Trial Outcomes & Findings for AR101 Real-World Open-Label Extension Study (NCT NCT03337542)

Last Updated: 2021-11-02

Results Overview

Number of participants with treatment-emergent adverse events including serious adverse events during the overall study period (safety and tolerability)

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

243 participants

Primary outcome timeframe

Approximately 6 months

Results posted on

2021-11-02

Participant Flow

Participant milestones

Participant milestones
Measure	AR101 Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Overall Study STARTED	243
Overall Study Safety Population	242
Overall Study COMPLETED	222
Overall Study NOT COMPLETED	21

Reasons for withdrawal

Reasons for withdrawal
Measure	AR101 Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Overall Study Withdrew Consent	17
Overall Study Allergic Adverse Event	1
Overall Study Chronic /Recurrent GI Adverse Event/Symptoms	1
Overall Study Lost to Follow-up	1
Overall Study Unable to Participate in Study Due to Relocation	1

Baseline Characteristics

AR101 Real-World Open-Label Extension Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Age, Continuous	9 years n=93 Participants
Sex: Female, Male Female	84 Participants n=93 Participants
Sex: Female, Male Male	158 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	15 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	227 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	31 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	2 Participants n=93 Participants
Race (NIH/OMB) Black or African American	7 Participants n=93 Participants
Race (NIH/OMB) White	174 Participants n=93 Participants
Race (NIH/OMB) More than one race	25 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=93 Participants

PRIMARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101.

Number of participants with treatment-emergent adverse events including serious adverse events during the overall study period (safety and tolerability)

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Subjects with at least 1 Adverse Event (AE)	219 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Mild AE	176 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Moderate AE	43 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Severe AE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Life-threatening AE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Death AE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Subjects with at least 1 Serious Adverse Event (SAE)	1 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Mild SAE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Moderate SAE	1 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Severe SAE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Life-threatening SAE	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) Death SAE	0 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Premature Discontinuation of Dosing Due to Adverse Events	1 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Early Discontinuation of Dosing Due to Chronic/Recurrent GI Adverse Events	1 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101 Number of participants analyzed: subjects who discontinued dosing due to chronic/recurrent gastrointestinal adverse events.

Outcome measures

Outcome measures
Measure	AR101 n=1 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Percentage of Subjects With Chronic/Recurrent GI Adverse Events Resolving Before 2, Between 2 and 4, Between 4 and 12, and ≥ 12 Weeks After Discontinuation of Dosing < 2 weeks	1 Participants
Percentage of Subjects With Chronic/Recurrent GI Adverse Events Resolving Before 2, Between 2 and 4, Between 4 and 12, and ≥ 12 Weeks After Discontinuation of Dosing 2-4 weeks	0 Participants
Percentage of Subjects With Chronic/Recurrent GI Adverse Events Resolving Before 2, Between 2 and 4, Between 4 and 12, and ≥ 12 Weeks After Discontinuation of Dosing 4-12 weeks	0 Participants
Percentage of Subjects With Chronic/Recurrent GI Adverse Events Resolving Before 2, Between 2 and 4, Between 4 and 12, and ≥ 12 Weeks After Discontinuation of Dosing ≥ 12 weeks	0 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101. Threshold of allergic hypersensitivity AE reporting ≥ 5%

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Allergic Hypersensitivity Adverse Events Throat irritation	43 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Pruritus	39 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Abdominal pain	30 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Abdominal discomfort	28 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Urticaria	27 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Cough	24 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Anaphylactic reaction	21 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Nausea	19 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Vomiting	19 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Rash	17 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Oral pruritus	14 Participants
Number of Participants With Allergic Hypersensitivity Adverse Events Rhinorrhoea	14 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101.

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

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With of Anaphylaxis as Defined in the Protocol	21 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101.

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Epinephrine Use as Rescue Medication	19 Participants

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101.

Number of participants with accidental/nonaccidental ingestion of peanut (not AR101 or food challenge material) and other allergenic foods.

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Accidental/Nonaccidental Ingestion of Peanut and Other Allergenic Foods	33 Participants

SECONDARY outcome

Timeframe: Baseline, Maintenance Visit 1 (Wk 4), Maintenance Visit 2 (Wk 8), Maintenance Visit 3 (Wk 12), Maintenance Visit 4 (Wk 16), Maintenance Visit 5 (Wk 20), Maintenance Visit 6 (Wk 24), Early Discontinuation (14 days after last dose), Study Exit (~6 months)

Population: Safety population defined as all subjects who received at least 1 dose of AR101. The C-ACT questionnaire was not completed by all subjects and/or parents/caregivers at all protocol-defined points of collection

The C-ACT for subjects aged 4 to 11 years included 4 questions for the subject and 3 questions for the parent/caregiver; the total score (sum of 7 questions) ranged from 0 (worst control) to 27 (total control).

