Trial Outcomes & Findings for The Safety and Efficacy of Sublingual/Oral Immunotherapy for the Treatment of Milk Protein Allergy (NCT NCT00732654)
NCT ID: NCT00732654
Last Updated: 2017-05-15
Results Overview
Cow's milk specific IgE was measured at baseline and after therapy (kUa/L)
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Change from baseline to after therapy (up to 18 months)
Results posted on
2017-05-15
Participant Flow
33 patients underwent screening Double-Blinded Placebo-Controlled Food Challenges (DBPCFCs). Two subjects failed screening because they tolerated at least 2.5g at the initial DBPCFC. One subject declined to continue in the study after the food challenge. 30 subjects were randomized.
Participant milestones
| Measure |
SLIT
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
SLIT/OIT B
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
SLIT/ OIT A
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
|---|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
10
|
|
Overall Study
COMPLETED
|
10
|
9
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The Safety and Efficacy of Sublingual/Oral Immunotherapy for the Treatment of Milk Protein Allergy
Baseline characteristics by cohort
| Measure |
OITA Group
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
OITB Group
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
SLIT Group
n=10 Participants
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
30 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
10 participants
n=7 Participants
|
10 participants
n=5 Participants
|
30 participants
n=4 Participants
|
|
History of anaphylaxis to Cow's Milk (CM)
|
6 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Other Food Allergies
|
6 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Asthma
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Atopic Dermatitis
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Change from baseline to after therapy (up to 18 months)Population: Data was not collected for one participant in the SLIT/OIT A group.
Cow's milk specific IgE was measured at baseline and after therapy (kUa/L)
Outcome measures
| Measure |
SLIT
n=10 Participants
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 7mg/day: Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
SLIT/OIT B
n=9 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 1000mg/day: Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
|
SLIT/ OIT A
n=9 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 2000 mg/day: Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
|
|---|---|---|---|
|
Change in CM-specific Immunogloblin E (IgE)
|
0.8 kUa/L
Interval -390.0 to 10.0
|
-13.5 kUa/L
Interval -170.8 to 471.7
|
-9.4 kUa/L
Interval -49.1 to 42.1
|
PRIMARY outcome
Timeframe: Change from baseline to after therapy (up to 18 months)Cow's milk specific IgG4 was measured at baseline and after therapy (kUa/L)
Outcome measures
| Measure |
SLIT
n=10 Participants
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 7mg/day: Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
SLIT/OIT B
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 1000mg/day: Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
|
SLIT/ OIT A
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 2000 mg/day: Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
|
|---|---|---|---|
|
Change in CM-specific Immunoglobulin G4 (IgG4)
|
9.9 kUa/L
Interval 0.4 to 705.7
|
157.2 kUa/L
Interval 12.6 to 2415.7
|
31.8 kUa/L
Interval -16.9 to 798.5
|
PRIMARY outcome
Timeframe: Change from baseline to after therapy (up to 18 months)Allergen provoked skin test (mm)
Outcome measures
| Measure |
SLIT
n=10 Participants
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 7mg/day: Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
SLIT/OIT B
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 1000mg/day: Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
|
SLIT/ OIT A
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 2000 mg/day: Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
|
|---|---|---|---|
|
Change in End Point Skin Test
|
-3.1 mm
Interval -6.1 to 2.0
|
-3 mm
Interval -10.9 to 2.3
|
-2.5 mm
Interval -5.1 to 0.9
|
PRIMARY outcome
Timeframe: Change from baseline to after therapy (up to 18 months)mg CM protein
Outcome measures
| Measure |
SLIT
n=10 Participants
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 7mg/day: Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
SLIT/OIT B
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 1000mg/day: Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
|
SLIT/ OIT A
n=10 Participants
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Protein Extract Immunotherapy goal of 4mg/day: Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
Milk Powder Immunotherapy goal dose 2000 mg/day: Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
|
|---|---|---|---|
|
Oral Food Challenge Threshold (OFC) Threshold
|
2458 mg
Interval 388.9 to 8139.0
|
8089 mg
Interval 6138.9 to 8130.0
|
7989 mg
Interval 4289.0 to 8129.0
|
Adverse Events
OITA Group
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
OITB Group
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
SLIT Group
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
OITA Group
n=10 participants at risk
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 2000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal dose of 2000mg/day given for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
OITB Group
n=10 participants at risk
These subjects will start with a dose escalation of the milk protein extract given sublingually, and then will switch to milk powder given orally and will undergo a dose escalation for a goal of 1000 mg. After dose escalation, they will continue on the oral daily maintenance dose for approximately one year.
Milk Powder Immunotherapy : Milk powder given orally in escalating doses with a goal of 1000mg/day for approximately 1 1/2 years.
Milk Protein Extract Immunotherapy : Sublingual extract given daily in escalating doses with goal of 4 mg/day for approximately 20 weeks.
|
SLIT Group
n=10 participants at risk
These subjects will have a dose escalation of the milk protein extract given sublingually. After dose escalation, they will continue on the sublingual daily maintenance dose for approximately one year.
Milk Protein Extract Immunotherapy : Sublingual extract daily in escalating doses to goal of 7mg/day for approximately 1 1/2 years.
|
|---|---|---|---|
|
Gastrointestinal disorders
Oropharyngeal inflammation
|
100.0%
10/10 • Number of events 305
|
100.0%
10/10 • Number of events 840
|
100.0%
10/10 • Number of events 1164
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place