Myotonic DystrophyPhase 3NCT04624750
Open Label Study in Adolescents and Children With Myotonic Disorders
A Phase 3 study testing Mexiletine in people with Myotonic Dystrophy, run by Lupin Ltd. at 1 site in France.
Trial summary
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- Mexiletine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- 3 Sep 2021to 8 Aug 2024
- Phase
- Phase 3
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female patients aged ≥ 6 and < 18 years who are able to comply with the study requirements
- A genetically confirmed diagnosis of NDM or DM (DM1or DM2)
- Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms)
- No significant cardiac abnormalities as determined by a cardiologist's assessment of the ECG and echocardiogram performed within 3 months prior to enrolment in the study. (If not done within 3 months before trial, electrocardiogram (ECG) and echocardiogram assessments will be performed at screening)
- No history of any significant liver disorder
- Patients receiving mexiletine treatment agree to stop treatment at least 7 days prior to initiation of treatment with Namuscla
Show all 10Show fewer
- Patients receiving other antimyotonic treatment agree to stop treatment for at least 7 times the half-life of respective drug
- Laboratory investigations for haematology, biochemistry, and urinalysis at screening are within the normal range, or showing no clinically relevant abnormal values, as judged by the Investigator.
- Female patients of childbearing potential must be using an acceptable form of birth control as determined by the Investigator (e.g., oral contraception, implantable, injectable/transdermal hormonal contraception, intrauterine device (IUD), barrier methods), tubal ligation or are practicing abstinence.
- Patients able to provide assent to study participation and a parent or legal guardian to sign the written informed consent prior to study entry.
Who cannot join
- Any contra-indication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC):
- Hypersensitivity to the active substance, or to any of the excipients
- Hypersensitivity to any local anaesthetic
- Ventricular tachyarrhythmia
- Complete heart block (i.e., third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 200 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
- QT interval > 450ms
Show all 20Show fewer
- Myocardial infarction (acute or past), or abnormal Q-waves
- Symptomatic coronary artery disease
- Heart failure with ejection fraction <50%
- Atrial tachyarrhythmia, fibrillation or flutter
- Sinus node dysfunction (including sinus rate < 50 bpm)
- Co-administration with medicinal products inducing torsades de pointes (class Ia, Ic, III antiarrhythmics): Co-administration of mexiletine and antiarrhythmics inducing torsades de pointesclass Ia: quinidine, procainamide, disopyramide, ajmaline; class Ic: encainide, flecainide, propafenone, moricizine; class III: amiodarone, sotalol, ibutilide, dofetilide, dronedarone, vernakalant) increases the risk of potentially lethal torsades de pointes.
- Co-administration with medicinal products with narrow therapeutic index
- Any other neurological or psychiatric condition that might affect the study assessments
- Any clinically significant illness, laboratory findings, ECG, or other clinical symptoms, which in the opinion of the Investigator could affect the patient's optimal participation in the study
- Strong inducer or inhibitor of CYP2D6 or CYP1A2 within 7 days prior to study drug administration
- Any concurrent illness, or medications which could affect the muscle function
- Seizure disorder, diabetes mellitus requiring treatment by insulin
- Pregnant or breastfeeding
- Concurrent participation in any other clinical trial.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-label, Non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children With Myotonic Disorders
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
Read the detailed description
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
Patients who meet the eligibility criteria will be enrolled stepwise, sequentially in 2 cohorts by age groups.
Cohort 1 - Adolescents aged 12 to <18 years, will be enrolled first. If no safety concerns are observed (based on data evaluation by the Data Safety Monitoring Board [DSMB]), and the dose for the age group 6 to <12 years is confirmed by PK model, enrolment for Cohort 2 will begin.
Cohort 2 - Children aged 6 to <12 years, will be enrolled. The overall treatment duration for each cohort will be approximately 56 days (8 weeks): a dose titration phase of 4 weeks and the maintenance phase of 4 weeks. The overall study duration would be approximately 22 months.
Dose titration phase: In this phase, patients will receive mexiletine starting at an age appropriate dose (as evaluated by the investigator and based on body weight) at a frequency of once a day. Dose will be up-titrated every 14 days based on tolerability of mexiletine up to a maximum of three-times a day as assessed by investigator.
Maintenance phase: During the maintenance phase, patients will continue to receive mexiletine at the best-tolerated dose from the titration phase for further 4 weeks. Following completion, all participants will be offered follow-up in PIP Study 7 (MEX-NM-303) (EudraCT: 2019-003758-97).
- Conditions
- Myotonic Dystrophy
- Being tested
- Mexiletine
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MexiletineDrug
Also called Namuscla ™
Patients will be enrolled sequentially into 2 cohorts. Cohort 1 - (patients aged 12 to < 18 years): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period.
Cohort 2 - (patients aged 6 to < 12 years,): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period.
Enrolment for Cohort 2 will begin after initial safety assessment of patients in Cohort 1 by the DSMB and no safety concerns are observed. The dose level for cohort 2 will be confirmed by PK modelling study.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 and 2 | Other | 7 patients aged 12 to < 18 years , inclusive in cohort-1 7 patients aged 6 to < 12 years, inclusive in cohort-2 Receives: Drug: Mexiletine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and frequency of adverse events (AEs)/serious adverse events (SAEs)
Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
Measured: Baseline to Day 56
Incidence of adverse events of special interest (AESI)
Incidence of adverse events of special interest (AESI)
Measured: Baseline to Day 56
Changes in ECG assessments from baseline
On resting ECG any alteration will be noted:
- Mild ECG abnormalities: PR interval ≥200 ms and QRS duration ≥100 ms
- Severe ECG abnormalities: PR interval ≥240 ms, QRS duration ≥120 ms, second or third degree AV block and a rhythm other than sinus
Measured: Baseline to Day 56
Efficacy of Namuscla treatment on the clinical outcomes based on the following functional evaluation mean change in Visual Analogue Scale (VAS) for muscle stiffness.
Mean change in Visual Analogue Scale (VAS) for muscle stiffness. The VAS is constructed as an absolute measure, with a 10 cm straight horizontal line having the endpoints "no stiffness at all" and "stiffness as worst possible". The patient's responses will be scored on the line to the nearest millimetre (a 100-point scale). (myotonia severity).
Measured: Baseline to Day 56
Efficacy of Namuscla treatment on the clinical outcomes(change from baseline to Days 14, 28, 42 and 56, respectively) based on the following functional evaluation
The score of handgrip myotonia as quantitatively measured using a commercially available grip dynamometer and computerised capture system. In standardised conditions (i.e. in a room at controlled temperature, after a definite period of rest), maximum voluntary contractions following forced right hand grip will be recorded and the time to relax from 90% to 5% of maximal force will be determined using automated analysis software
Measured: Baseline to Day 56
Secondary outcomes (15)
Mean change in VAS score for muscle pain, weakness and fatigue
The VAS is constructed as an absolute measure, with a 10 cm straight horizontal line having the endpoints "no stiffness at all" and "stiffness as worst possible". The patient's responses will be scored on the line to the nearest millimetre (a 100-point scale). The score of stiffness severity as self-reported by the patient on a 10-point VAS will be used for adolescents and children older than 8 years and will be summarised descriptively by visit
Measured: Baseline - Day 56
Clinical myotonia assessment for mean change in time to open the eyes
Mean change in time to open the eyes after forced eye closure as measured on a stopwatch (when eyelid myotonia present). Subjects will be asked to squeeze their eyes closed for 5 seconds then rapidly open them for 5 seconds then rapidly open. Five trials of each manoeuvre will be performed in sequence at each visit and the time measured on a stopwatch
Measured: Baseline - Day 56
Clinical myotonia assessment of clinical change in flexor myotonia
Clinical change in flexor myotonia (right hand flexor muscles). Subjects will be asked to make a tight fist for 5 seconds then rapidly open. Five trials of each manoeuvre will be performed in sequence at each visit and the time measured on a stopwatch
Measured: Baseline - Day 56
Clinical myotonia assessment of mean change in time to perform Timed-up and go (TUG) test
Mean change in time to perform Timed-up and go (TUG) test. Measures, in seconds, the time taken by an individual to stand up from a standard arm chair (approximate seat height of 46 cm, arm height 65 cm), walk a distance of 3 meters (approximately 10 feet), turn, walk back to the chair, and sit down.
Measured: Baseline - Day 56
Mean change in health-related quality-of-life as measured by the Paediatric Quality of Life (PedsQL) score
Mean change from baseline to Day 56, respectively in health-related quality-of-life as measured by the Paediatric Quality of Life (PedsQL) score. These multidimensional scales assess the frequency of health problems using generic and disease-specific approaches, respectively. Subjects and/or parent or proxies report a score of 0 to 4 (never to almost always) and questionnaires are tailored to age groups.
Measured: Baseline - Day 56
Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient
Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient, a parent or proxy and by the investigator at baseline and Day 56. Evaluated on a 4-point scale as very efficient, good, fair or poor.
Measured: Baseline - Day 56
Mean change in Myotonia Behaviour Scale (MBS) scores
Mean change from baseline to Day 56 in Myotonia Behaviour Scale (MBS) scores The Myotonia Behaviour Scale (MBS) (Hammaren et al., 2005) 0 No stiffness
- Some stiffness exists, which can be ignored
- Some stiffness exists, which can be ignored at times, but doesn't impair daily activities
- Stiffness exists, which demands a higher level of mental awareness when performing some duties and activities
- Severe stiffness exists, which impairs every duty and activity
- Incapacitating stiffness exists, which demands constant moving not to be totally locked up, with regard to movement
Measured: Baseline - Day 56
Changes in clinical laboratory values for laboratory safety assessments - Potassium
Changes in Potassium values from baseline to Day 56.
Measured: Baseline - Day 56
Acceptability of the capsule formulation with respect to the swallowability.
Acceptability of the capsule formulation with respect to the swallowability. It will be assessed by interviewing patients and their caregivers at Day 56.
Measured: Baseline - Day 56
Palatability of alternative administration
Palatability of alternative administration (capsule content with milk/juice or sprinkled on food) by 5-point facial hedonic scale correlated with 100-point Visual Analogue Scale (VAS) at each clinic visit
Measured: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Magnesium values
Changes in Magnesium values from baseline to Day 56.
Measured: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Sodium values
Changes in Sodium values from baseline to Day 56.
Measured: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Calcium values
Changes in Calcium values from baseline to Day 56.
Measured: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Chloride values
Changes in Chloride values from baseline to Day 56.
Measured: Baseline - Day 56
Mean change in Faces scale for muscle pain, weakness and fatigue
A Faces (or other symbol) scale for children aged 6 to 8 years will be used to measure the score of muscle stiffness (myotonia severity). Faces scale will be used to assess pain, weakness and tiredness in study participants with a 10 cm straight horizontal line having the endpoints "no [symptom] at all" and "[symptom] as worst possible"
Measured: Baseline - Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Nov 2020
- Study start3 Sep 2021
- Primary completion14 Jun 2024
- Study completion8 Aug 2024
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
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open-label, multi-centre, single arm, interventional, study to evaluate the safety, steady-state PK, and efficacy of mexiletine for the treatment of myotonia in paediatric population aged 6 to \< 18 years. The study comprises a screening period of 30 days, a dose-titration period of 4 weeks, and a maintenance period of 4 weeks. After last visit, all patients will be offered follow-up in clinical study MEX-NM-303 (PIP Study 7).
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lupin Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Christine Barnérias, MD, Principal investigator, Hopital universitaire Necker-Enfants Malades
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04624750
- Sponsor's protocol ID
- MEX-NM-301
- Status last verified
- Nov 2024
- First submitted
- 20 Oct 2020
- Last updated
- 25 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children With Myotonic Disorders
- ClinicalTrials.gov ID
- NCT04624750
- Acronym / sponsor ID
- MEX-NM-301
- Conditions
- Myotonic Dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Lupin Ltd.Industry (pharma, biotech, devices)
- Study start
- 3 Sep 2021 (actual)
- Primary completion
- 14 Jun 2024 (actual)
- Study completion
- 8 Aug 2024 (actual)
- First posted
- 12 Nov 2020 (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.
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
Detailed description
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
Patients who meet the eligibility criteria will be enrolled stepwise, sequentially in 2 cohorts by age groups.
Cohort 1 - Adolescents aged 12 to <18 years, will be enrolled first. If no safety concerns are observed (based on data evaluation by the Data Safety Monitoring Board [DSMB]), and the dose for the age group 6 to <12 years is confirmed by PK model, enrolment for Cohort 2 will begin.
Cohort 2 - Children aged 6 to <12 years, will be enrolled. The overall treatment duration for each cohort will be approximately 56 days (8 weeks): a dose titration phase of 4 weeks and the maintenance phase of 4 weeks. The overall study duration would be approximately 22 months.
Dose titration phase: In this phase, patients will receive mexiletine starting at an age appropriate dose (as evaluated by the investigator and based on body weight) at a frequency of once a day. Dose will be up-titrated every 14 days based on tolerability of mexiletine up to a maximum of three-times a day as assessed by investigator.
Maintenance phase: During the maintenance phase, patients will continue to receive mexiletine at the best-tolerated dose from the titration phase for further 4 weeks. Following completion, all participants will be offered follow-up in PIP Study 7 (MEX-NM-303) (EudraCT: 2019-003758-97).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open-label, multi-centre, single arm, interventional, study to evaluate the safety, steady-state PK, and efficacy of mexiletine for the treatment of myotonia in paediatric population aged 6 to < 18 years. The study comprises a screening period of 30 days, a dose-titration period of 4 weeks, and a maintenance period of 4 weeks. After last visit, all patients will be offered follow-up in clinical study MEX-NM-303 (PIP Study 7).
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 and 2 | Other | Drug: Mexiletine7 patients aged 12 to < 18 years , inclusive in cohort-1 7 patients aged 6 to < 12 years, inclusive in cohort-2 |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Male or female patients aged ≥ 6 and < 18 years who are able to comply with the study requirements
- A genetically confirmed diagnosis of NDM or DM (DM1or DM2)
- Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms)
- No significant cardiac abnormalities as determined by a cardiologist's assessment of the ECG and echocardiogram performed within 3 months prior to enrolment in the study. (If not done within 3 months before trial, electrocardiogram (ECG) and echocardiogram assessments will be performed at screening)
- No history of any significant liver disorder
Key exclusion criteria (20 listed)
- Any contra-indication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC):
- Any other neurological or psychiatric condition that might affect the study assessments
- Any clinically significant illness, laboratory findings, ECG, or other clinical symptoms, which in the opinion of the Investigator could affect the patient's optimal participation in the study
- Strong inducer or inhibitor of CYP2D6 or CYP1A2 within 7 days prior to study drug administration
- Any concurrent illness, or medications which could affect the muscle function
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 |
|---|---|---|
| MexiletineAlso: Namuscla ™Patients will be enrolled sequentially into 2 cohorts. Cohort 1 - (patients aged 12 to < 18 years): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period. Cohort 2 - (patients aged 6 to < 12 years,): approximately 8 weeks - 4 weeks of dose titration period + 4 weeks of maintenance period. Enrolment for Cohort 2 will begin after initial safety assessment of patients in Cohort 1 by the DSMB and no safety concerns are observed. The dose level for cohort 2 will be confirmed by PK modelling study. | Drug | Cohort 1 and 2 |
Primary outcome
Number and frequency of adverse events (AEs)/serious adverse events (SAEs)
Time frame: Baseline to Day 56
Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
Incidence of adverse events of special interest (AESI)
Time frame: Baseline to Day 56
Incidence of adverse events of special interest (AESI)
Changes in ECG assessments from baseline
Time frame: Baseline to Day 56
On resting ECG any alteration will be noted: * Mild ECG abnormalities: PR interval ≥200 ms and QRS duration ≥100 ms * Severe ECG abnormalities: PR interval ≥240 ms, QRS duration ≥120 ms, second or third degree AV block and a rhythm other than sinus
Efficacy of Namuscla treatment on the clinical outcomes based on the following functional evaluation mean change in Visual Analogue Scale (VAS) for muscle stiffness.
Time frame: Baseline to Day 56
Mean change in Visual Analogue Scale (VAS) for muscle stiffness. The VAS is constructed as an absolute measure, with a 10 cm straight horizontal line having the endpoints "no stiffness at all" and "stiffness as worst possible". The patient's responses will be scored on the line to the nearest millimetre (a 100-point scale). (myotonia severity).
Efficacy of Namuscla treatment on the clinical outcomes(change from baseline to Days 14, 28, 42 and 56, respectively) based on the following functional evaluation
Time frame: Baseline to Day 56
The score of handgrip myotonia as quantitatively measured using a commercially available grip dynamometer and computerised capture system. In standardised conditions (i.e. in a room at controlled temperature, after a definite period of rest), maximum voluntary contractions following forced right hand grip will be recorded and the time to relax from 90% to 5% of maximal force will be determined using automated analysis software
15 secondary outcomes
Mean change in VAS score for muscle pain, weakness and fatigue
Time frame: Baseline - Day 56
Clinical myotonia assessment for mean change in time to open the eyes
Time frame: Baseline - Day 56
Clinical myotonia assessment of clinical change in flexor myotonia
Time frame: Baseline - Day 56
Clinical myotonia assessment of mean change in time to perform Timed-up and go (TUG) test
Time frame: Baseline - Day 56
Mean change in health-related quality-of-life as measured by the Paediatric Quality of Life (PedsQL) score
Time frame: Baseline - Day 56
Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient
Time frame: Baseline - Day 56
Mean change in Myotonia Behaviour Scale (MBS) scores
Time frame: Baseline - Day 56
Changes in clinical laboratory values for laboratory safety assessments - Potassium
Time frame: Baseline - Day 56
Acceptability of the capsule formulation with respect to the swallowability.
Time frame: Baseline - Day 56
Palatability of alternative administration
Time frame: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Magnesium values
Time frame: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Sodium values
Time frame: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Calcium values
Time frame: Baseline - Day 56
Changes in clinical laboratory values from baseline to Day 56 for laboratory safety assessments - Changes in Chloride values
Time frame: Baseline - Day 56
Mean change in Faces scale for muscle pain, weakness and fatigue
Time frame: Baseline - Day 56
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2024.
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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.
Christine Barnérias, MD principal investigator
Hopital universitaire Necker-Enfants Malades
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mexiletine in people with Myotonic Dystrophy, run by Lupin Ltd. at 1 site in France.
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 10 inclusion and 20 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 25 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number and frequency of adverse events (AEs)/serious adverse events (SAEs) (time frame: Baseline to Day 56). The study lists 5 primary outcomes in all. 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 25 Nov 2024.