Trial Outcomes & Findings for Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children (RAMSES) (NCT NCT03126227)
NCT ID: NCT03126227
Last Updated: 2021-11-02
Results Overview
Frequency of Treatment Emergent Adverse Events, including Serious Adverse Events, during the overall study period. Treatment-emergent adverse events were defined as adverse events with onset after the first dose of study product.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
506 participants
Primary outcome timeframe
Approximately 6 months
Results posted on
2021-11-02
Participant Flow
Participant milestones
| Measure |
AR101 Powder Provided in Capsules
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Overall Study
STARTED
|
338
|
168
|
|
Overall Study
COMPLETED
|
260
|
158
|
|
Overall Study
NOT COMPLETED
|
78
|
10
|
Reasons for withdrawal
| Measure |
AR101 Powder Provided in Capsules
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Overall Study
Adverse Event
|
43
|
4
|
|
Overall Study
Death
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Physician Decision
|
2
|
0
|
|
Overall Study
Protocol Violation
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
19
|
3
|
|
Overall Study
Discontinued due to early termination and continuation in follow-on study due to study closure
|
8
|
1
|
|
Overall Study
Lack of dosing compliance
|
2
|
0
|
|
Overall Study
Did not meet eligibility criteria subsequent to enrollment
|
2
|
0
|
|
Overall Study
Scheduling conflict
|
1
|
0
|
Baseline Characteristics
Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children (RAMSES)
Baseline characteristics by cohort
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
Total
n=505 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.0 years
n=93 Participants
|
9.5 years
n=4 Participants
|
9.0 years
n=27 Participants
|
|
Age, Customized
4-11 years
|
226 Participants
n=93 Participants
|
114 Participants
n=4 Participants
|
340 Participants
n=27 Participants
|
|
Age, Customized
12-17 years
|
111 Participants
n=93 Participants
|
54 Participants
n=4 Participants
|
165 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
119 Participants
n=93 Participants
|
66 Participants
n=4 Participants
|
185 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
218 Participants
n=93 Participants
|
102 Participants
n=4 Participants
|
320 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
19 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
27 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
318 Participants
n=93 Participants
|
159 Participants
n=4 Participants
|
477 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
39 Participants
n=93 Participants
|
25 Participants
n=4 Participants
|
64 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
2 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
11 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
245 Participants
n=93 Participants
|
113 Participants
n=4 Participants
|
358 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
33 Participants
n=93 Participants
|
11 Participants
n=4 Participants
|
44 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=93 Participants
|
14 Participants
n=4 Participants
|
21 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment.
Frequency of Treatment Emergent Adverse Events, including Serious Adverse Events, during the overall study period. Treatment-emergent adverse events were defined as adverse events with onset after the first dose of study product.
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Subjects with at least 1 AE
|
334 Participants
|
159 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Mild AE
|
171 Participants
|
110 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Moderate AE
|
151 Participants
|
47 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Severe AE
|
11 Participants
|
1 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Life Threatening AE
|
1 Participants
|
0 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Death
|
0 Participants
|
1 Participants
|
|
Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events
Subjects with at least 1 serious adverse event
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Premature Discontinuation of Dosing Due to Adverse Events
|
41 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Premature Discontinuation of Dosing Due to Chronic/Recurrent Gastrointestinal Adverse Events
|
20 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment. Number of participants analyzed: subjects who discontinued dosing due to chronic/recurrent gastrointestinal adverse events. None of the subjects in Placebo group discontinued dosing due to chronic/recurrent gastrointestinal adverse events.
