Duchenne Muscular Dystrophy CardiomyopathyPhase 2DMDNCT03340675
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
A Phase 2 study testing Ifetroban in people with Duchenne Muscular Dystrophy Cardiomyopathy and Cardiomyopathy, Dilated, run by Cumberland Pharmaceuticals at 10 sites in United States.
Trial summary
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. Read more about this study
- Who is it for?
- Ages 7 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
- What is being tested?
- Ifetroban
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 10 sitesUnited States
- Study dates
- 19 Oct 2020to 23 Jan 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 7 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males 7 years of age and older with the diagnosis of DMD, defined as phenotype consistent with DMD and either positive genotype, first degree relative with positive genotype, or confirmatory muscle biopsy.
- Stable dose of oral corticosteroids for at least 8 weeks or has not received corticosteroids for at least 30 days.
- Stable cardiac function defined as change in left ventricular ejection fraction (LVEF) of < 15% and no heart failure admission over the last 12 months; LVEF 35% or greater by cine cardiac magnetic resonance imaging (MRI) or echocardiography; myocardial damage in one or more left ventricular segments evident by late gadolinium enhancement allowed; concurrent angiotensin-converting enzyme inhibitors (ACEI), beta-blocker (BB) or angiotensin receptor blocker (ARB) therapy allowed (selection of which dictated by clinical care) if started three months or greater from first dose of IMP without change in dose. Aldosterone receptor antagonists (eg. Spironolactone or eplerenone) allowed if started 12 months or greater from first dose of Investigational Medicinal Product (IMP). No changes throughout the study allowed, except in the event of a decline in left ventricular ejection fraction (LVEF) >5% following the baseline CMR as measured by a subsequent CMR at the same center. Should this occur, changes in cardiac medications are allowed on the study.
- Late-stage cohort: Subjects are eligible for the late-stage cohort if the subject has: i. LVEF 35%-45% by cine cardiac magnetic resonance imaging (MRI) or echocardiography or ii. historically documented LVEF 35%-45% by cine cardiac magnetic resonance imaging (MRI) or echocardiography and if their baseline MRI is less than 50%.
- Subjects aged 18 years and older, informed consent obtained directly. For subjects ages 7-17 years old (yo), both assent from the subject and permission from a parent or guardian.
Who cannot join
- Clinically significant illness other than DMD
- Clinically significant laboratory abnormality not associated with DMD
- Major surgery within six weeks prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study procedures
- Require antiarrhythmic therapy and/or initiation of diuretic therapy for management of acute heart failure in the last 6 months
- A LVEF of < 35% by Cardiac Magnetic Resonance Imaging (CMR) and/or fractional shortening of < 15% based on echocardiography (ECHO) during screening
- A known bleeding disorder or has received anticoagulant treatment within 2 weeks of study entry
Show all 15Show fewer
- Allergy to gadolinium contrast or known renal insufficiency defined as abnormal cystatin C or creatinine above the upper limit of normal for age. The male serum reference ranges as follows:
- Age 7-9 years - 0.2-0.6 mg/dL
- Age 10-11 years - 0.3-0.7 mg/dL
- Age 12-13 years - 0.4-0.8 mg/dL
- Age 14-15 years - 0.5-0.9 mg/dL
- Age 16 years or older - 0.8-1.3 mg/dL
- Non-MR compatible implants (e.g. neurostimulator, automatic implantable cardioverter-defibrillator [AICD])
- Subjects who participated in a therapeutic clinical trial within 30 days or five half-lives (whichever is longer) of study entry
- Any other condition that could interfere with the subject's participation
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30341
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Lurie Children's Hospital
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Riley Children's Hospital
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Mattel Children's Hospital
Los Angeles, California, 90095
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Saint. Louis Children's Hospital
St Louis, Missouri, 63110
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD.
