Facial Angiofibroma, Tuberous Sclerosis
Conditions
Brief summary
The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).
Detailed description
Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to be well tolerated. The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).
Interventions
DRUGrapamycin
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
DRUGplacebo
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
Sponsors
Dermatology Specialties Limited Partnership
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained 2. Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma 3. An FA severity score of 2 or 3 on the IGA scale 4. Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation 5. Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator
Exclusion criteria
1. Patients who cannot carry out the treatment plan or follow-up assessment 2. Patients with serious skin lesions such as erosions or ulcers 3. Patients with known hypersensitivity to any component of the study product 4. Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment 5. Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment 6. Patients who participated in any other clinical trial within 3 months prior to the day of enrolment 7. Patients judged unsuitable for this clinical trial by the investigator or sub-investigator 8. Pregnant or lactating females 9. Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception 10. Patients with immune dysfunction or receiving any form of immunosuppression 11. Patients with severe FA, with a score of 4 on the IGA scale 12. Patients with an FA severity score of less than 2 on the IGA scale
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Obtaining Successful Treatment | After 26 weeks treatment | Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Investigator's Global Assessment | At baseline and after 26 weeks treatment | The change in grading on the Investigator's Global Assessment (IGA) scale from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe |
| Change From Baseline in Facial Angiofibroma Severity Index (FASI) | At baseline and after 26 weeks treatment | The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline. FASI grades lesions according to their erythema, size and extent by summing the scores of each category. The final FASI scores range from (mild) 2-9 (severe). Erythema Skin color 0 Light Red 1 Red 2 Dark Red/purple 3 Size None 0 Small (\< 5mm) 1 Large (\> 5mm) 2 Confluent 3 Extension \<50 % cheek surface 2 \>50% cheek surface 3 |
| Time to Treatment Success | From first dose to 26 weeks (± 2 weeks) | The time elapsed from the first dose to the time of treatment success, according to the Investigator's Global Assessment (IGA) scale. The total time of treatment was 26 weeks, although Covid-19 visit delays led to an extension of up to 2 weeks (28 weeks total) for some patients. Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe |
| Objective (Clinician) Percentage Change Rating Scale | After 26 weeks treatment | Percentage improvement in facial angiofibroma since beginning treatment, as assessed by the clinician. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where clinicians estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline. |
| Categorical Change in Facial Angiofibroma | After 26 weeks treatment | Change in facial angiofibroma since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver. This was a single assessment time-point, where the participant or parent/caregiver evaluated the change in the facial angiofibroma lesion appearance from their perspective since baseline. |
| Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale | After 26 weeks treatment | Percentage change in facial angiofibroma since beginning treatment, as assessed by the participant or parent/caregiver. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where the participant or parent/caregiver estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline. |
Countries
Australia, Czechia, Hungary, New Zealand, Serbia, Slovakia, Spain, Taiwan, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 0.5% Rapamycin Cream, Topical Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
rapamycin: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks | 36 |
| 1.0% Rapamycin Cream, Topical Rapamycin cream topical, 1.0% w/w, applied once daily before bed on affected area for 26 weeks
rapamycin: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks | 33 |
| Placebo Placebo cream topical, applied once daily before bed on affected area for 26 weeks
placebo: Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks | 38 |
| Total | 107 |
Baseline characteristics
| Characteristic | 1.0% Rapamycin Cream, Topical | Placebo | 0.5% Rapamycin Cream, Topical | Total |
|---|---|---|---|---|
| Age, Continuous | 25.5 years STANDARD_DEVIATION 15 | 26.3 years STANDARD_DEVIATION 13.8 | 27.8 years STANDARD_DEVIATION 15 | 26.6 years STANDARD_DEVIATION 14.5 |
| Age, Customized Age group 12-17 years | 8 Participants | 7 Participants | 5 Participants | 20 Participants |
| Age, Customized Age group 18-65 years | 21 Participants | 25 Participants | 25 Participants | 71 Participants |
| Age, Customized Age group 6-11 years | 4 Participants | 6 Participants | 6 Participants | 16 Participants |
