Phase II Trial of PRT-064040 Nasal Spray for the Treatment of Migraine in Adults

Multicenter, Randomized, Double-blind, Placebo-controlled, Dose-ranging Phase II Trial of PRT-064040 Nasal Spray for the Acute Treatment of Migraine

The purpose of this study is to learn about the efficacy and safety of PRT-064040 Nasal Spray versus placebo in the acute treatment of moderate or severe migraine.

Study Overview

• Status: Recruiting
• Conditions: Migraine
• Intervention / Treatment: Drug: PRT-064040 nasal spray; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 456
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Ke Yao; Phone Number: 86-028-60830544; Email: ke.yao@btyy.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100039
      • Recruiting
      • The First Medical Center of PLA General Hospital
      • Contact: Shengyuan Yu; Phone Number: 86-13501171068; Email: yusy1963@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Key Inclusion Criteria:

1. Male and female participants aged 18-75 years (inclusive);
2. BMI < 35 kg/m²;
3. Participant has at least 1-year history of migraines (with or without aura), consistent with a diagnosis according to the International Classification of Headache Disorder, 3rd Edition;
4. Age at first migraine onset < 50 years;
5. Migraine attacks, on average, lasting about 4-72 hours if untreated or treatment-resistant;
6. 2-8 attacks of moderate to severe intensity per month within the last 3 months；
7. Less than 15 days with headache (migraine or non-migraine) per month within the last 3 months；
8. Participants on prophylactic migraine medication are permitted to remain on therapy provided they have been on a stable dose for at least 3 months prior to screening visit and the dose is not expected to change until the EOT visit;
9. Patients diagnosed with chronic migraine who, owing to stable preventive therapy, have < 15 headache days and 2-8 attacks of moderate to severe intensity per month in the 3 months before screening, and who meet all other entry criteria, may be enrolled;
10. Able to comprehend and complete study questionnaires with electronic patient-reported outcome (e-PRO) application.

Exclusion Criteria:

Key Exclusion Criteria:

1. Participant with history of migraine with brainstem aura, retinal migraine, or hemiplegic migraine.

2. Participant with evidence of poorly controlled, unstable, or recently diagnosed cardiovascular or cardiometabolic disease, or prior history thereof, including:

   • Ischemic heart disease, coronary vasospasm, or cerebral ischemia diagnosed within 6 months before screening;
   • Previous stroke, transient ischemic attack, myocardial infarction, acute coronary syndrome, cardiac surgery, or percutaneous coronary intervention;
   • Abnormal 12-lead ECG at screening;
   • Poorly controlled diabetes mellitus or hypertension;
   • Poorly controlled or severe peripheral vascular disease.
3. Participant with any nasal structural abnormality, mucosal lesion, or disorder that could interfere with intranasal drug absorption.
4. Participant with dysgeusia, hypogeusia, or related taste disorders.
5. Participant with acute or chronic pain syndromes, or any other pain that in the investigator's opinion could confound study assessments.
6. Participant with history of regular use of ergotamine or triptans ≥ 10 days per month for ≥ 3 months; or regular use of non-narcotic analgesics (e.g., acetaminophen, NSAIDs, gabapentin) ≥ 15 days per month for ≥ 3 months.
7. Clinically relevant abnormal findings in hepatitis B surface antigen, hepatitis C antibody, Treponema pallidum antibody, or HIV antibody tests at screening.
8. Participant with history of alcohol or drug abuse within 1 year before screening, or positive urine drug screen at screening.
9. Pregnant or lactating women, or positive pregnancy test at screening.
10. Participant with known hypersensitivity to the investigational product or any of its excipients, or history of significant allergic reactions.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PRT-064040 nasal spray (dose 1); Participants administered a single intranasal dose of PRT-064040 nasal spray (dose 1) on occurrence of migraine that reached moderate or severe intensity within 45 days after randomization.	Drug: PRT-064040 nasal spray; A single dose of PRT-064040 nasal spray
Experimental: PRT-064040 nasal spray (dose 2); Participants administered a single intranasal dose of PRT-064040 nasal spray (dose 2) on occurrence of migraine that reached moderate or severe intensity within 45 days after randomization.	Drug: PRT-064040 nasal spray; A single dose of PRT-064040 nasal spray
Experimental: PRT-064040 nasal spray (dose 3); Participants administered a single intranasal dose of PRT-064040 nasal spray (dose 3) on occurrence of migraine that reached moderate or severe intensity within 45 days after randomization.	Drug: PRT-064040 nasal spray; A single dose of PRT-064040 nasal spray
Experimental: Placebo; Participants administered a single intranasal dose of placebo on occurrence of migraine that reached moderate or severe intensity within 45 days after randomization.	Drug: Placebo; A single dose of placebo matched to PRT-064040 nasal spray

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of Participants With Freedom From Pain at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants With Freedom From Most Bothersome Symptom (MBS) at 2 Hours Post-dose	2 hours post-dose

Secondary Outcome Measures

Outcome Measure	Time Frame
Percentage of Participants With Pain Relief at 15 Minutes Post-dose	15 minutes post-dose
Percentage of Participants With Pain Relief at 30 Minutes Post-dose	30 minutes post-dose
Percentage of Participants With Pain Relief at 60 Minutes Post-dose	60 minutes post-dose
Percentage of Participants With Pain Relief at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants Who Were Able to Function Normally at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants With Sustained Pain Relief From 2 Hours to 24 Post-dose	From 2 to 24 hours post-dose
Percentage of Participants With Sustained Pain Relief From 2 Hours to 48 Post-dose	From 2 to 48 hours post-dose
Percentage of Participants Who Were Able to Function Normally at 30 Minutes Post-dose	30 minutes post-dose
Percentage of Participants Who Were Able to Function Normally at 60 Minutes Post-dose	60 minutes post-dose
Percentage of Participants With Freedom From Phonophobia at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants With Freedom From Photophobia at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants With Freedom From Nausea at 2 Hours Post-dose	2 hours post-dose
Percentage of Participants With Sustained Pain Freedom From 2 Hours to 24 Post-dose	From 2 to 24 hours post-dose
Percentage of Participants With Sustained Pain Freedom From 2 Hours to 48 Post-dose	From 2 to 48 hours post-dose
Percentage of Participants With Pain Relapse From 2 to 48 Hours Post-dose	From 2 hours to 48 hours post-dose
Percentage of Participants With Rescue Medication Use Within 24 Hours Post-dose	Through 24 hours post-dose
Percentage of Participants Who Were Able to Function Normally at 15 Minutes Post-dose	15 minutes post-dose
The incidence and severity of adverse events	Through 7(±2) days post-dose

Collaborators and Investigators

• Sponsor: Sichuan Purity Pharmaceutical Technology Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): January 26, 2026
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: December 3, 2025
• First Submitted That Met QC Criteria: December 12, 2025
• First Posted (Actual): December 26, 2025

Study Record Updates

• Last Update Posted (Actual): January 29, 2026
• Last Update Submitted That Met QC Criteria: January 27, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Headache Disorders, Primary; Headache Disorders; Migraine Disorders
• Other Study ID Numbers: BT-PRT-064-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No