Myotonic DystrophyPhase 3NCT06523400
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Phase 3 study testing Mexiletine granules for prolonged-release oral suspension in people with Myotonic Dystrophy, run by Lupin Ltd. at 7 sites in 6 countries.
Trial summary
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study) Study website: https://myotonicdystrophystudy.com/ Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- Mexiletine granules for prolonged-release oral suspension
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 176 participants
- Where
- 7 sitesin 6 countries
- Study dates
- 13 Feb 2025to Jun 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- DM1 or DM2 diagnosis confirmed genetically;
- Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)/legal guardian and written assent from the patient (if patient < 18 years of age);
- Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment;
- Male or non-pregnant female ≥16 years of age;
- Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg;
- Female patients of childbearing potential must be using a highly effective form of birth control for the duration of the study and for at least 7 days after last dose of study drug;
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- No significant cardiac abnormalities as determined by a cardiologist's assessment;
- Have sufficient finger flexor strength to grasp the handle of the dynamometer used to measure myotonia;
- Presence of clinical handgrip myotonia (delayed relaxation of grip of ≥ 3 seconds after maximum voluntary contraction) at screening using VHOT;
- Be able to walk independently 10 meters (cane, walker, orthoses allowed);
- DM1 patients only - Muscular impairment rating scale (MIRS) score of 2, 3 or 4.-
Who cannot join
- Are pregnant or lactating;
- Have any one of the following medical conditions: uncontrolled diabetes mellitus, cancer other than skin cancer less than five years previously (e.g., basal-cell carcinoma (BCC) and squamous-cell carcinoma (SCC) of skin allowed), multiple sclerosis, seizure disorders, or other serious medical illness;
- Severe renal impairment (glomerular filtration rate (GFR) < 30 mL/min);
- Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;
- Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;
- Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
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- High incidence of falls or fall-associated fractures (>5 falls during the past 12 months);
- Preexisting elevated liver function tests > 3 times the upper limit of normal (ULN) at screening (alanine transaminase (ALT)/aspartate transaminase (AST), gamma-glutamyl transferase (GGT)) and/or any abnormal chemistry, hematology or urine lab considered clinically significant by investigator;
- Serum potassium values < 3.5 mmol/L or > 5.0 mmol/L or serum magnesium values < 1.7 mg/dL. Electrolytic imbalance such as hypokalaemia, hyperkalaemia or hypomagnesaemia may increase the proarrhythmic effects of mexiletine. Electrolyte imbalances need to be corrected before administering mexiletine and will be monitored throughout treatment.
- Treatment with mexiletine within 4 weeks prior to baseline (Day 1);
- Intake of any anti-myotonic treatment within 4 weeks prior to baseline (Day 1) or 5 half-lives, whichever is longer such as metformin, such as propafenone, flecainide, lamotrigine, carbamazepine or any other channel-blocker/ anticonvulsive drugs;
- Use of any concomitant medications that could increase the cardiac risk;
- Known allergy to mexiletine or any local anesthetics;
- Participation in another interventional clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer;
- Wheelchair-bound or bed-ridden;
- Any cardiac safety associated condition including any of the following criteria detected by screening cardiac evaluations including 24-hr Holter monitor, echocardiogram and clinical evaluations:
- PR interval ≥240 ms or QRS duration ≥120 ms on resting ECG
- Personal history of 3rd degree or 2nd degree type 2 atrioventricular block or sinus node dysfunction with pauses ≥3 seconds, complete bundle branch block, bifascicular and trifascicular block or any heart block susceptible to evolve to complete heart block
- Personal history of sustained atrial fibrillation, flutter or tachycardia (duration >30 seconds)
- Personal history of non-sustained (ventricular triplets or more) or sustained ventricular tachycardia
- Myocardial infarction (acute or past) or coronary artery stenosis >50%, presence of abnormal Q waves
- New York Heart Association (NYHA) Class II to IV heart failure
- Left ventricular systolic dysfunction with ejection fraction <50%
- Sinus node dysfunction (including ECG sinus rate <50 beats per minute (BPM))
- Co-administration of antiarrhythmics inducing torsades de pointes (class Ia: quinidine, procainamide, disopyramide, ajmaline; class Ic: encainide, flecainide, propafenone, moricizine; class III: amiodarone, sotalol, ibutilide, dofetilide, dronedarone, vernakalant)
- Co-administration of other classes of antiarrhythmics (class Ib: lidocaine, phenytoin, tocainide; class II: propranolol, esmolol, timolol, metoprolol, atenolol, carvedilol, bisoprolol, nebivolol; class IV: verapamil, diltiazem)
- Patients with implantable cardioverter defibrillators (ICDs) and pacemakers are excluded
- Presence of symptomatic coronary artery disease
Trial locations and contacts
7 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 6 countries
United Kingdom2 sites, 2 recruiting
- Recruiting
Saint George's University Hospitals NHS Foundation Trust
London
neurogenetics@stgeorges.nhs.uk
- Recruiting
University College Hospital
London
Belgium1 site, 1 recruiting
- Recruiting
Laboratory for Muscle Diseases and Neuropathies
Leuven
Italy1 site, 1 recruiting
- Recruiting
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
Rome
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 (Phase 3)
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)
Study website: https://myotonicdystrophystudy.com/
Read the detailed description
This is a multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4- week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients in total complete/early terminate the study.
In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo).
Study website: https://myotonicdystrophystudy.com/
- Conditions
- Myotonic Dystrophy
- Being tested
- Mexiletine granules for prolonged-release oral suspension, Placebo
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mexiletine granules for prolonged-release oral suspensionDrug
Mexiletine PR
PlaceboDrug
Matching Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 0 mg (matching sachet volumes to low, medium and high dose active drug) Receives: Drug: Placebo |
| Mexiletine prolonged-release (PR) | Active comparator | Mexiletine PR 167 mg (mexiletine HCl 200 mg) Mexiletine PR 333 mg (mexiletine HCl 400 mg) OR Mexiletine PR 500 mg (mexiletine HCl 600 mg) Receives: Drug: Mexiletine granules for prolonged-release oral suspension |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the efficacy of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2).
Handgrip relaxation time in DM1 patients by mean change in baseline of handgrip relaxation time (seconds) after maximal voluntary isometric contraction (MVIC)
Measured: 26 weeks
Secondary outcomes (19)
To assess the safety of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2).
Number and frequency of AEs/SAEs throughout the study while on treatment
Measured: 26 weeks
Mean change in VAS
Score for muscle stiffness (myotonia severity) as self-reported by patients on a Visual Analog Scale (VAS)
Measured: 26 weeks
Mean change in MBS scores
Mean change in Myotonia Behavior Scale (MBS) scores
Measured: 26 weeks
Mean change in health-related quality of life
Mean change in health-related quality of life (measured by INQoL)
Measured: 26 weeks
Mean change in DM1-Activ-c scale (DM1 patients only)
Mean change in measure of activity and social participation (measured by a Rasch-built scale with a 0-100 interval range)
Measured: 26 weeks
Mean change in time to perform the 10-meter Walk Test
Mean change in time (seconds) to perform the 10-meter Walk Test (10mWT)
Measured: 26 weeks
Mean change in handgrip relaxation time
Mean change in maximal voluntary isometric contraction (MVIC) and relaxation time by Video-recording of Hand Opening Time (VHOT) functional evaluation
Measured: 26 weeks
Mean change in time to perform Timed-up and go (TUG) test
Mean change in time (seconds) to perform Timed-up and go (TUG) test
Measured: 26 Weeks
Mean change in health-related quality of life measured by EQ-5D
Mean change in health-related quality of life measured by EQ-5D (5-point scale)
Measured: 26 weeks
Mean change in Myotonic Dystrophy Health Index (MDHI) score (DM1 patients)
Mean change in Myotonic Dystrophy Health Index (MDHI) score (DM1 patients) as measured by patient's perceived health
Measured: 26 weeks
Mean change in Myotonic Dystrophy 2 Health Index (MD2HI) score (DM2 patients)
Mean change in Myotonic Dystrophy 2 Health Index (MDHI) score (DM2 patients) as measured by patient's perceived health
Measured: 26 weeks
Assess the safety of mexiletine PR by changes in ECG
Mean change in ECG (PR, QRS, QTc intervals, average HR) from baseline
Measured: 26 weeks
Assess the safety of mexiletine PR by AEs
Incidence of treatment emergent adverse events (TEAEs), treatment-related TEAEs, serious AEs, and patient discontinuation rate
Measured: 26 weeks
Assess the safety of mexiletine PR by standard clinical laboratory evaluations
Assess the safety of mexiletine PR by standard clinical laboratory evaluations as measured by hematology, chemistry, and urinalysis assessments from change in baseline
Measured: 26 weeks
Assess the safety of mexiletine PR by vital signs
Assess the safety of mexiletine PR by vital signs (pulse, respiration, body temperature, and blood pressure) from change in baseline
Measured: 26 weeks
Assess the safety of mexiletine PR by physical examinations
Assess the safety of mexiletine PR by change in baseline from physical examinations
Measured: 26 weeks
To assess the pharmacokinetics of mexiletine PR
To assess the pharmacokinetics of mexiletine PR as measured by PK parameter AUC0-t
Measured: 26 weeks
To assess the pharmacokinetics of mexiletine PR by AUC0-t
To assess the pharmacokinetics of mexiletine PR as measured by PK parameter AUC0-t (area under the concentration-time curve from time zero to last timepoint)
Measured: 26 weeks
To assess the pharmacokinetics of mexiletine PR by Cmax
To assess the pharmacokinetics of mexiletine PR as measured by PK parameter Cmax (maximum plasma concentration)
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2024
- Study start13 Feb 2025
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- A multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4-week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients complete/early terminate the study. In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo).
