dose 5

Drug: HT-100

Interventions

HT-100DRUG

May be administered in either fed or fasted state

Also known as: halofuginone hydrobromide delayed-release tablet
Cohort 1: HT-100 tablet, Dose 1Cohort 2: HT-100 tablet, Dose 2Cohort 3: HT-100 tablet, Dose 3Cohort 4: HT-100 tablet, Dose 4Cohort 5: HT-100 tablet, Dose 5

Eligibility Criteria

Age6 Years - 20 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

University of California, Davis Medical Center

Sacramento, California, 95817, United States

Location

Kennedy Krieger Institute, Johns Hopkins School of Medicine

Baltimore, Maryland, 21205, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Related Links

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Diana M Escolar, MD

    AkashiTherapeutics

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 31, 2013

First Posted

November 7, 2013

Study Start

October 1, 2013

Primary Completion

April 30, 2016

Study Completion

April 30, 2016

Last Updated

September 3, 2020

Record last verified: 2019-07

Locations

US(5)