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=20 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing
< 2 weeks
|
17 Participants
|
0 Participants
|
|
Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing
2-4 weeks
|
2 Participants
|
0 Participants
|
|
Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing
4-12 weeks
|
0 Participants
|
0 Participants
|
|
Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing
≥ 12 weeks
|
0 Participants
|
0 Participants
|
|
Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing
Unknown
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Throat irritation
|
1434 number of events
|
51 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Abdominal pain
|
1184 number of events
|
83 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Abdominal discomfort
|
911 number of events
|
65 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Nausea
|
557 number of events
|
26 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Pruritus
|
510 number of events
|
106 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Abdominal pain upper
|
374 number of events
|
31 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Oral pruritus
|
361 number of events
|
6 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Tongue pruritus
|
305 number of events
|
5 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Sneezing
|
296 number of events
|
41 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Vomiting
|
266 number of events
|
14 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Urticaria
|
251 number of events
|
117 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Cough
|
195 number of events
|
64 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Lip pruritus
|
170 number of events
|
0 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Rhinorrhoea
|
164 number of events
|
51 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Paraesthesia oral
|
159 number of events
|
39 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Rash
|
139 number of events
|
62 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Nasal congestion
|
127 number of events
|
47 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Throat tightness
|
100 number of events
|
3 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Headache
|
56 number of events
|
10 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Diarrhoea
|
52 number of events
|
15 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Erythema
|
51 number of events
|
20 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Anaphylactic reaction
|
44 number of events
|
9 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Wheezing
|
41 number of events
|
6 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Flushing
|
37 number of events
|
10 number of events
|
|
Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment
Eye pruritus
|
34 number of events
|
13 number of events
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Anaphylaxis is likely when any 1 of the 3 following sets of criteria is fulfilled: 1. Acute onset of an illness (minutes to hours) with involvement of: (a) Skin/mucosal tissue (eg, generalized hives, itch/flush, swollen lips/tongue/uvula); AND (b) Airway compromise (eg, dyspnea, stridor, wheeze/bronchospasm, hypoxia, reduced PEFR); AND/OR (c) Reduced BP or associated symptoms (eg, hypotonia, syncope, incontinence). 2. Two or more of the following that occur rapidly after exposure to the allergen (minutes to hours): (a) Skin/mucosal tissue; (b) Airway compromise; (c) Reduced BP or associated symptoms; (d) Persistent GI symptoms (eg, nausea, vomiting, crampy abdominal pain). 3. Reduced BP after exposure to the allergen (minutes to hours). Infants and children: low systolic BP (age-specific) or \> 30% drop in systolic BP; Adults: systolic BP \< 90 mm Hg or \> 30% drop from their baseline.
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Anaphylaxis as Defined in the Protocol
|
36 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Epinephrine Use as Rescue Medication
|
37 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Accidental Ingestion of Peanut and Other Allergenic Foods
|
26 Participants
|
31 Participants
|
SECONDARY outcome
Timeframe: Baseline, Interim dose (approx. 10 weeks), End of Up-Dosing (20 to 40 weeks), Study Exit (14 days after End of Up-Dosing, usually approx. 6 months)Population: Safety population defined as all subjects who received at least 1 dose of randomized study treatment (included subjects ages 4-11 only). The ACT questionnaire was not completed by all subjects and/or parents at all protocol-defined points of collection.
The Asthma Control Test (ACT) Questionnaire ACT for subjects aged 4-11 years included 4 questions for the subject and 3 questions for the parent; the total score (sum of 7 questions) ranged from 0 (worst control) to 27 (total control)
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=226 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=114 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Baseline
|
23.9 Change in ACT total score from baseline
Standard Deviation 2.57
|
23.7 Change in ACT total score from baseline
Standard Deviation 2.93
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Interim (80 mg) visit
|
24.2 Change in ACT total score from baseline
Standard Deviation 2.52
|
23.7 Change in ACT total score from baseline
Standard Deviation 3.47
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
End of Up-Dosing (300 mg) Visit
|
24.2 Change in ACT total score from baseline
Standard Deviation 2.63
|
23.8 Change in ACT total score from baseline
Standard Deviation 3.55
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Study Exit
|
24.0 Change in ACT total score from baseline
Standard Deviation 3.03
|
24.5 Change in ACT total score from baseline
Standard Deviation 3.50
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Change from Baseline to Interim (80 mg) Visit
|
0.3 Change in ACT total score from baseline
Standard Deviation 2.54
|
0.2 Change in ACT total score from baseline
Standard Deviation 2.93
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Change from Baseline to End of Up-Dosing (300 mg) Visit
|
0.5 Change in ACT total score from baseline
Standard Deviation 3.08
|
0.4 Change in ACT total score from baseline
Standard Deviation 2.61
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11
Change from Baseline to Early Discontinuation / Study Exit
|
0.3 Change in ACT total score from baseline
Standard Deviation 3.29
|
0.7 Change in ACT total score from baseline
Standard Deviation 2.76
|
SECONDARY outcome
Timeframe: Baseline, Interim dose (approx. 10 weeks), End of Up-Dosing (20 to 40 weeks), Study Exit (14 days after End of Up-Dosing, usually approx. 6 months)Population: Safety population defined as all subjects who received at least 1 dose of randomized study treatment (included subjects ages 12-17 only). The ACT questionnaire was not completed by all subjects and/or parents at all protocol-defined points of collection.
The Asthma Control Test (ACT) Questionnaire ACT for subjects aged 12-17 years consisted of 5 questions, and the total score (sum of 5 questions) ranged from 5 (worst control) to 25 (total control).
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=111 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=54 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Change from Baseline to Interim (80 mg) Visit
|
0.3 Change in ACT total score from baseline
Standard Deviation 2.72
|
0.2 Change in ACT total score from baseline
Standard Deviation 2.93
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Change from Baseline to End of Up-Dosing (300 mg) Visit
|
0.9 Change in ACT total score from baseline
Standard Deviation 1.92
|
0.5 Change in ACT total score from baseline
Standard Deviation 3.10
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Baseline
|
22.8 Change in ACT total score from baseline
Standard Deviation 2.40
|
22.9 Change in ACT total score from baseline
Standard Deviation 3.05
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Interim (80 mg) visit
|
23.0 Change in ACT total score from baseline
Standard Deviation 2.69
|
23.1 Change in ACT total score from baseline
Standard Deviation 2.90
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
End of Up-Dosing (300 mg) Visit
|
23.7 Change in ACT total score from baseline
Standard Deviation 1.73
|
23.2 Change in ACT total score from baseline
Standard Deviation 2.13
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Study Exit
|
23.8 Change in ACT total score from baseline
Standard Deviation 1.74
|
22.6 Change in ACT total score from baseline
Standard Deviation 2.63
|
|
Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17
Change from Baseline to Early Discontinuation / Study Exit
|
0.7 Change in ACT total score from baseline
Standard Deviation 1.68
|
-0.2 Change in ACT total score from baseline
Standard Deviation 2.71
|
SECONDARY outcome
Timeframe: Approximately 6 monthsPopulation: Safety population defined as all subjects who received at least 1 dose of randomized study treatment
Outcome measures
| Measure |
AR101 Powder Provided in Capsules
n=337 Participants
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 Participants
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Frequency of Adverse Events That Led to Early Withdrawal
|
36 Participants
|
5 Participants
|
Adverse Events
AR101 Powder Provided in Capsules
Serious events: 2 serious events
Other events: 334 other events
Deaths: 0 deaths
Placebo Powder
Serious events: 2 serious events
Other events: 159 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
AR101 Powder Provided in Capsules
n=337 participants at risk
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 participants at risk
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute lymphocytic leukemia
|
0.30%
1/337 • Approximately 6 months
|
0.00%
0/168 • Approximately 6 months
|
|
Infections and infestations
Pneumonia mycoplasma
|
0.30%
1/337 • Approximately 6 months
|
0.00%
0/168 • Approximately 6 months
|
|
Infections and infestations
Appendicitis
|
0.00%
0/337 • Approximately 6 months
|
0.60%
1/168 • Approximately 6 months
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.00%
0/337 • Approximately 6 months
|
0.60%
1/168 • Approximately 6 months
|
Other adverse events
| Measure |
AR101 Powder Provided in Capsules
n=337 participants at risk
Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol
AR101: AR101 powder provided in capsules
|
Placebo Powder
n=168 participants at risk
Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product.
Placebo: Placebo powder provided in capsules
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
52.5%
177/337 • Approximately 6 months
|
20.2%
34/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Abdominal discomfort
|
40.7%
137/337 • Approximately 6 months
|
16.1%
27/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Vomiting
|
38.6%
130/337 • Approximately 6 months
|
16.7%
28/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Nausea
|
33.8%
114/337 • Approximately 6 months
|
11.9%
20/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Abdominal pain upper
|
22.3%
75/337 • Approximately 6 months
|
14.3%
24/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Oral pruritus
|
14.8%
50/337 • Approximately 6 months
|
3.0%
5/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Paraesthesia oral
|
13.6%
46/337 • Approximately 6 months
|
5.4%
9/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Diarrhoea
|
12.8%
43/337 • Approximately 6 months
|
11.3%
19/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Lip pruritus
|
10.7%
36/337 • Approximately 6 months
|
0.60%
1/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Tongue pruritus
|
10.1%
34/337 • Approximately 6 months
|
1.8%
3/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Dyspepsia
|
7.7%
26/337 • Approximately 6 months
|
1.8%
3/168 • Approximately 6 months
|
|
Gastrointestinal disorders
Lip swelling
|
6.2%
21/337 • Approximately 6 months
|
3.0%
5/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
46.0%
155/337 • Approximately 6 months
|
17.9%
30/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
32.6%
110/337 • Approximately 6 months
|
22.6%
38/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
23.7%
80/337 • Approximately 6 months
|
18.5%
31/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
19.9%
67/337 • Approximately 6 months
|
12.5%
21/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
19.3%
65/337 • Approximately 6 months
|
17.3%
29/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
14.5%
49/337 • Approximately 6 months
|
18.5%
31/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
12.5%
42/337 • Approximately 6 months
|
6.5%
11/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Throat tightness
|
10.1%
34/337 • Approximately 6 months
|
1.2%
2/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
7.4%
25/337 • Approximately 6 months
|
1.2%
2/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
5.6%
19/337 • Approximately 6 months
|
7.7%
13/168 • Approximately 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
4.2%
14/337 • Approximately 6 months
|
5.4%
9/168 • Approximately 6 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
37.1%
125/337 • Approximately 6 months
|
24.4%
41/168 • Approximately 6 months
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
26.4%
89/337 • Approximately 6 months
|
21.4%
36/168 • Approximately 6 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.9%
40/337 • Approximately 6 months
|
16.1%
27/168 • Approximately 6 months
|
|
Skin and subcutaneous tissue disorders
Erythema
|
6.8%
23/337 • Approximately 6 months
|
6.0%
10/168 • Approximately 6 months
|
|
Infections and infestations
Upper respiratory tract infection
|
22.3%
75/337 • Approximately 6 months
|
29.8%
50/168 • Approximately 6 months
|
|
Infections and infestations
Nasopharyngitis
|
11.0%
37/337 • Approximately 6 months
|
13.7%
23/168 • Approximately 6 months
|
|
Infections and infestations
Influenza
|
8.6%
29/337 • Approximately 6 months
|
5.4%
9/168 • Approximately 6 months
|
|
Infections and infestations
Viral infection
|
6.5%
22/337 • Approximately 6 months
|
7.1%
12/168 • Approximately 6 months
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.9%
20/337 • Approximately 6 months
|
6.0%
10/168 • Approximately 6 months
|
|
Infections and infestations
Gastroenteritis viral
|
4.7%
16/337 • Approximately 6 months
|
8.3%
14/168 • Approximately 6 months
|
|
General disorders
Pyrexia
|
18.7%
63/337 • Approximately 6 months
|
17.3%
29/168 • Approximately 6 months
|
|
General disorders
Chest discomfort
|
5.9%
20/337 • Approximately 6 months
|
0.60%
1/168 • Approximately 6 months
|
|
Nervous system disorders
Headache
|
27.0%
91/337 • Approximately 6 months
|
26.2%
44/168 • Approximately 6 months
|
|
Eye disorders
Eye pruritus
|
7.4%
25/337 • Approximately 6 months
|
7.1%
12/168 • Approximately 6 months
|
|
Immune system disorders
Anaphylactic reaction
|
10.7%
36/337 • Approximately 6 months
|
5.4%
9/168 • Approximately 6 months
|
|
Ear and labyrinth disorders
Ear pruritus
|
5.9%
20/337 • Approximately 6 months
|
1.8%
3/168 • Approximately 6 months
|
|
Vascular disorders
Flushing
|
7.7%
26/337 • Approximately 6 months
|
6.5%
11/168 • Approximately 6 months
|
Additional Information
Director of Regulatory Affairs
Aimmune Therapeutics, Inc.
Phone: 650-409-5164
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee * Institutions cannot publish until the multi-center sponsor publication is published * Or, institutions cannot publish until 18 months after study completion * And Sponsor review of any publications is required prior to any institution publications according to contractual agreements
- Publication restrictions are in place
Restriction type: OTHER