Trial Outcomes & Findings for Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex (NCT NCT03826628)

Last Updated: 2023-09-08

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

107 participants

Primary outcome timeframe

After 26 weeks treatment

Results posted on

2023-09-08

Participant Flow

Participant milestones

Participant milestones
Measure	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	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	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
Overall Study STARTED	36	33	38
Overall Study COMPLETED	32	31	35
Overall Study NOT COMPLETED	4	2	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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	Total n=107 Participants Total of all reporting groups
Age, Continuous	27.8 years STANDARD_DEVIATION 15 • n=5 Participants	25.5 years STANDARD_DEVIATION 15 • n=7 Participants	26.3 years STANDARD_DEVIATION 13.8 • n=5 Participants	26.6 years STANDARD_DEVIATION 14.5 • n=4 Participants
Age, Customized Age group · 6-11 years	6 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants	16 Participants n=4 Participants
Age, Customized Age group · 12-17 years	5 Participants n=5 Participants	8 Participants n=7 Participants	7 Participants n=5 Participants	20 Participants n=4 Participants
Age, Customized Age group · 18-65 years	25 Participants n=5 Participants	21 Participants n=7 Participants	25 Participants n=5 Participants	71 Participants n=4 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	14 Participants n=7 Participants	23 Participants n=5 Participants	58 Participants n=4 Participants
Sex: Female, Male Male	15 Participants n=5 Participants	19 Participants n=7 Participants	15 Participants n=5 Participants	49 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	13 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	31 Participants n=5 Participants	29 Participants n=7 Participants	34 Participants n=5 Participants	94 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Asian	7 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants	18 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) White	28 Participants n=5 Participants	26 Participants n=7 Participants	30 Participants n=5 Participants	84 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Study Site Site #00 - ACRI Johns Hopkins	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Study Site Site #01 - University Hospital Brno	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Study Site Site #02 - Children's Health Queensland	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Study Site Site #03 - Christchurch Hospital	2 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants
Study Site Site #04 - University of Navarra	4 Participants n=5 Participants	4 Participants n=7 Participants	3 Participants n=5 Participants	11 Participants n=4 Participants
Study Site Site #05 - Mayo Clinic	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Study Site Site #06 - Phoenix Children's Hospital	2 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	5 Participants n=4 Participants
Study Site Site #07 - Spectrum Health, Michigan	3 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Study Site Site #08 - University of California, San Diego	3 Participants n=5 Participants	4 Participants n=7 Participants	2 Participants n=5 Participants	9 Participants n=4 Participants
Study Site Site #09 - University of Virginia	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Study Site Site #10 - University of Pecs	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Study Site Site #11 - Slovakia National Institute of Children's Diseases	3 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	7 Participants n=4 Participants
Study Site Site #12 - Bethesda Children's Hospital	5 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants	16 Participants n=4 Participants
Study Site Site #13 - University of Szeged	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Study Site Site #14 - Clinic of Neurology and Psychiatry for Children and Youth, Belgrade	2 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Study Site Site #15 - Clinical Center of Serbia	2 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants
Study Site Site #16 - National Taiwan University	5 Participants n=5 Participants	5 Participants n=7 Participants	5 Participants n=5 Participants	15 Participants n=4 Participants
Height	164 centimeters STANDARD_DEVIATION 17 • n=5 Participants	165 centimeters STANDARD_DEVIATION 16 • n=7 Participants	161 centimeters STANDARD_DEVIATION 17 • n=5 Participants	163 centimeters STANDARD_DEVIATION 17 • n=4 Participants
Weight	68.1 kilograms STANDARD_DEVIATION 22.8 • n=5 Participants	68.9 kilograms STANDARD_DEVIATION 24.3 • n=7 Participants	68.4 kilograms STANDARD_DEVIATION 25.8 • n=5 Participants	68.5 kilograms STANDARD_DEVIATION 24.2 • n=4 Participants
BMI	24.7 kg/m^2 STANDARD_DEVIATION 5.6 • n=5 Participants	24.8 kg/m^2 STANDARD_DEVIATION 6.6 • n=7 Participants	25.4 kg/m^2 STANDARD_DEVIATION 6.4 • n=5 Participants	25 kg/m^2 STANDARD_DEVIATION 6.2 • n=4 Participants
Rapamycin concentration Below limit of detection	36 Participants n=5 Participants	33 Participants n=7 Participants	38 Participants n=5 Participants	107 Participants n=4 Participants
Rapamycin concentration Above limit of detection	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Investigator's Global Assessment (IGA) 2 - Mild	13 Participants n=5 Participants	16 Participants n=7 Participants	19 Participants n=5 Participants	48 Participants n=4 Participants
Investigator's Global Assessment (IGA) 3 - Moderate	23 Participants n=5 Participants	17 Participants n=7 Participants	19 Participants n=5 Participants	59 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 4	7 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants	18 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 5	12 Participants n=5 Participants	13 Participants n=7 Participants	16 Participants n=5 Participants	41 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 6	8 Participants n=5 Participants	6 Participants n=7 Participants	8 Participants n=5 Participants	22 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 7	6 Participants n=5 Participants	5 Participants n=7 Participants	5 Participants n=5 Participants	16 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 8	2 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Facial Angiofibroma Severity Index (FASI) 9	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants

PRIMARY outcome

Timeframe: After 26 weeks treatment

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Percentage of Participants Obtaining Successful Treatment	4 Participants	3 Participants	2 Participants

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Time to Treatment Success	25.89 weeks Standard Error 0.7	26.71 weeks Standard Error 0.28	19.7 weeks Standard Error 0.22

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Change From Baseline in Investigator's Global Assessment -2	4 Participants	3 Participants	2 Participants
Change From Baseline in Investigator's Global Assessment -1	16 Participants	17 Participants	7 Participants
Change From Baseline in Investigator's Global Assessment +1	0 Participants	0 Participants	1 Participants
Change From Baseline in Investigator's Global Assessment Missing	1 Participants	0 Participants	0 Participants
Change From Baseline in Investigator's Global Assessment 0	15 Participants	13 Participants	28 Participants

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Change From Baseline in Facial Angiofibroma Severity Index (FASI) -4	1 Participants	3 Participants	1 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) -3	6 Participants	3 Participants	1 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) -1	10 Participants	13 Participants	17 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) 0	7 Participants	4 Participants	14 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) +2	0 Participants	0 Participants	1 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) Missing	1 Participants	0 Participants	0 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) -5	1 Participants	1 Participants	0 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) -2	10 Participants	9 Participants	3 Participants
Change From Baseline in Facial Angiofibroma Severity Index (FASI) +1	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale	57.06 Percentage Change Standard Error 4.36	54.06 Percentage Change Standard Error 5.21	30.84 Percentage Change Standard Error 3.89

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Objective (Clinician) Percentage Change Rating Scale	47.83 Percentage Change Standard Error 3.88	49.39 Percentage Change Standard Error 5.22	20.76 Percentage Change Standard Error 4.05

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	0.5% Rapamycin Cream, Topical n=36 Participants 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	1.0% Rapamycin Cream, Topical n=33 Participants 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	Placebo n=38 Participants 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
Categorical Change in Facial Angiofibroma -1 - Worse	1 Participants	0 Participants	0 Participants
Categorical Change in Facial Angiofibroma 0 - No Difference	0 Participants	1 Participants	12 Participants
Categorical Change in Facial Angiofibroma +1 - Slightly Better	2 Participants	7 Participants	11 Participants
Categorical Change in Facial Angiofibroma Missing	4 Participants	5 Participants	2 Participants
Categorical Change in Facial Angiofibroma +2 - Moderately Better	13 Participants	7 Participants	9 Participants
Categorical Change in Facial Angiofibroma +3 - Significantly Better	16 Participants	13 Participants	4 Participants

Adverse Events

0.5% Rapamycin Cream, Topical

Serious events: 2 serious events

Other events: 29 other events

Deaths: 0 deaths

1.0% Rapamycin Cream, Topical

Serious events: 2 serious events

Other events: 27 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	0.5% Rapamycin Cream, Topical n=36 participants at risk 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	1.0% Rapamycin Cream, Topical n=33 participants at risk 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	Placebo n=38 participants at risk 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
Nervous system disorders Seizure	2.8% 1/36 • Number of events 1 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/33 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	2.6% 1/38 • Number of events 2 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.
Gastrointestinal disorders Constipation, nausea, malnutrition, diarrhea	0.00% 0/36 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/33 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	5.3% 2/38 • Number of events 2 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.
Cardiac disorders Chest pain	2.8% 1/36 • Number of events 1 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/33 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/38 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.
Renal and urinary disorders Kidney bleeding	0.00% 0/36 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	3.0% 1/33 • Number of events 1 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/38 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.
Cardiac disorders Supraventricular tachycardia	0.00% 0/36 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/33 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	2.6% 1/38 • Number of events 1 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.
Injury, poisoning and procedural complications Collapsed lung	0.00% 0/36 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	3.0% 1/33 • Number of events 1 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.	0.00% 0/38 • 30 weeks Adverse events were collected during each clinical visit and follow-up visit. Patients or their parents/guardians were questioned by the investigator regarding any adverse events experienced since the previous clinical visit.

Other adverse events

Additional Information

Ioana Stanescu

AFT Pharmaceuticals Ltd.

Phone: +64 9 488 0232

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60