Funding Source - FDA OOPD
Read the detailed description
This is a phase 2 randomized, double-blind, placebo-controlled, multiple dose study with an optional open-label extension to determine the safety, pharmacokinetics (PK) and efficacy of two doses of oral ifetroban in subjects with DMD. DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban or placebo once daily for 12 months. Subjects will be enrolled into one of three treatment groups, low-dose ifetroban, high-dose ifetroban or placebo. Each dose level will be evaluated by eight subjects with early stage (LVEF > 45%) DMD-associated cardiomyopathy and eight subjects with more advanced stage (LVEF 35-45%) cardiac disease as there may be differences in the treatment effect based on cardiac involvement. Each subject treated will be evaluated for first-dose and steady-state exposure PK. All subjects who receive treatment will be assessed for safety. All subjects with at least one efficacy assessment post-baseline will be evaluated for efficacy. Blood and urine will be collected for standard and novel cardiac biomarkers. Target enrollment met for early-stage subjects (LVEF > 45%); study closed to enrollment prior to meeting target enrollment in the late-stage cohort (LVEF 35-45%). All subjects who complete the 12-month study are eligible to enter an optional open label extension period consisting of treatment with high-dose ifetroban only. Subjects in the open label extension will be evaluated for safety and cardiac efficacy every 12 months.
- Being tested
- Ifetroban, Placebo
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IfetrobanDrug
Weight based, once daily oral ifetroban
PlaceboDrug
Matching oral placebo
| Study group | Role | What they receive |
|---|---|---|
| Oral Ifetroban - Low Dose | Experimental | Weight based, once daily oral ifetroban Receives: Drug: Ifetroban |
| Oral Ifetroban - High Dose | Experimental | Weight based, once daily oral ifetroban Receives: Drug: Ifetroban |
| Placebos | Placebo comparator | Matching Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
Percentage of subjects with one or more treatment emergent adverse event
Measured: Baseline through 12 months
Secondary outcomes (14)
Pharmacokinetics Area Under the Curve
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. Area under the curve until the last measurement was calculated for the plasma concentration versus time curves for ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
Measured: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post-dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Maximum Serum Concentration (Cmax)
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the maximum plasma concentration of ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
Measured: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Time to Reach Cmax (Tmax) Concentration
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the time to maximum plasma concentration of ifetroban and its acyl glucuronide metabolite following administration of assigned oral ifetroban dose
Measured: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Plasma Terminal Half-life Concentration
Plasma ifetroban were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. The terminal elimination half-life was calculated from the plasma concentration versus time curves for ifetroban following administration of assigned oral ifetroban dose
Measured: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Change From Baseline in Left Ventricular Ejection Fraction
Change from baseline at month 12 in left ventricular ejection fraction
Measured: Baseline visit and Month 12 visit
Change From Baseline in FEV1
Change from baseline at month 12 in Forced Expiratory Volume in 1 second
Measured: Baseline through month 12
Change From Baseline in Percent Predicted FEV1
Change from baseline at month 12 in percent predicted Forced Expiratory Volume in 1 second
Measured: Baseline through month 12
Change From Baseline in FVC
Change from baseline at month 12 in Forced Vital Capacity
Measured: Baseline through month 12
Change From Baseline in Percent Predicted FVC
Change from baseline at month 12 in percent predicted Forced Vital Capacity
Measured: Baseline through month 12
Change From Baseline in Maximal Expiratory Pressure
Change from baseline at month 12 in maximal expiratory pressure
Measured: Baseline through month 12
Change From Baseline in Maximal Inspiratory Pressure
Change from baseline at month 12 in maximal inspiratory pressure
Measured: Baseline through month 12
Change From Baseline in Peak Expiratory Flow Rate
Change from baseline at month 12 in peak expiratory flow rate
Measured: Baseline through month 12
Change From Baseline in Percent Predicted Peak Expiratory Flow Rate
Change from baseline at month 12 in percent predicted peak expiratory flow rate
Measured: Baseline through month 12
Change From Baseline in Quality-of-life
The Pediatric Quality of Life Inventory (PedQL) questionnaire measures core dimensions of health as delineated by the World Health Organization.