| BMI | 24.8 kg/m^2 STANDARD_DEVIATION 6.6 | 25.4 kg/m^2 STANDARD_DEVIATION 6.4 | 24.7 kg/m^2 STANDARD_DEVIATION 5.6 | 25 kg/m^2 STANDARD_DEVIATION 6.2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 4 Participants | 5 Participants | 13 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 29 Participants | 34 Participants | 31 Participants | 94 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Facial Angiofibroma Severity Index (FASI) 4 | 5 Participants | 6 Participants | 7 Participants | 18 Participants |
| Facial Angiofibroma Severity Index (FASI) 5 | 13 Participants | 16 Participants | 12 Participants | 41 Participants |
| Facial Angiofibroma Severity Index (FASI) 6 | 6 Participants | 8 Participants | 8 Participants | 22 Participants |
| Facial Angiofibroma Severity Index (FASI) 7 | 5 Participants | 5 Participants | 6 Participants | 16 Participants |
| Facial Angiofibroma Severity Index (FASI) 8 | 3 Participants | 3 Participants | 2 Participants | 8 Participants |
| Facial Angiofibroma Severity Index (FASI) 9 | 1 Participants | 0 Participants | 1 Participants | 2 Participants |
| Height | 165 centimeters STANDARD_DEVIATION 16 | 161 centimeters STANDARD_DEVIATION 17 | 164 centimeters STANDARD_DEVIATION 17 | 163 centimeters STANDARD_DEVIATION 17 |
| Investigator's Global Assessment (IGA) 2 - Mild | 16 Participants | 19 Participants | 13 Participants | 48 Participants |
| Investigator's Global Assessment (IGA) 3 - Moderate | 17 Participants | 19 Participants | 23 Participants | 59 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 5 Participants | 6 Participants | 7 Participants | 18 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) White | 26 Participants | 30 Participants | 28 Participants | 84 Participants |
| Rapamycin concentration Above limit of detection | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Rapamycin concentration Below limit of detection | 33 Participants | 38 Participants | 36 Participants | 107 Participants |
| Sex: Female, Male Female | 14 Participants | 23 Participants | 21 Participants | 58 Participants |
| Sex: Female, Male Male | 19 Participants | 15 Participants | 15 Participants | 49 Participants |
| Study Site Site #00 - ACRI Johns Hopkins | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Study Site Site #01 - University Hospital Brno | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Study Site Site #02 - Children's Health Queensland | 1 Participants | 0 Participants | 1 Participants | 2 Participants |
| Study Site Site #03 - Christchurch Hospital | 2 Participants | 3 Participants | 2 Participants | 7 Participants |
| Study Site Site #04 - University of Navarra | 4 Participants | 3 Participants | 4 Participants | 11 Participants |
| Study Site Site #05 - Mayo Clinic | 0 Participants | 1 Participants | 1 Participants | 2 Participants |
| Study Site Site #06 - Phoenix Children's Hospital | 2 Participants | 1 Participants | 2 Participants | 5 Participants |
| Study Site Site #07 - Spectrum Health, Michigan | 3 Participants | 3 Participants | 3 Participants | 9 Participants |
| Study Site Site #08 - University of California, San Diego | 4 Participants | 2 Participants | 3 Participants | 9 Participants |
| Study Site Site #09 - University of Virginia | 0 Participants | 1 Participants | 1 Participants | 2 Participants |
| Study Site Site #10 - University of Pecs | 1 Participants | 2 Participants | 1 Participants | 4 Participants |
| Study Site Site #11 - Slovakia National Institute of Children's Diseases | 2 Participants | 2 Participants | 3 Participants | 7 Participants |
| Study Site Site #12 - Bethesda Children's Hospital | 4 Participants | 7 Participants | 5 Participants | 16 Participants |
| Study Site Site #13 - University of Szeged | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Study Site Site #14 - Clinic of Neurology and Psychiatry for Children and Youth, Belgrade | 3 Participants | 3 Participants | 2 Participants | 8 Participants |
| Study Site Site #15 - Clinical Center of Serbia | 2 Participants | 3 Participants | 2 Participants | 7 Participants |
| Study Site Site #16 - National Taiwan University | 5 Participants | 5 Participants | 5 Participants | 15 Participants |
| Weight | 68.9 kilograms STANDARD_DEVIATION 24.3 | 68.4 kilograms STANDARD_DEVIATION 25.8 | 68.1 kilograms STANDARD_DEVIATION 22.8 | 68.5 kilograms STANDARD_DEVIATION 24.2 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 36 | 0 / 33 | 0 / 38 |
| other Total, other adverse events | 29 / 36 | 27 / 33 | 27 / 38 |
| serious Total, serious adverse events | 2 / 36 | 2 / 33 | 2 / 38 |
Outcome results
Primary
Percentage of Participants Obtaining Successful Treatment
Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe
Time frame: After 26 weeks treatment
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| 0.5% Rapamycin Cream, Topical | Percentage of Participants Obtaining Successful Treatment | 4 Participants |
| 1.0% Rapamycin Cream, Topical | Percentage of Participants Obtaining Successful Treatment | 3 Participants |
| Placebo | Percentage of Participants Obtaining Successful Treatment | 2 Participants |
Secondary
Categorical Change in Facial Angiofibroma
Change in facial angiofibroma since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver. This was a single assessment time-point, where the participant or parent/caregiver evaluated the change in the facial angiofibroma lesion appearance from their perspective since baseline.
Time frame: After 26 weeks treatment
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +2 - Moderately Better | 13 Participants |
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +3 - Significantly Better | 16 Participants |
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | 0 - No Difference | 0 Participants |
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | Missing | 4 Participants |
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +1 - Slightly Better | 2 Participants |
| 0.5% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | -1 - Worse | 1 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +3 - Significantly Better | 13 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +2 - Moderately Better | 7 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | -1 - Worse | 0 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | 0 - No Difference | 1 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | Missing | 5 Participants |
| 1.0% Rapamycin Cream, Topical | Categorical Change in Facial Angiofibroma | +1 - Slightly Better | 7 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | Missing | 2 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | +3 - Significantly Better | 4 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | -1 - Worse | 0 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | 0 - No Difference | 12 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | +1 - Slightly Better | 11 Participants |
| Placebo | Categorical Change in Facial Angiofibroma | +2 - Moderately Better | 9 Participants |
Secondary
Change From Baseline in Facial Angiofibroma Severity Index (FASI)
The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline. FASI grades lesions according to their erythema, size and extent by summing the scores of each category. The final FASI scores range from (mild) 2-9 (severe). Erythema Skin color 0 Light Red 1 Red 2 Dark Red/purple 3 Size None 0 Small (\< 5mm) 1 Large (\> 5mm) 2 Confluent 3 Extension \<50 % cheek surface 2 \>50% cheek surface 3
Time frame: At baseline and after 26 weeks treatment
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -2 | 10 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | Missing | 1 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | 0 | 7 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -1 | 10 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -5 | 1 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +2 | 0 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -3 | 6 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -4 | 1 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +1 | 0 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -1 | 13 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -5 | 1 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -4 | 3 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -3 | 3 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -2 | 9 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | 0 | 4 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +1 | 0 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +2 | 0 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | Missing | 0 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -3 | 1 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -5 | 0 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +1 | 1 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -4 | 1 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | Missing | 0 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -1 | 17 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | -2 | 3 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | +2 | 1 Participants |
| Placebo | Change From Baseline in Facial Angiofibroma Severity Index (FASI) | 0 | 14 Participants |
Secondary
Change From Baseline in Investigator's Global Assessment
The change in grading on the Investigator's Global Assessment (IGA) scale from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe
Time frame: At baseline and after 26 weeks treatment
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | +1 | 0 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | 0 | 15 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | -2 | 4 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | -1 | 16 Participants |
| 0.5% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | Missing | 1 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | 0 | 13 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | -2 | 3 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | -1 | 17 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | +1 | 0 Participants |
| 1.0% Rapamycin Cream, Topical | Change From Baseline in Investigator's Global Assessment | Missing | 0 Participants |
| Placebo | Change From Baseline in Investigator's Global Assessment | Missing | 0 Participants |
| Placebo | Change From Baseline in Investigator's Global Assessment | +1 | 1 Participants |
| Placebo | Change From Baseline in Investigator's Global Assessment | -2 | 2 Participants |
| Placebo | Change From Baseline in Investigator's Global Assessment | 0 | 28 Participants |
| Placebo | Change From Baseline in Investigator's Global Assessment | -1 | 7 Participants |
Secondary
Objective (Clinician) Percentage Change Rating Scale
Percentage improvement in facial angiofibroma since beginning treatment, as assessed by the clinician. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where clinicians estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.
Time frame: After 26 weeks treatment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Objective (Clinician) Percentage Change Rating Scale | 47.83 Percentage Change | Standard Error 3.88 |
| 1.0% Rapamycin Cream, Topical | Objective (Clinician) Percentage Change Rating Scale | 49.39 Percentage Change | Standard Error 5.22 |
| Placebo | Objective (Clinician) Percentage Change Rating Scale | 20.76 Percentage Change | Standard Error 4.05 |
Secondary
Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale
Percentage change in facial angiofibroma since beginning treatment, as assessed by the participant or parent/caregiver. A large value indicates most improvement to facial angiofibroma (minimum=0, maximum=100). This was a single assessment time-point, where the participant or parent/caregiver estimated the percentage change in the facial angiofibroma lesion appearance from their perspective since baseline.
Time frame: After 26 weeks treatment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale | 57.06 Percentage Change | Standard Error 4.36 |
| 1.0% Rapamycin Cream, Topical | Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale | 54.06 Percentage Change | Standard Error 5.21 |
| Placebo | Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale | 30.84 Percentage Change | Standard Error 3.89 |
Secondary
Time to Treatment Success
The time elapsed from the first dose to the time of treatment success, according to the Investigator's Global Assessment (IGA) scale. The total time of treatment was 26 weeks, although Covid-19 visit delays led to an extension of up to 2 weeks (28 weeks total) for some patients. Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe
Time frame: From first dose to 26 weeks (± 2 weeks)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 0.5% Rapamycin Cream, Topical | Time to Treatment Success | 25.89 weeks | Standard Error 0.7 |
| 1.0% Rapamycin Cream, Topical | Time to Treatment Success | 26.71 weeks | Standard Error 0.28 |
| Placebo | Time to Treatment Success | 19.7 weeks | Standard Error 0.22 |