Duchenne Muscular DystrophyPhase 2NCT01978366
Open Label Extension Study of HT-100 in Patients With DMD
A Phase 2 study testing HT-100 in people with Duchenne Muscular Dystrophy, run by Processa Pharmaceuticals at 5 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 to 20 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- HT-100
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 5 sitesUnited States
- Study dates
- Oct 2013to 30 Apr 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 20 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, 21205
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of California, Davis Medical Center
Sacramento, California, 95817
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
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).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- HT-100
- Keywords
- Duchenne muscular dystrophy, halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HT-100Drug
Also called halofuginone hydrobromide delayed-release tablet
May be administered in either fed or fasted state
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: HT-100 tablet, Dose 1 | Experimental | • Multiple dose administration: Dose 1 Receives: Drug: HT-100 |
| Cohort 2: HT-100 tablet, Dose 2 | Experimental | • Multiple dose administration: Dose 2 Receives: Drug: HT-100 |
| Cohort 3: HT-100 tablet, Dose 3 | Experimental | • Multiple dose administration: Dose 3 Receives: Drug: HT-100 |
| Cohort 4: HT-100 tablet, Dose 4 | Experimental | • Multiple dose administration: Dose 4 Receives: Drug: HT-100 |
| Cohort 5: HT-100 tablet, Dose 5 | Experimental | • Multiple dose administration: Dose 5 Receives: Drug: HT-100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Months 2, 4, 6, 7
Secondary outcomes (2)
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
Measured: Months 4, 6, 7
Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Halofuginone plasma concentrations
Measured: Months 4, 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2013 (estimated)
- Study startOct 2013
- Primary completion30 Apr 2016
- Study completion30 Apr 2016
Why it stopped: Dosing stopped
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Processa Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Diana M Escolar, MD, Study director, AkashiTherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01978366
- Sponsor's protocol ID
- HALO-DMD-02
- Other IDs
- HALO
- Status last verified
- Jul 2019
- First submitted
- 31 Oct 2013
- Last updated
- 3 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
- ClinicalTrials.gov ID
- NCT01978366
- Acronym / sponsor ID
- HALO-DMD-02
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Dosing stopped
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Processa PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2013 (actual)
- Primary completion
- 30 Apr 2016 (actual)
- Study completion
- 30 Apr 2016 (actual)
- First posted
- 7 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: HT-100 tablet, Dose 1 | Experimental | Drug: HT-100• Multiple dose administration: Dose 1 |
| Cohort 2: HT-100 tablet, Dose 2 | Experimental | Drug: HT-100• Multiple dose administration: Dose 2 |
| Cohort 3: HT-100 tablet, Dose 3 | Experimental | Drug: HT-100• Multiple dose administration: Dose 3 |
| Cohort 4: HT-100 tablet, Dose 4 | Experimental | Drug: HT-100• Multiple dose administration: Dose 4 |
| Cohort 5: HT-100 tablet, Dose 5 | Experimental | Drug: HT-100• Multiple dose administration: Dose 5 |
Who may qualify
- Ages
- 6 to 20 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HT-100Also: halofuginone hydrobromide delayed-release tabletMay be administered in either fed or fasted state | Drug | 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 |
Primary outcome
Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
Time frame: Months 2, 4, 6, 7
* Target Safety profile by review of adverse events (AEs) * Physical examination findings * Clinical laboratory test results * Other diagnostic testing
2 secondary outcomes
Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Time frame: Months 4, 6, 7
Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
Time frame: Months 4, 6
Results and safety
The study was stopped early (reason given: Dosing stopped). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2020.
- Sponsor company websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Diana M Escolar, MD study director
AkashiTherapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HT-100 in people with Duchenne Muscular Dystrophy, run by Processa Pharmaceuticals at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Dosing stopped). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. (time frame: Months 2, 4, 6, 7). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2020; AzTrials last refreshed its copy on 6 Nov 2026.