Duchenne Muscular DystrophyPhase 1/2NCT01847573
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
A Phase 1/2 study testing HT-100 in people with Duchenne Muscular Dystrophy, run by Processa Pharmaceuticals at 5 sites in United States.
Trial 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). 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
- May 2013to 30 Mar 2016
- Phase
- Phase 1/2
- 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
Main 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)
Main 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
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: A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- HT-100
- Keywords
- 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 | * Single dose administration: Dose 1
* Multiple dose administration: Dose 1 Receives: Drug: HT-100 |
| Cohort 2: HT-100 tablet, Dose 2 | Experimental | * Single dose administration: Dose 2
* Multiple dose administration: Dose 2 Receives: Drug: HT-100 |
| Cohort 3: HT-100 tablet, Dose 3 | Experimental | * Single dose administration: Dose 3
* Multiple dose administration: Dose 3 Receives: Drug: HT-100 |
| Cohort 4a: HT-100 tablet, Dose 4 | Experimental | * Single dose administration: Dose 4
* Multiple dose administration: Dose 4 Receives: Drug: HT-100 |
| Cohort 4b: HT-100 tablet, Dose 5 | Experimental | * Multiple dose administration: Dose 5 Receives: Drug: HT-100 |
| Cohort 5: HT-100 tablet, Dose 6 | 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 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
Measured: 1 week
Secondary outcomes (3)
Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
Halofuginone plasma concentrations
Measured: 1 week
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
Measured: 4 weeks
Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
Pharmacodynamic measures relevant to DMD pathology:
- Pulmonary function
- Motor function
- Muscle composition
- Biochemical and imaging markers
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2013 (estimated)
- Study startMay 2013
- Primary completion30 Mar 2016
- Study completion30 Mar 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 1/2
- 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, Akashi Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01847573
- Sponsor's protocol ID
- HALO-DMD-01
- Other IDs
- HALO
- Status last verified
- Mar 2019
- First submitted
- 2 May 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
- A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01847573
- Acronym / sponsor ID
- HALO-DMD-01
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Dosing stopped
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Processa PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2013
- Primary completion
- 30 Mar 2016 (actual)
- Study completion
- 30 Mar 2016 (actual)
- First posted
- 7 May 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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: HT-100 tablet, Dose 1 | Experimental | Drug: HT-100* Single dose administration: Dose 1 * Multiple dose administration: Dose 1 |
| Cohort 2: HT-100 tablet, Dose 2 | Experimental | Drug: HT-100* Single dose administration: Dose 2 * Multiple dose administration: Dose 2 |
| Cohort 3: HT-100 tablet, Dose 3 | Experimental | Drug: HT-100* Single dose administration: Dose 3 * Multiple dose administration: Dose 3 |
| Cohort 4a: HT-100 tablet, Dose 4 | Experimental | Drug: HT-100* Single dose administration: Dose 4 * Multiple dose administration: Dose 4 |
| Cohort 4b: HT-100 tablet, Dose 5 | Experimental | Drug: HT-100* Multiple dose administration: Dose 5 |
| Cohort 5: HT-100 tablet, Dose 6 | 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
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 4a: HT-100 tablet, Dose 4, Cohort 4b: HT-100 tablet, Dose 5, Cohort 5: HT-100 tablet, Dose 6 |
Primary outcome
Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
Time frame: 1 week
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
3 secondary outcomes
Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
Time frame: 1 week
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
Time frame: 4 weeks
Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
Time frame: 4 weeks
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
Akashi Therapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 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?
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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 administering single and multiple ascending doses of HT-100 in DMD boys (time frame: 1 week). 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.