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

Phase 2

Recruiting

• Conditions: Myasthenia Gravis
• Interventions: Drug: DAS-001

• Registration Number: NCT04226170
• Lead Sponsor: DAS-MG, Inc

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.

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 Timeline

• Study First Submitted: 2020-01-09
• Study First Submitted QC: 2020-01-09
• Study First Posted: 2020-01-13
• Study Start: 2023-11-08
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-10
• Last Update Posted: 2024-07-12
• Primary Completion: 2025-07-30
• Study Completion: 2026-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
treatment	DAS-001	ondansetron + pyridostigmine
Placebo	DAS-001	placebo+ pyridostigmine

Primary Outcome Measures

Name	Time	Method
Number of participants with change in the gastrointestinal (GI) side effects	6 weeks	difference in GI side effects as measured by the GSRS-self (Gastrointestinal System Rating Scale - self-administered)

Secondary Outcome Measures

Name	Time	Method
Number of participants with change in in clinical laboratory evaluations	6 weeks	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)
Number of participants with change in in physical examine	6 weeks	physical examination changes General appearance,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Number of participants with change in Electrocardiography (ECG)	6 weeks	ECG (standard digital 12-lead in singlicate)
Plasma concentrations of pyridostigmine	6 weeks	Cmax
Plasma concentrations of ondansetron	6 weeks	Cmax
Number of participants with change in the side effects	6 weeks	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0" . Incidence and nature of adverse events

Trial Locations

Locations (1)

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States