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

NCT ID: NCT01847573

Last Updated: 2020-09-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-31

Study Completion Date

2016-03-30

Brief Summary

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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.

Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: HT-100 tablet, Dose 1

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

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Cohort 2: HT-100 tablet, Dose 2

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

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Cohort 3: HT-100 tablet, Dose 3

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

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Cohort 4a: HT-100 tablet, Dose 4

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

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Cohort 4b: HT-100 tablet, Dose 5

\* Multiple dose administration: Dose 5

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Cohort 5: HT-100 tablet, Dose 6

\* Multiple dose administration: Dose 5

Group Type EXPERIMENTAL

HT-100

Intervention Type DRUG

May be administered in either fed or fasted state

Interventions

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HT-100

May be administered in either fed or fasted state

Intervention Type DRUG

Other Intervention Names

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halofuginone hydrobromide delayed-release tablet

Eligibility Criteria

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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)

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
Minimum Eligible Age

6 Years

Maximum Eligible Age

20 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Processa Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Diana M Escolar, MD

Role: STUDY_DIRECTOR

Akashi Therapeutics

Locations

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University of California, Davis Medical Center

Sacramento, California, United States

Site Status

Kennedy Krieger Institute, Johns Hopkins School of Medicine

Baltimore, Maryland, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Countries

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United States

Related Links

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http://www.akashirx.com/

Sponsor company website

Other Identifiers

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HALO

Identifier Type: OTHER

Identifier Source: secondary_id

HALO-DMD-01

Identifier Type: -

Identifier Source: org_study_id

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