Trial Outcomes & Findings for A Randomized, Double-Blind, Placebo-Controlled Study of Oral Milk Immunotherapy for Cow's Milk Allergy (NCT NCT00465569)
NCT ID: NCT00465569
Last Updated: 2017-04-20
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Baseline and 23 weeks
Results posted on
2017-04-20
Participant Flow
Children between the ages of 6 and 21 years with a known history of IgE-mediated milk allergy were recruited from the pediatric allergy clinics at the Johns Hopkins University Hospital, Baltimore, Maryland, and Duke University Medical Center, Durham, North Carolina.
Eligibility criteria were a positive skin prick test (SPT) response to milk extract (wheal ≥ histamine control) or milk immunoglobulin E (IgE) level of \> 0.35 kilounits per liter (kU/L) and a positive milk challenge result at baseline defined as reacting with clear signs, symptoms, or both to a cumulative dose of 2.5 g or less of milk protein.
Participant milestones
| Measure |
Active Treatment
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
Pre-measured doses of ProFree, a dry, milk free powder
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
7
|
|
Overall Study
COMPLETED
|
12
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Active Treatment
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
Pre-measured doses of ProFree, a dry, milk free powder
|
|---|---|---|
|
Overall Study
persistent eczema during dose escalation
|
1
|
0
|
Baseline Characteristics
A Randomized, Double-Blind, Placebo-Controlled Study of Oral Milk Immunotherapy for Cow's Milk Allergy
Baseline characteristics by cohort
| Measure |
Active Treatment
n=13 Participants
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
n=7 Participants
Placebo
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
13 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
9.3 years
STANDARD_DEVIATION 3.3 • n=99 Participants
|
10.2 years
STANDARD_DEVIATION 3.3 • n=107 Participants
|
9.5 years
STANDARD_DEVIATION 2.6 • n=206 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=99 Participants
|
7 participants
n=107 Participants
|
20 participants
n=206 Participants
|
|
History of eczema
|
7 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
Current eczema
|
4 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
History of asthma
|
12 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Current asthma
|
9 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
History of other food allergy
|
10 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline and 23 weeksPopulation: One participant in the active treatment arm did not complete the study due to persistent eczema during dose escalation. The median milk threshold dose in both groups was 40 with a full range of 40-1340 at the baseline challenge.
Outcome measures
| Measure |
Active Treatment
n=12 Participants
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
n=7 Participants
Placebo
|
|---|---|---|
|
The Median Milk Threshold Dose Inducing a Reaction
Baseline
|
40 miligrams
Interval 40.0 to 1340.0
|
40 miligrams
Interval 40.0 to 1340.0
|
|
The Median Milk Threshold Dose Inducing a Reaction
Change at 23 weeks
|
5100 miligrams
Interval 2500.0 to 8100.0
|
0 miligrams
Interval -1300.0 to 0.0
|
SECONDARY outcome
Timeframe: Baseline and 23 weeksPopulation: One participant in the active treatment arm did not complete the study due to persistent eczema during dose escalation
IgE is measured in kilounits per liter (kU/L). Measurements were obtained at Baseline and at 23 weeks
Outcome measures
| Measure |
Active Treatment
n=12 Participants
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
n=7 Participants
Placebo
|
|---|---|---|
|
Changes in Cow Milk-IgE
|
8 percentage of change
Interval -50.0 to 64.0
|
-5 percentage of change
Interval -13.0 to 71.0
|
SECONDARY outcome
Timeframe: Baseline and 23 weeksPopulation: One participant in the active treatment arm did not complete the study due to persistent eczema during dose escalation
IgG4 is measured in ug/mL. Measurements were obtained at Baseline and at 23 weeks
Outcome measures
| Measure |
Active Treatment
n=12 Participants
Pre-measured doses of dry, nonfat powered milk prepared by the clinical research-registered dieticians
cow's milk powder: Milk powder given orally in escalating doses to a goal of 500 mg for approximately 23 weeks
|
Placebo
n=7 Participants
Placebo
|
|---|---|---|
|
Changes in Cow Milk Immunoglobulin G4 (IgG4)
|
767 percentage of change
Interval 29.0 to 1321.0
|
14 percentage of change
Interval -14.0 to 157.0
|
Adverse Events
Active Treatment
Serious events: 0 serious events
Other events: 1107 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 134 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Active Treatment
n=2437 participants at risk
Adverse event information is based on percentages of doses. There were 2437 total doses in the active treatment arm with a median of 177 and a range of 155-242 per participant. There was a total of 13 participants in the active treatment arm who were analyzed for the Adverse Events data reported below.
|
Placebo
n=1193 participants at risk
Adverse event information is based on percentages of doses. There were 1193 total doses in the placebo arm with a median of 171 and a range of 152-199 per participant. There was a total of 7 participants in the placebo arm who were analyzed for the Adverse Events data reported below.
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Local symptoms
|
35.7%
870/2437 • Number of events 870
|
8.7%
104/1193 • Number of events 104
|
|
Gastrointestinal disorders
Gastrointestinal
|
18.8%
458/2437 • Number of events 458
|
1.3%
16/1193 • Number of events 16
|
|
Respiratory, thoracic and mediastinal disorders
Lower respiratory
|
8.1%
198/2437 • Number of events 198
|
2.3%
28/1193 • Number of events 28
|
|
Skin and subcutaneous tissue disorders
Skin
|
0.90%
22/2437 • Number of events 22
|
0.08%
1/1193 • Number of events 1
|
|
General disorders
Multiple systems
|
1.2%
29/2437 • Number of events 29
|
0.00%
0/1193
|
|
Skin and subcutaneous tissue disorders
Eczema flare
|
0.04%
1/2437 • Number of events 1
|
0.08%
1/1193 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place