Trial Outcomes & Findings for Safety and Tolerability of POD-DHE (INP104) in Migraine (STOP 301) (NCT NCT03557333)
NCT ID: NCT03557333
Last Updated: 2021-03-11
Results Overview
Number of participants with Serious Adverse Events (SAEs) whether or not related to study drug.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
360 participants
Primary outcome timeframe
From study enrollment up to Week 26 (for the 24-Week Treatment Group) and up to Week 54 (for the 52-WeekTreatment Group)
Results posted on
2021-03-11
Participant Flow
360 participants were enrolled and were dispensed INP104. Only those participants who dosed at least once with INP104 were included in the 24-Week Treatment Group, which was 354.
Participant milestones
| Measure |
24-Week Treatment Group
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 24-weeks.
|
|---|---|
|
Overall Study
STARTED
|
354
|
|
Overall Study
COMPLETED
|
262
|
|
Overall Study
NOT COMPLETED
|
92
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Tolerability of POD-DHE (INP104) in Migraine (STOP 301)
Baseline characteristics by cohort
| Measure |
24-Week Treatment Group
n=354 Participants
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 24-weeks.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
353 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
304 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
50 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
29 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
324 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
79 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
266 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
354 Participants
n=99 Participants
|
|
Mean number of migraines in 28-day baseline period
|
4.60 number of migraines
STANDARD_DEVIATION 2.313 • n=99 Participants
|
|
Mean duration of migraine history
|
19.5 years
STANDARD_DEVIATION 12.13 • n=99 Participants
|
PRIMARY outcome
Timeframe: From study enrollment up to Week 26 (for the 24-Week Treatment Group) and up to Week 54 (for the 52-WeekTreatment Group)Number of participants with Serious Adverse Events (SAEs) whether or not related to study drug.
Outcome measures
| Measure |
24-Week Treatment Group
n=354 Participants
Participants dosed with INP104 as needed for up to 24 weeks of treatment.
|
52-Week Treatment Group
n=73 Participants
This group is a subset of participants who completed 24 weeks of treatment and continued on study for up to 52 weeks of treatment.
|
|---|---|---|
|
Number of Participants With Serious Adverse Events (SAEs)
|
5 Participants
|
3 Participants
|
PRIMARY outcome
Timeframe: From first use of INP104 up to Week 26 (for the 24-Week Treatment Group) and up to Week 54 (for the 52-WeekTreatment Group)Number of participants with non-serious treatment emergent adverse events (AEs), whether or not related to study drug.
Outcome measures
| Measure |
24-Week Treatment Group
n=354 Participants
Participants dosed with INP104 as needed for up to 24 weeks of treatment.
|
52-Week Treatment Group
n=73 Participants
This group is a subset of participants who completed 24 weeks of treatment and continued on study for up to 52 weeks of treatment.
|
|---|---|---|
|
Number of Participants With Non-serious Treatment Emergent Adverse Events (AEs)
|
241 Participants
|
61 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 24 (for the 24-Week Treatment Group) and Baseline up to Week 52 (for the 52-WeekTreatment Group)Population: Only those participants with assessments at baseline and at the indicated time point have data analyzed in that row.
Mean change from baseline in Quantitative Scoring Scale for Evaluation of the Nasal Mucosa (QSS-NM) score, reported at designated intervals on study. This scale was scored by otolaryngologists during routine endoscopy of the upper nasal cavity of participants. A minimum score of 0 means no issues were detected. A maximum score of 34 indicates severe issues (worse outcome).
Outcome measures
| Measure |
24-Week Treatment Group
n=354 Participants
Participants dosed with INP104 as needed for up to 24 weeks of treatment.
|
52-Week Treatment Group
n=73 Participants
This group is a subset of participants who completed 24 weeks of treatment and continued on study for up to 52 weeks of treatment.
|
|---|---|---|
|
Change in Nasal Mucosa
Week 4
|
0.2 mean change in score on a scale
Standard Deviation 1.26
|
0.0 mean change in score on a scale
Standard Deviation 0.62
|
|
Change in Nasal Mucosa
Week 8
|
0.2 mean change in score on a scale
Standard Deviation 1.43
|
0.1 mean change in score on a scale
Standard Deviation 0.91
|
|
Change in Nasal Mucosa
Week 12
|
0.1 mean change in score on a scale
Standard Deviation 0.93
|
0.1 mean change in score on a scale
Standard Deviation 0.78
|
|
Change in Nasal Mucosa
Week 24
|
0.1 mean change in score on a scale
Standard Deviation 0.88
|
0.0 mean change in score on a scale
Standard Deviation 0.79
|
|
Change in Nasal Mucosa
Week 36
|
—
|
-0.1 mean change in score on a scale
Standard Deviation 0.68
|
|
Change in Nasal Mucosa
Week 52
|
—
|
-0.1 mean change in score on a scale
Standard Deviation 0.61
|
PRIMARY outcome
Timeframe: Baseline up to Week 24 (for the 24-Week Treatment Group) and Baseline up to Week 52 (for the 52-WeekTreatment Group)Population: Only those subjects with data at baseline and the indicated time point are included in the analysis for that row.
Mean change from baseline in olfactory function score, assessed using the University of Pennsylvania Smell Identification Test (UPSIT), and reported at designated intervals on study. The UPSIT is a 40 question scratch and sniff test of olfactory function. The minimum score of 0 indicates worst olfactory function, and the maximum score of 40 indicates the highest level of olfactory function detectable by the test.
Outcome measures
| Measure |
24-Week Treatment Group
n=354 Participants
Participants dosed with INP104 as needed for up to 24 weeks of treatment.
|
52-Week Treatment Group
n=73 Participants
This group is a subset of participants who completed 24 weeks of treatment and continued on study for up to 52 weeks of treatment.
|
|---|---|---|
|
Change in Olfactory Function
Week 12
|
-0.48 mean change in score
Standard Deviation 2.690
|
-0.18 mean change in score
Standard Deviation 2.573
|
|
Change in Olfactory Function
Week 24
|
-0.22 mean change in score
Standard Deviation 2.270
|
0.03 mean change in score
Standard Deviation 2.373
|
|
Change in Olfactory Function
Week 36
|
—
|
-0.13 mean change in score
Standard Deviation 2.930
|
|
Change in Olfactory Function
Week 52
|
—
|
-0.80 mean change in score
Standard Deviation 2.710
|
Adverse Events
24-Week Treatment Group
Serious events: 5 serious events
Other events: 241 other events
Deaths: 0 deaths
52-Week Treatment Group
Serious events: 3 serious events
Other events: 61 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
24-Week Treatment Group
n=354 participants at risk
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 24 weeks.
|
52-Week Treatment Group
n=73 participants at risk
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 52 weeks. Participants in this treatment group are subset of those included in the 24-Week Treatment Group.
|
|---|---|---|
|
Eye disorders
Visual impairment
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.00%
0/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
1.4%
1/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Nervous system disorders
Status migrainosus
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
1.4%
1/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Reproductive system and breast disorders
Ovarian mass
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
1.4%
1/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.28%
1/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
Other adverse events
| Measure |
24-Week Treatment Group
n=354 participants at risk
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 24 weeks.
|
52-Week Treatment Group
n=73 participants at risk
Participants in this group dosed at least once with INP104, and treated their migraine headaches as needed up to 2 times in 24 hours or up to 3 times per week with INP104, for up to 52 weeks. Participants in this treatment group are subset of those included in the 24-Week Treatment Group.
|
|---|---|---|
|
Ear and labyrinth disorders
Ear discomfort
|
0.85%
3/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Gastrointestinal disorders
Nausea
|
7.9%
28/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
11.0%
8/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Gastrointestinal disorders
Vomiting
|
2.8%
10/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
4.1%
3/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Upper respiratory tract infection
|
10.7%
38/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
20.5%
15/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Nasopharyngitis
|
8.5%
30/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
13.7%
10/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Sinusitis
|
3.7%
13/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
5.5%
4/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Urinary tract infection
|
2.5%
9/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
6.8%
5/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Influenza
|
1.7%
6/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Pharyngitis streptococcal
|
2.0%
7/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Acute sinusitis
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Bronchitis
|
1.4%
5/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Infections and infestations
Viral upper respiratory tract infection
|
2.0%
7/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Injury, poisoning and procedural complications
Product package associated injury
|
1.7%
6/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
5.5%
4/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Injury, poisoning and procedural complications
Foot fracture
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
4.1%
3/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Investigations
Olfactory test abnormal
|
2.5%
9/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
9.6%
7/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.85%
3/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.1%
4/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Nervous system disorders
Dizziness
|
2.3%
8/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
5.5%
4/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Nervous system disorders
Paraesthesia
|
0.85%
3/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Nervous system disorders
Somnolence
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Product Issues
Product taste abnormal
|
5.1%
18/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
4.1%
3/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
59/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
24.7%
18/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal discomfort
|
5.4%
19/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
6.8%
5/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
3.1%
11/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
0.00%
0/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.8%
10/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
1.4%
1/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.85%
3/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
4.1%
3/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal mucosal disorder
|
1.7%
6/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Social circumstances
Menopause
|
0.56%
2/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
2.7%
2/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
|
Vascular disorders
Hypertension
|
1.1%
4/354 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
4.1%
3/73 • Week 0 to Week 24 (for the 24-Week Treatment Group) and Week 0 to Week 52 (for the 52-Week Treatment Group)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place