NCT01847573

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.

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_1

Timeline
Completed

Started May 2013

Typical duration for phase_1

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

May 1, 2013

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

May 2, 2013

Completed
5 days until next milestone

First Posted

Study publicly available on registry

May 7, 2013

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 30, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 30, 2016

Completed
Last Updated

September 3, 2020

Status Verified

March 1, 2019

Enrollment Period

2.9 years

First QC Date

May 2, 2013

Last Update Submit

August 31, 2020

Conditions

Duchenne Muscular Dystrophy

Keywords

halofuginone hydrobromideanti-fibroticanti-inflammatorymuscle regenerationprotein synthesis inhibitor

Outcome Measures

Primary Outcomes (1)

  • 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

    1 week

Secondary Outcomes (3)

  • Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys

    1 week

  • Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks

    4 weeks

  • Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys

    4 weeks

Study Arms (6)

Cohort 1: HT-100 tablet, Dose 1

EXPERIMENTAL

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

Drug: HT-100

Cohort 2: HT-100 tablet, Dose 2

EXPERIMENTAL

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

Drug: HT-100

Cohort 3: HT-100 tablet, Dose 3

EXPERIMENTAL

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

Drug: HT-100

Cohort 4a: HT-100 tablet, Dose 4

EXPERIMENTAL

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

Drug: HT-100

Cohort 4b: HT-100 tablet, Dose 5

EXPERIMENTAL

\* Multiple dose administration: Dose 5

Drug: HT-100

Cohort 5: HT-100 tablet, Dose 6

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 4a: HT-100 tablet, Dose 4Cohort 4b: HT-100 tablet, Dose 5Cohort 5: HT-100 tablet, Dose 6

Eligibility Criteria

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

You may qualify if:

  • 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)

You may not qualify if:

  • 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

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

    Akashi Therapeutics

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 2, 2013

First Posted

May 7, 2013

Study Start

May 1, 2013

Primary Completion

March 30, 2016

Study Completion

March 30, 2016

Last Updated

September 3, 2020

Record last verified: 2019-03

Locations

US(5)