Study of Pyridostigmine With Ondansetron in Subjects With Anti-AchR Positive Myasthenia Gravis

NCT04226170 | Terminated Phase 2 FDA Drug

• 1 other identifier: DAS-001-001
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• 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)

  6 weeks

Secondary Outcomes (6)

• Number of participants with change in the side effects

  6 weeks

• Number of participants with change in in physical examine

  6 weeks

• Number of participants with change in in clinical laboratory evaluations

  6 weeks

• Number of participants with change in Electrocardiography (ECG)

  6 weeks

• Plasma concentrations of pyridostigmine

  6 weeks

Study Arms (2)

treatment

ACTIVE COMPARATOR

ondansetron + pyridostigmine

Drug: DAS-001

Placebo

PLACEBO COMPARATOR

placebo+ pyridostigmine

Drug: DAS-001

Interventions

DAS-001 DRUG

ondansetron + pyridostigmine

Placebo; treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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).

Sponsors & Collaborators

• DAS-MG, Inc: lead

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The only unblinded study member will be the pharmacist
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: randomized to ondansetron + pyridostigmine or placebo+ pyridostigmine in a 3:1 ratio.

Study Record Dates

• First Submitted: January 9, 2020
• First Posted: January 13, 2020
• Study Start: November 8, 2023
• Primary Completion: April 30, 2025
• Study Completion: April 30, 2025
• Last Updated: September 11, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)