Trial Outcomes & Findings for Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification (NCT NCT00124709)
NCT ID: NCT00124709
Last Updated: 2011-03-23
Results Overview
Proportion of disease-free days in Step 2 or less (per Patient) using total number of days in study as the denominator- double-blind phase. Intent to Treat Population: defined as all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
1091 participants
Primary outcome timeframe
36 months
Results posted on
2011-03-23
Participant Flow
Participant milestones
| Measure |
Pimecrolimus (Elidel) Treatment Group
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Overall Study
STARTED
|
546
|
545
|
|
Overall Study
Patients Who Received Study Medication
|
543
|
544
|
|
Overall Study
COMPLETED
|
291
|
273
|
|
Overall Study
NOT COMPLETED
|
255
|
272
|
Reasons for withdrawal
| Measure |
Pimecrolimus (Elidel) Treatment Group
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Overall Study
Randomized but did not take medication
|
3
|
1
|
|
Overall Study
Adverse Event
|
3
|
0
|
|
Overall Study
Lack of Efficacy
|
5
|
10
|
|
Overall Study
Protocol Violation
|
3
|
5
|
|
Overall Study
Withdrawal by Subject
|
127
|
136
|
|
Overall Study
Lost to Follow-up
|
114
|
120
|
Baseline Characteristics
Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification
Baseline characteristics by cohort
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=543 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=544 Participants
Management with vehicle/topical corticosteroid rescue.
|
Total
n=1087 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
7.1 months
STANDARD_DEVIATION 3.76 • n=5 Participants
|
7.4 months
STANDARD_DEVIATION 4.06 • n=7 Participants
|
7.3 months
STANDARD_DEVIATION 3.91 • n=5 Participants
|
|
Age, Customized
Less than 3 months
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Customized
3 months to Less than 6 months
|
230 Participants
n=5 Participants
|
223 Participants
n=7 Participants
|
453 Participants
n=5 Participants
|
|
Age, Customized
6 months to Less than 12 months
|
231 Participants
n=5 Participants
|
220 Participants
n=7 Participants
|
451 Participants
n=5 Participants
|
|
Age, Customized
12 months to Less than or equal to 18 months
|
81 Participants
n=5 Participants
|
99 Participants
n=7 Participants
|
180 Participants
n=5 Participants
|
|
Age, Customized
Greater than 18 months
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
194 Participants
n=5 Participants
|
218 Participants
n=7 Participants
|
412 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
349 Participants
n=5 Participants
|
326 Participants
n=7 Participants
|
675 Participants
n=5 Participants
|
|
Parents' Index of Quality of Life - Atopic Dermatitis Score
|
6.5 Scores on PIQoL-AD Scale
STANDARD_DEVIATION 4.73 • n=5 Participants
|
6.9 Scores on PIQoL-AD Scale
STANDARD_DEVIATION 4.92 • n=7 Participants
|
6.7 Scores on PIQoL-AD Scale
STANDARD_DEVIATION 4.83 • n=5 Participants
|
PRIMARY outcome
Timeframe: 36 monthsPopulation: Intent to Treat Population: all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.
Proportion of disease-free days in Step 2 or less (per Patient) using total number of days in study as the denominator- double-blind phase. Intent to Treat Population: defined as all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.
Outcome measures
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=530 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=523 Participants
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Atopic Dermatitis (AD) Disease Control Over 36 Months
|
0.4404 Proportion of disease free days
Standard Deviation 0.29876
|
0.4346 Proportion of disease free days
Standard Deviation 0.29317
|
PRIMARY outcome
Timeframe: 6 yearsNote: The results for this efficacy variable are not reported due to early termination of the study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 yearsNote: The results of this secondary outcome is not reported due to early termination of the study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 years (36 month Double-Blind Phase)Population: Intent to Treat Population defined as all randomized patients who were dispensed study medication and had at least one post-baseline efficacy measurement.
Percentage of Patients who had allergic rhinitis, allergic conjunctivitis and food allergies at the end of the 36 month double blind study. Note: The results at six years are not reported due to early termination of the study.