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 176 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lupin Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Lupin Atlantis Holdings S.A.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06523400
- Sponsor's protocol ID
- MEX-DM-302
- Status last verified
- Sep 2026
- First submitted
- 3 May 2024
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 (Phase 3)
- ClinicalTrials.gov ID
- NCT06523400
- Acronym / sponsor ID
- MEX-DM-302
- Conditions
- Myotonic Dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 176 participants (estimated)
- Lead sponsor
- Lupin Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Lupin Atlantis Holdings S.A.
- Study start
- 13 Feb 2025 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 26 Jul 2024 (actual)
- 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.
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)
Study website: https://myotonicdystrophystudy.com/
Detailed description
This is a multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4- week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients in total complete/early terminate the study.
In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo).
Study website: https://myotonicdystrophystudy.com/
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- A multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4-week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients complete/early terminate the study. In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo).
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo0 mg (matching sachet volumes to low, medium and high dose active drug) |
| Mexiletine prolonged-release (PR) | Active comparator | Drug: Mexiletine granules for prolonged-release oral suspensionMexiletine PR 167 mg (mexiletine HCl 200 mg) Mexiletine PR 333 mg (mexiletine HCl 400 mg) OR Mexiletine PR 500 mg (mexiletine HCl 600 mg) |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- DM1 or DM2 diagnosis confirmed genetically;
- Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)/legal guardian and written assent from the patient (if patient < 18 years of age);
- Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment;
- Male or non-pregnant female ≥16 years of age;
- Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg;
Key exclusion criteria (28 listed)
- Are pregnant or lactating;
- Have any one of the following medical conditions: uncontrolled diabetes mellitus, cancer other than skin cancer less than five years previously (e.g., basal-cell carcinoma (BCC) and squamous-cell carcinoma (SCC) of skin allowed), multiple sclerosis, seizure disorders, or other serious medical illness;
- Severe renal impairment (glomerular filtration rate (GFR) < 30 mL/min);
- Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;
- Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;
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 |
|---|---|---|
| Mexiletine granules for prolonged-release oral suspensionMexiletine PR | Drug | Mexiletine prolonged-release (PR) |
| PlaceboMatching Placebo | Drug | Placebo |
Primary outcome
To assess the efficacy of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2).
Time frame: 26 weeks
Handgrip relaxation time in DM1 patients by mean change in baseline of handgrip relaxation time (seconds) after maximal voluntary isometric contraction (MVIC)
19 secondary outcomes
To assess the safety of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2).
Time frame: 26 weeks
Mean change in VAS
Time frame: 26 weeks
Mean change in MBS scores
Time frame: 26 weeks
Mean change in health-related quality of life
Time frame: 26 weeks
Mean change in DM1-Activ-c scale (DM1 patients only)
Time frame: 26 weeks
Mean change in time to perform the 10-meter Walk Test
Time frame: 26 weeks
Mean change in handgrip relaxation time
Time frame: 26 weeks
Mean change in time to perform Timed-up and go (TUG) test
Time frame: 26 Weeks
Mean change in health-related quality of life measured by EQ-5D
Time frame: 26 weeks
Mean change in Myotonic Dystrophy Health Index (MDHI) score (DM1 patients)
Time frame: 26 weeks
Mean change in Myotonic Dystrophy 2 Health Index (MD2HI) score (DM2 patients)
Time frame: 26 weeks
Assess the safety of mexiletine PR by changes in ECG
Time frame: 26 weeks
Assess the safety of mexiletine PR by AEs
Time frame: 26 weeks
Assess the safety of mexiletine PR by standard clinical laboratory evaluations
Time frame: 26 weeks
Assess the safety of mexiletine PR by vital signs
Time frame: 26 weeks
Assess the safety of mexiletine PR by physical examinations
Time frame: 26 weeks
To assess the pharmacokinetics of mexiletine PR
Time frame: 26 weeks
To assess the pharmacokinetics of mexiletine PR by AUC0-t
Time frame: 26 weeks
To assess the pharmacokinetics of mexiletine PR by Cmax
Time frame: 26 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- 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.
7 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mexiletine granules for prolonged-release oral suspension in people with Myotonic Dystrophy, run by Lupin Ltd. at 7 sites in 6 countries.
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 11 inclusion and 28 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: looking for participants now, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom, Belgium, Denmark, Germany, Italy and 1 more country. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nikki Adetoro (1 443-447-4534, nikkiadetoro@lupin.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the efficacy of once daily mexiletine PR for the symptomatic treatment of myotonia in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). (time frame: 26 weeks). 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 24 Sep 2026.