Outcome measures

Outcome measures
Measure	AR101 n=169 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Baseline	24 score on a scale Standard Deviation 2.58
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 1	24.4 score on a scale Standard Deviation 2.70
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 2	25.0 score on a scale Standard Deviation 1.97
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 3	25.0 score on a scale Standard Deviation 2.49
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 4	25.3 score on a scale Standard Deviation 1.88
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 5	24.8 score on a scale Standard Deviation 2.36
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Maintenance Visit 6	24.9 score on a scale Standard Deviation 2.31
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Early Discontinuation	26.4 score on a scale Standard Deviation 0.89
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Exit Visit	24.2 score on a scale Standard Deviation 3.15
Assessment of Asthma Control Using the Childhood Asthma Control Test (C-ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 4-11 Change from Baseline to Study Exit	0.5 score on a scale Standard Deviation 2.94

SECONDARY outcome

Population: Safety population defined as all subjects who received at least 1 dose of AR101. The ACT questionnaire was not completed by all subjects and/or parents/caregivers at all protocol-defined points of collection

The ACT for subjects aged 12 to 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

Outcome measures
Measure	AR101 n=73 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Baseline	23.3 score on a scale Standard Deviation 1.92
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 1	23.8 score on a scale Standard Deviation 1.70
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 2	23.9 score on a scale Standard Deviation 1.65
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 3	23.7 score on a scale Standard Deviation 2.32
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 4	23.3 score on a scale Standard Deviation 2.72
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 5	23.6 score on a scale Standard Deviation 2.14
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Maintenance Visit 6	23.8 score on a scale Standard Deviation 2.19
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Early Discontinuation	21.5 score on a scale Standard Deviation 4.95
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Exit Visit	23.5 score on a scale Standard Deviation 2.58
Assessment of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline, Every 4 Weeks, Early Discontinuation and Study Exit, Ages 12-17 Change from Baseline to Study Exit	0.3 score on a scale Standard Deviation 2.68

SECONDARY outcome

Timeframe: Approximately 6 months

Population: Safety population defined as all subjects who received at least 1 dose of AR101.

Outcome measures

Outcome measures
Measure	AR101 n=242 Participants Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Number of Participants With Adverse Events That Led to Early Withdrawal	1 Participants

Adverse Events

Treatment Arm Description

Serious events: 1 serious events

Other events: 219 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment Arm Description n=242 participants at risk Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Immune system disorders Anaphylactic reaction	0.41% 1/242 • 6 months

Other adverse events

Other adverse events
Measure	Treatment Arm Description n=242 participants at risk Subjects will receive maintenance dosing with AR101. Maintenance doses are provided in sachets, where each sachet contains 300 mg of peanut protein. Subjects are to ingest 300 mg orally once a day during maintenance. AR101: AR101 powder provided in sachets
Gastrointestinal disorders Abdominal discomfort	18.6% 45/242 • 6 months
Gastrointestinal disorders Vomiting	16.1% 39/242 • 6 months
Gastrointestinal disorders Abdominal pain	14.0% 34/242 • 6 months
Gastrointestinal disorders Nausea	12.4% 30/242 • 6 months
Gastrointestinal disorders Abdominal pain upper	6.2% 15/242 • 6 months
Gastrointestinal disorders Oral pruritis	6.2% 15/242 • 6 months
Respiratory, thoracic and mediastinal disorders Cough	19.4% 47/242 • 6 months
Respiratory, thoracic and mediastinal disorders Throat irritation	17.8% 43/242 • 6 months
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.5% 23/242 • 6 months
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	9.5% 23/242 • 6 months
Respiratory, thoracic and mediastinal disorders Rhinorrhea	9.5% 23/242 • 6 months
Respiratory, thoracic and mediastinal disorders Wheezing	7.0% 17/242 • 6 months
Respiratory, thoracic and mediastinal disorders Sneezing	5.0% 12/242 • 6 months
Infections and infestations Upper respiratory tract infection	17.4% 42/242 • 6 months
Infections and infestations Nasopharyngitis	7.9% 19/242 • 6 months
Infections and infestations Viral infection	5.4% 13/242 • 6 months
Skin and subcutaneous tissue disorders Pruritus	16.9% 41/242 • 6 months
Skin and subcutaneous tissue disorders Urticaria	13.6% 33/242 • 6 months
Skin and subcutaneous tissue disorders Rash	9.1% 22/242 • 6 months
General disorders Pyrexia	12.4% 30/242 • 6 months
Nervous system disorders Headache	12.8% 31/242 • 6 months
Immune system disorders Anaphylactic reaction	8.7% 21/242 • 6 months
Eye disorders Eye pruritus	5.4% 13/242 • 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