Outcome measures
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=533 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=532 Participants
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Incidence of Allergic Rhinitis, Allergic Conjunctivitis and Food Allergies
Diagnosis of Food Allergy
|
16.1 Percentage of Participants
|
13.2 Percentage of Participants
|
|
Incidence of Allergic Rhinitis, Allergic Conjunctivitis and Food Allergies
Diagnosis of Allergic rhinitis
|
18.6 Percentage of Participants
|
16.4 Percentage of Participants
|
|
Incidence of Allergic Rhinitis, Allergic Conjunctivitis and Food Allergies
Diagnosis of Allergic conjunctivitis
|
12.4 Percentage of Participants
|
10.5 Percentage of Participants
|
SECONDARY outcome
Timeframe: 48 monthsPopulation: Safety population: all randomized patients who were dispensed study medication.
Corticosteroid and pimecrolimus study medication days of exposure during the 36 month double-blind phase. Note: Although the double-blind phase was designed to be 36 months (3 years) in length, the last double-blind visit for some patients occurred after 36 months.
Outcome measures
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=530 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=523 Participants
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Corticosteroid and Pimecrolimus Drug Use
Double -blind phase
|
264.8 Days of Exposure
Standard Deviation 259.49
|
249.6 Days of Exposure
Standard Deviation 251.71
|
|
Corticosteroid and Pimecrolimus Drug Use
0-12 months (Year 1)
|
146.6 Days of Exposure
Standard Deviation 105.75
|
147.6 Days of Exposure
Standard Deviation 112.69
|
|
Corticosteroid and Pimecrolimus Drug Use
13-24 months (Year 2)
|
75.0 Days of Exposure
Standard Deviation 105.08
|
65.0 Days of Exposure
Standard Deviation 97.42
|
|
Corticosteroid and Pimecrolimus Drug Use
25-36 months (Year 3)
|
42.2 Days of Exposure
Standard Deviation 83.65
|
36.6 Days of Exposure
Standard Deviation 76.31
|
|
Corticosteroid and Pimecrolimus Drug Use
37-48 months (Year 4)
|
1.1 Days of Exposure
Standard Deviation 5.74
|
1.0 Days of Exposure
Standard Deviation 5.34
|
SECONDARY outcome
Timeframe: 36 month Double-Blind PhasePopulation: Intent-to-Treat population: all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.
Longest duration of atopic dermatitis (AD) remission during the 36 month double-blind treatment phase. A remission day was defined as a diary day with a positive response ("yes") to the question "No or almost no eczema?" and a response of no treatment except emollients to the question "Medication used".
Outcome measures
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=530 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=523 Participants
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Atopic Dermatitis (AD) Remission Time
|
105.9547 Days
Standard Deviation 137.61350
|
117.1071 Days
Standard Deviation 150.33993
|
SECONDARY outcome
Timeframe: From Baseline to Visit 5 , 6, 8, 10, 12, and 14Population: Intent-to-Treat Population: all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.
Change from Baseline in the total Parents' Index of Quality of Life-Atopic Dermatitis (PIQoL-AD) score in the double-blind phase. PIQoL-AD Score = (sum of valid items/number of valid items) \* 28. Scores range from a minimum value of 0 to a maximum value of 28 with a high total overall score indicating poor quality of life.
