Myasthenia GravisPhase 2NCT04226170
Study of Pyridostigmine With Ondansetron in Subjects With Anti-AchR Positive Myasthenia Gravis
A Phase 2 study testing DAS-001 in people with Myasthenia Gravis, run by DAS-MG, Inc at 1 site in United States.
Trial summary
This is a phase II, single center, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- DAS-001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Nov 2023to 30 Apr 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female subjects aged 18 years old and up who have been diagnosed with anti-AchR antibody positive myasthenia gravis.
- Subjects can be either:
- currently taking pyridostigmine and after Lead-in Period experience dose limiting GI AEs related to pyridostigmine (must include nausea, vomiting or diarrhea) during Lead-in
- Subjects must be willing and able to complete a GI symptom diary within a consistent timeframe on a daily basis.
- Must be clinically stable in judgement of treating neurologists for past 3 months.
- Must be positive for acetylcholine receptor antibodies.
Show all 9Show fewer
- Subjects must be able to swallow liquid.
- Subjects must be in otherwise good health as determined by their medical history, physical examination, vital signs, and laboratory tests. A subject with a medical abnormality may be included only if the investigator or designee considers that the abnormality will not introduce significant additional risk to the subject's health or interfere with study objectives.
- Subjects must have signed an informed consent form indicating that they understand the purpose of, and procedures required for the study and are willing to participate in the study and comply with the study procedures and restrictions.
Who cannot join
- Any acute or chronic diseases which are associated with GI distress (such as nausea, vomiting, or diarrhea), which could interfere with the subjects' safety during the trial, expose them to undue risk, or interfere with the study objectives.
- History or presence of hepatic, or renal disease or other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs.
- History of substance abuse, known drug addiction, or positive test for drugs of abuse or alcohol.
- Patients currently using marijuana for any reason (medical or recreational).
- Known hypersensitivity to pyridostigmine, or to ondansetron or similar 5-HT3 serotonin receptor antagonists.
- ECG changes including QT interval prolongation and congenital long QT syndrome. Electrolyte abnormalities (e.g., hypokalemia or hypomagnesemia), congestive heart failure, bradyarrhythmia's or other medicinal products that lead to QT prolongation.
Show all 15Show fewer
- Treatment with drugs affecting peripheral cholinergic transmission within 1 month of study entry (with the exception of pyridostigmine).
- Subjects unlikely to co-operate during the study, and/or be questionably compliant in the opinion of the investigator.
- Patients currently being treated with narcotics.
- Patients being treated with aminoglycoside antibiotics, which are contraindicated in myasthenia gravis.
- Patients unable to be contacted in case of an emergency.
- Intake of an investigational drug within 30 days of study entry.
- Pregnancy and women of childbearing potential not willing to follow the birth control requirements as described in the informed consent or breastfeeding.
- History or presence of obstructive pulmonary disease or urinary obstruction (contraindication for pyridostigmine).
- This use of selective serotonin reuptake inhibitors (SSRIs).
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
Official title: A Phase II, Study to Evaluate the Safety and Tolerability of Pyridostigmine When Given With Ondansetron to Subjects With Anti-AchR Positive Myasthenia Gravis
This is a phase II, single center, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis.
Read the detailed description
Methodology: This is a phase II, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis.
Study Design: The clinical trial will be conducted over a 6-week treatment period with 2 groups.
- Group A: Patients currently taking pyridostigmine and experiencing pyridostigmine-related gastrointestinal (GI) adverse events (AEs) within the past 14 days.
- Group B: Patients not currently taking pyridostigmine due to a documented history of GI AEs.
Randomized to either the control (pyridostigmine+ placebo) or the test group (pyridostigmine + ondansetron) and treated for 6 weeks. Following enrolment, patients may (if needed) titrate up their pyridostigmine dose at the investigator's discretion each week to the highest dose deemed appropriate, tolerable and safe.
- Conditions
- Myasthenia Gravis
- Being tested
- DAS-001
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DAS-001Drug
ondansetron + pyridostigmine
| Study group | Role | What they receive |
|---|---|---|
| treatment | Active comparator | ondansetron + pyridostigmine Receives: Drug: DAS-001 |
| Placebo | Placebo comparator | placebo+ pyridostigmine Receives: Drug: DAS-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with change in the gastrointestinal (GI) side effects
difference in GI side effects as measured by the GSRS-self (Gastrointestinal System Rating Scale - self-administered)
Measured: 6 weeks
Secondary outcomes (6)
Number of participants with change in the side effects
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0" . Incidence and nature of adverse events
Measured: 6 weeks
Number of participants with change in in physical examine
physical examination changes General appearance,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Measured: 6 weeks
Number of participants with change in in clinical laboratory evaluations
changes in clinical laboratory evaluations (Creatinine, Potassium(K+),Sodium (Na+) , Chloride (Cl-), Magnesium (Mg++), Calcium, Inorganic phosphate, Glucose, Urea,Bilirubin (Total) ,Bilirubin (direct), AST, ALT, GGT, Alkaline phosphatase, Total Protein, Albumin,Hematocrit Hemoglobin Platelet count Red blood cell (RBC) count WBC count WBC differential Mean cell volume (MCV) Mean cell hemoglobin (MCH) MCH concentration (MCHC)
Measured: 6 weeks
Number of participants with change in Electrocardiography (ECG)
ECG (standard digital 12-lead in singlicate)
Measured: 6 weeks
Plasma concentrations of pyridostigmine
Cmax
Measured: 6 weeks
Plasma concentrations of ondansetron
Cmax
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start8 Nov 2023
- Primary completion30 Apr 2025
- Study completion30 Apr 2025
Why it stopped: lack of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies 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
- Single group
- Model description
- randomized to ondansetron + pyridostigmine or placebo+ pyridostigmine in a 3:1 ratio.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- DAS-MG, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04226170
- Sponsor's protocol ID
- DAS-001-001
- Status last verified
- May 2025
- First submitted
- 9 Jan 2020
- Last updated
- 11 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Study to Evaluate the Safety and Tolerability of Pyridostigmine When Given With Ondansetron to Subjects With Anti-AchR Positive Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT04226170
- Acronym / sponsor ID
- DAS-001-001
- Conditions
- Myasthenia Gravis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of enrollment
- Enrolment
- 2 participants (actual)
- Lead sponsor
- DAS-MG, IncIndustry (pharma, biotech, devices)
- Study start
- 8 Nov 2023 (actual)
- Primary completion
- 30 Apr 2025 (actual)
- Study completion
- 30 Apr 2025 (actual)
- First posted
- 13 Jan 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 a phase II, single center, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis.
Detailed description
Methodology: This is a phase II, randomized, double-blind, placebo-controlled, study in patients with a diagnosis of anti-AchR antibody positive myasthenia gravis.
Study Design: The clinical trial will be conducted over a 6-week treatment period with 2 groups.
- Group A: Patients currently taking pyridostigmine and experiencing pyridostigmine-related gastrointestinal (GI) adverse events (AEs) within the past 14 days.
- Group B: Patients not currently taking pyridostigmine due to a documented history of GI AEs.
Randomized to either the control (pyridostigmine+ placebo) or the test group (pyridostigmine + ondansetron) and treated for 6 weeks. Following enrolment, patients may (if needed) titrate up their pyridostigmine dose at the investigator's discretion each week to the highest dose deemed appropriate, tolerable and safe.
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
- randomized to ondansetron + pyridostigmine or placebo+ pyridostigmine in a 3:1 ratio.
| Arm | Type | Receives |
|---|---|---|
| treatment | Active comparator | Drug: DAS-001ondansetron + pyridostigmine |
| Placebo | Placebo comparator | Drug: DAS-001placebo+ pyridostigmine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male and female subjects aged 18 years old and up who have been diagnosed with anti-AchR antibody positive myasthenia gravis.
- Subjects can be either:
- currently taking pyridostigmine and after Lead-in Period experience dose limiting GI AEs related to pyridostigmine (must include nausea, vomiting or diarrhea) during Lead-in
- Subjects must be willing and able to complete a GI symptom diary within a consistent timeframe on a daily basis.
- Must be clinically stable in judgement of treating neurologists for past 3 months.
Key exclusion criteria (15 listed)
- Any acute or chronic diseases which are associated with GI distress (such as nausea, vomiting, or diarrhea), which could interfere with the subjects' safety during the trial, expose them to undue risk, or interfere with the study objectives.
- History or presence of hepatic, or renal disease or other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs.
- History of substance abuse, known drug addiction, or positive test for drugs of abuse or alcohol.
- Patients currently using marijuana for any reason (medical or recreational).
- Known hypersensitivity to pyridostigmine, or to ondansetron or similar 5-HT3 serotonin receptor antagonists.
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 |
|---|---|---|
| DAS-001ondansetron + pyridostigmine | Drug | Placebo, treatment |
Primary outcome
Number of participants with change in the gastrointestinal (GI) side effects
Time frame: 6 weeks
difference in GI side effects as measured by the GSRS-self (Gastrointestinal System Rating Scale - self-administered)
6 secondary outcomes
Number of participants with change in the side effects
Time frame: 6 weeks
Number of participants with change in in physical examine
Time frame: 6 weeks
Number of participants with change in in clinical laboratory evaluations
Time frame: 6 weeks
Number of participants with change in Electrocardiography (ECG)
Time frame: 6 weeks
Plasma concentrations of pyridostigmine
Time frame: 6 weeks
Plasma concentrations of ondansetron
Time frame: 6 weeks
Results and safety
The study was stopped early (reason given: lack of enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
- Individual participant dataSharing planned: No
Related trials for Myasthenia Gravis
Active, not recruitingPhase 3
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
RecruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis
Ocular Myasthenia Gravis
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingPhase 3
A Study to Evaluate The Long-Term Safety And Efficacy of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis
Ocular Myasthenia Gravis
Recruiting
Holistic Assessment and Remote Digital MONitoring of mYasthenia Gravis Via MyoSense 360
Myasthenia Gravis, Generalized
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing DAS-001 in people with Myasthenia Gravis, run by DAS-MG, Inc at 1 site 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 9 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: stopped early, as of the sponsor's last update on 11 Sep 2025. 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 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?
The study was stopped early (reason given: lack of enrollment). 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?
Number of participants with change in the gastrointestinal (GI) side effects (time frame: 6 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 11 Sep 2025.