The core module includes 23 items comprising 14 dimensions. The Gastrointestinal module includes 74 items comprising 14 dimensions. The Neuromuscular module includes 25 items comprising 3 dimensions. Each item in the modules is measured on a 5-point Likert scale of 0-4 with 0 indicating 'never' and 4 indicating 'almost always'.
The Likert scores for each subscale are reversed scored and linearly transform to a 0-100 scale with 0=100, 1-75, 2=50, 5=25, and 4=0.
A higher score indicates better health-related quality of life. Subscale scores are averaged to generate the total score reported with a range of 0-100.
Results are presented as change from baseline at 12 months. The questionnaire was completed by the participant and the parent/guardian of the participant.
Measured: Baseline through 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2017
- Study start19 Oct 2020
- Primary completion6 Mar 2024
- Study completion23 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular Dystrophy CardiomyopathyStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, placebo-controlled, double-blind, dose-ranging
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cumberland Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Vanderbilt University Medical Center
- Responsible party
- Sponsor
- Investigators
- Larry Markham, MD, Principal investigator, Riley Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03340675
- Sponsor's protocol ID
- CPI-IFE-007
- Other IDs
- FD-R-6371
- Acronym
- DMD
- Status last verified
- Mar 2026
- First submitted
- 3 Nov 2017
- Last updated
- 17 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Mitchell R, Frederick NE, Holzman ER, Agobe F, Allaway HCM, Bagher P. Ifetroban reduces coronary artery dysfunction in a mouse model of Duchenne muscular dystrophy. Am J Physiol Heart Circ Physiol. 2021 Jul 1;321(1):H52-H58. doi: 10.1152/ajpheart.00180.2021. Epub 2021 May 28.
PubMed 34048282
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03340675
- Acronym / sponsor ID
- DMD / CPI-IFE-007
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Cumberland PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Vanderbilt University Medical Center
- Study start
- 19 Oct 2020 (actual)
- Primary completion
- 6 Mar 2024 (actual)
- Study completion
- 23 Jan 2026 (actual)
- First posted
- 13 Nov 2017 (actual)
- Results
- Posted 26 Jan 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD.
Funding Source - FDA OOPD
Detailed description
This is a phase 2 randomized, double-blind, placebo-controlled, multiple dose study with an optional open-label extension to determine the safety, pharmacokinetics (PK) and efficacy of two doses of oral ifetroban in subjects with DMD. DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban or placebo once daily for 12 months. Subjects will be enrolled into one of three treatment groups, low-dose ifetroban, high-dose ifetroban or placebo. Each dose level will be evaluated by eight subjects with early stage (LVEF > 45%) DMD-associated cardiomyopathy and eight subjects with more advanced stage (LVEF 35-45%) cardiac disease as there may be differences in the treatment effect based on cardiac involvement. Each subject treated will be evaluated for first-dose and steady-state exposure PK. All subjects who receive treatment will be assessed for safety. All subjects with at least one efficacy assessment post-baseline will be evaluated for efficacy. Blood and urine will be collected for standard and novel cardiac biomarkers. Target enrollment met for early-stage subjects (LVEF > 45%); study closed to enrollment prior to meeting target enrollment in the late-stage cohort (LVEF 35-45%). All subjects who complete the 12-month study are eligible to enter an optional open label extension period consisting of treatment with high-dose ifetroban only. Subjects in the open label extension will be evaluated for safety and cardiac efficacy every 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Randomized, placebo-controlled, double-blind, dose-ranging
| Arm | Type | Receives |
|---|---|---|
| Oral Ifetroban - Low Dose | Experimental | Drug: IfetrobanWeight based, once daily oral ifetroban |
| Oral Ifetroban - High Dose | Experimental | Drug: IfetrobanWeight based, once daily oral ifetroban |
| Placebos | Placebo comparator | Drug: PlaceboMatching Placebo |
Who may qualify
- Ages
- 7 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Males 7 years of age and older with the diagnosis of DMD, defined as phenotype consistent with DMD and either positive genotype, first degree relative with positive genotype, or confirmatory muscle biopsy.
- Stable dose of oral corticosteroids for at least 8 weeks or has not received corticosteroids for at least 30 days.
- Stable cardiac function defined as change in left ventricular ejection fraction (LVEF) of < 15% and no heart failure admission over the last 12 months; LVEF 35% or greater by cine cardiac magnetic resonance imaging (MRI) or echocardiography; myocardial damage in one or more left ventricular segments evident by late gadolinium enhancement allowed; concurrent angiotensin-converting enzyme inhibitors (ACEI), beta-blocker (BB) or angiotensin receptor blocker (ARB) therapy allowed (selection of which dictated by clinical care) if started three months or greater from first dose of IMP without change in dose. Aldosterone receptor antagonists (eg. Spironolactone or eplerenone) allowed if started 12 months or greater from first dose of Investigational Medicinal Product (IMP). No changes throughout the study allowed, except in the event of a decline in left ventricular ejection fraction (LVEF) >5% following the baseline CMR as measured by a subsequent CMR at the same center. Should this occur, changes in cardiac medications are allowed on the study.
- Subjects aged 18 years and older, informed consent obtained directly. For subjects ages 7-17 years old (yo), both assent from the subject and permission from a parent or guardian.
Key exclusion criteria (15 listed)
- Clinically significant illness other than DMD
- Clinically significant laboratory abnormality not associated with DMD
- Major surgery within six weeks prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study procedures
- Require antiarrhythmic therapy and/or initiation of diuretic therapy for management of acute heart failure in the last 6 months
- A LVEF of < 35% by Cardiac Magnetic Resonance Imaging (CMR) and/or fractional shortening of < 15% based on echocardiography (ECHO) during screening
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 |
|---|---|---|
| IfetrobanWeight based, once daily oral ifetroban | Drug | Oral Ifetroban - High Dose, Oral Ifetroban - Low Dose |
| PlaceboMatching oral placebo | Drug | Placebos |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: Baseline through 12 months
Percentage of subjects with one or more treatment emergent adverse event
14 secondary outcomes
Pharmacokinetics Area Under the Curve
Time frame: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post-dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Maximum Serum Concentration (Cmax)
Time frame: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Time to Reach Cmax (Tmax) Concentration
Time frame: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Pharmacokinetics Plasma Terminal Half-life Concentration
Time frame: Pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7
Change From Baseline in Left Ventricular Ejection Fraction
Time frame: Baseline visit and Month 12 visit
Change From Baseline in FEV1
Time frame: Baseline through month 12
Change From Baseline in Percent Predicted FEV1
Time frame: Baseline through month 12
Change From Baseline in FVC
Time frame: Baseline through month 12
Change From Baseline in Percent Predicted FVC
Time frame: Baseline through month 12
Change From Baseline in Maximal Expiratory Pressure
Time frame: Baseline through month 12
Change From Baseline in Maximal Inspiratory Pressure
Time frame: Baseline through month 12
Change From Baseline in Peak Expiratory Flow Rate
Time frame: Baseline through month 12
Change From Baseline in Percent Predicted Peak Expiratory Flow Rate
Time frame: Baseline through month 12
Change From Baseline in Quality-of-life
Time frame: Baseline through 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34048282Mitchell R, Frederick NE, Holzman ER, Agobe F, Allaway HCM, Bagher P. Ifetroban reduces coronary artery dysfunction in a mouse model of Duchenne muscular dystrophy. Am J Physiol Heart Circ Physiol. 2021 Jul 1;321(1):H52-H58. doi: 10.1152/ajpheart.00180.2021. Epub 2021 May 28.
- Individual participant dataSharing planned: No
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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.
Larry Markham, MD principal investigator
Riley Children's Hospital
10 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 Ifetroban in people with Duchenne Muscular Dystrophy Cardiomyopathy and Cardiomyopathy, Dilated, run by Cumberland Pharmaceuticals at 10 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 5 inclusion and 15 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: finished, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 26 Jan 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Incidence of Treatment-Emergent Adverse Events (time frame: Baseline through 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03340675, which also lists 1 linked publication.
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 17 Mar 2026.