Outcome measures
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=533 Participants
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=523 Participants
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 5 (Week 14)
|
-2.6 Scores on PIQoL-AD Scale
Standard Deviation 3.67
|
-2.3 Scores on PIQoL-AD Scale
Standard Deviation 3.37
|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 6 (Week 27)
|
-2.7 Scores on PIQoL-AD Scale
Standard Deviation 3.84
|
-2.7 Scores on PIQoL-AD Scale
Standard Deviation 3.83
|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 8 (Week 53)
|
-2.9 Scores on PIQoL-AD Scale
Standard Deviation 3.97
|
-3.1 Scores on PIQoL-AD Scale
Standard Deviation 4.23
|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 10 (Week 88)
|
-3.1 Scores on PIQoL-AD Scale
Standard Deviation 4.13
|
-3.5 Scores on PIQoL-AD Scale
Standard Deviation 4.23
|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 12 (Week 122)
|
-3.3 Scores on PIQoL-AD Scale
Standard Deviation 4.15
|
-3.6 Scores on PIQoL-AD Scale
Standard Deviation 4.39
|
|
Patient/Caregiver Quality of Life
Change in Baseline to Visit 14 (Week 158)
|
-3.5 Scores on PIQoL-AD Scale
Standard Deviation 4.28
|
-3.8 Scores on PIQoL-AD Scale
Standard Deviation 4.35
|
Adverse Events
Pimecrolimus (Elidel) Treatment Group
Serious events: 42 serious events
Other events: 482 other events
Deaths: 0 deaths
Control Treatment Group
Serious events: 37 serious events
Other events: 467 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=543 participants at risk
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=544 participants at risk
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Infections and infestations
Pneumonia
|
1.1%
6/543
|
1.1%
6/544
|
|
Infections and infestations
Gastroenteritis rotavirus
|
0.55%
3/543
|
0.74%
4/544
|
|
Infections and infestations
Bronchiolitis
|
0.37%
2/543
|
0.55%
3/544
|
|
Infections and infestations
Gastroenteritis
|
0.37%
2/543
|
0.37%
2/544
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.37%
2/543
|
0.37%
2/544
|
|
Infections and infestations
Respiratory syncytial virus bronchiolitis
|
0.37%
2/543
|
0.18%
1/544
|
|
Infections and infestations
Upper respiratory tract infection
|
0.37%
2/543
|
0.00%
0/544
|
|
Infections and infestations
Gastroenteritis viral
|
0.18%
1/543
|
0.18%
1/544
|
|
Infections and infestations
Pharyngitis
|
0.18%
1/543
|
0.18%
1/544
|
|
Infections and infestations
Pneumonia viral
|
0.18%
1/543
|
0.18%
1/544
|
|
Infections and infestations
Bronchitis
|
0.00%
0/543
|
0.37%
2/544
|
|
Infections and infestations
Adenovirus infection
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Croup, infectious
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Influenza
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Lobar pneumonia
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Lymph node abscess
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Staphylococcal infection
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Tonsillitis, streptococcal
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Varicella
|
0.18%
1/543
|
0.00%
0/544
|
|
Infections and infestations
Abscess
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Beta hemolytic streptococcal infection
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Eczema, infected
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Laryngotracheo bronchitis
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Pneumonia, bacterial
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Pyelonephritis
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Rotavirus infection
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/543
|
0.18%
1/544
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/543
|
0.18%
1/544
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
1.3%
7/543
|
0.92%
5/544
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial hyper-reactivity
|
0.55%
3/543
|
0.00%
0/544
|
|
Respiratory, thoracic and mediastinal disorders
Status asthmaticus
|
0.55%
3/543
|
0.00%
0/544
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.37%
2/543
|
0.18%
1/544
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.18%
1/543
|
0.18%
1/544
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
0.18%
1/543
|
0.18%
1/544
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
0.18%
1/543
|
0.00%
0/544
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.18%
1/543
|
0.00%
0/544
|
|
Respiratory, thoracic and mediastinal disorders
Epiglottic edema
|
0.18%
1/543
|
0.00%
0/544
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.18%
1/543
|
0.00%
0/544
|
|
Metabolism and nutrition disorders
Dehydration
|
0.74%
4/543
|
0.92%
5/544
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.18%
1/543
|
0.00%
0/544
|
|
Immune system disorders
Anaphylactic reaction
|
0.00%
0/543
|
0.37%
2/544
|
|
Immune system disorders
Food Allergy
|
0.00%
0/543
|
0.37%
2/544
|
|
Immune system disorders
Hypersensitivity
|
0.18%
1/543
|
0.00%
0/544
|
|
Immune system disorders
Milk allergy
|
0.00%
0/543
|
0.18%
1/544
|
|
Nervous system disorders
Febrile convulsions
|
0.37%
2/543
|
0.37%
2/544
|
|
Injury, poisoning and procedural complications
Post procedural hemorrhage
|
0.18%
1/543
|
0.00%
0/544
|
|
Injury, poisoning and procedural complications
Therapeutic agent toxicity
|
0.18%
1/543
|
0.00%
0/544
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/543
|
0.18%
1/544
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/543
|
0.18%
1/544
|
|
Injury, poisoning and procedural complications
Hepatic trauma
|
0.00%
0/543
|
0.18%
1/544
|
|
Injury, poisoning and procedural complications
Subdural hematoma
|
0.00%
0/543
|
0.18%
1/544
|
|
Blood and lymphatic system disorders
Idiopathic thrombocytopenic purpura
|
0.18%
1/543
|
0.00%
0/544
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/543
|
0.18%
1/544
|
|
Congenital, familial and genetic disorders
Von Willebrand's disease
|
0.18%
1/543
|
0.00%
0/544
|
|
Gastrointestinal disorders
Intussusception
|
0.18%
1/543
|
0.00%
0/544
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to lung
|
0.18%
1/543
|
0.00%
0/544
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Nephroblastoma
|
0.18%
1/543
|
0.00%
0/544
|
|
Renal and urinary disorders
Hematuria
|
0.18%
1/543
|
0.00%
0/544
|
|
Reproductive system and breast disorders
Prepuce redundant
|
0.18%
1/543
|
0.00%
0/544
|
|
Eye disorders
Retinal hemorrhage
|
0.00%
0/543
|
0.18%
1/544
|
|
General disorders
Adverse drug reaction
|
0.00%
0/543
|
0.18%
1/544
|
Other adverse events
| Measure |
Pimecrolimus (Elidel) Treatment Group
n=543 participants at risk
Pimecrolimus (Elidel) 1% Cream twice a day/topical corticosteroid rescue
|
Control Treatment Group
n=544 participants at risk
Management with vehicle/topical corticosteroid rescue.
|
|---|---|---|
|
Infections and infestations
Upper Respiratory Tract Infection
|
53.0%
288/543
|
48.7%
265/544
|
|
Infections and infestations
Otitis Media
|
48.6%
264/543
|
47.8%
260/544
|
|
Infections and infestations
Nasopharyngitis
|
34.3%
186/543
|
32.2%
175/544
|
|
Infections and infestations
Gastroenteritis
|
10.1%
55/543
|
9.2%
50/544
|
|
Infections and infestations
Sinusitis
|
13.8%
75/543
|
11.2%
61/544
|
|
Infections and infestations
Rhinitis
|
9.6%
52/543
|
9.0%
49/544
|
|
Infections and infestations
Croup infectious
|
8.5%
46/543
|
9.4%
51/544
|
|
Infections and infestations
Pharyngitis streptococcal
|
6.4%
35/543
|
10.1%
55/544
|
|
Infections and infestations
Pneumonia
|
8.3%
45/543
|
8.1%
44/544
|
|
Infections and infestations
Conjunctivitis, bacterial
|
7.2%
39/543
|
8.3%
45/544
|
|
Infections and infestations
Viral upper respiratory tract infection
|
6.8%
37/543
|
7.0%
38/544
|
|
Infections and infestations
Bronchitis
|
7.6%
41/543
|
5.5%
30/544
|
|
Infections and infestations
Bronchiolitis
|
5.2%
28/543
|
6.1%
33/544
|
|
Infections and infestations
Impetigo
|
4.1%
22/543
|
6.8%
37/544
|
|
Infections and infestations
Viral rash
|
5.3%
29/543
|
5.3%
29/544
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
34.4%
187/543
|
32.0%
174/544
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
22.5%
122/543
|
19.7%
107/544
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
18.8%
102/543
|
16.4%
89/544
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
12.5%
68/543
|
11.6%
63/544
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
11.8%
64/543
|
9.7%
53/544
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
9.6%
52/543
|
7.9%
43/544
|
|
Gastrointestinal disorders
Diarrhea
|
10.1%
55/543
|
8.5%
46/544
|
|
Gastrointestinal disorders
Teething
|
24.7%
134/543
|
24.8%
135/544
|
|
Gastrointestinal disorders
Vomiting
|
6.3%
34/543
|
5.3%
29/544
|
|
Immune system disorders
Food Allergy
|
25.8%
140/543
|
21.7%
118/544
|
|
Immune system disorders
Milk allergy
|
9.2%
50/543
|
6.4%
35/544
|
|
General disorders
Pyrexia
|
22.7%
123/543
|
22.6%
123/544
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
8.1%
44/543
|
8.5%
46/544
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.6%
41/543
|
5.7%
31/544
|
|
Eye disorders
Conjunctivitis, allergic
|
14.0%
76/543
|
11.6%
63/544
|
|
Eye disorders
Conjunctivitis
|
9.2%
50/543
|
8.1%
44/544
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER