Trial Outcomes & Findings for A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients (NCT NCT00961896)

Last Updated: 2015-11-03

Results Overview

Clinical response parameters were defined as (i) complete response (i.e., there is no longer any visible evidence of a lesion consistent with BCC at this site), (ii) partial response (i.e., although a BCC still remains at this site, it has demonstrated a visible decrease in size compared with baseline), and (iii) no response / worsening (i.e., the BCC has not demonstrated any visible decrease in size compared with baseline).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

4 weeks, 6 weeks, 9 weeks

Results posted on

2015-11-03

Participant Flow

Participant milestones

Participant milestones
Measure	All Part I Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Overall Study STARTED	8	3	7
Overall Study COMPLETED	8	3	6
Overall Study NOT COMPLETED	0	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	All Part I Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Overall Study Abnormal laboratory value	0	0	1

Baseline Characteristics

A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Part I Participants n=8 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	LDE225 0.25% [Part II] n=3 Participants Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] n=7 Participants Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%	Total n=18 Participants Total of all reporting groups
Age, Continuous	50 Years STANDARD_DEVIATION 13.5 • n=5 Participants	52 Years STANDARD_DEVIATION 11.7 • n=7 Participants	48 Years STANDARD_DEVIATION 13.4 • n=5 Participants	50 Years STANDARD_DEVIATION 12.9 • n=4 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants	10 Participants n=4 Participants

PRIMARY outcome

Timeframe: 4 weeks, 6 weeks, 9 weeks

Population: All participants were analyzed.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=13 Tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=14 Tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] n=12 Tumors (BCCs) Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] n=22 Tumors (BCCs) Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 4, at least partial	92 Percentage of BCCs	7 Percentage of BCCs	83 Percentage of BCCs	77 Percentage of BCCs
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 4, complete	23 Percentage of BCCs	0 Percentage of BCCs	0 Percentage of BCCs	0 Percentage of BCCs
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 6, at least partial	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	83 Percentage of BCCs	96 Percentage of BCCs
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 6, complete	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	0 Percentage of BCCs	0 Percentage of BCCs
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 9, at least partial (LDE 0.75%, n=10)	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part II at LDE225 0.25% was 6 weeks only.	100 Percentage of BCCs
Percentage of BCCs With Complete and at Least Partial Clinical Clearance Week 9, complete (LDE 0.75%, n=10)	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part I was 4 weeks only.	NA Percentage of BCCs Treatment duration for Part II at LDE225 0.25% was 6 weeks only.	0 Percentage of BCCs

PRIMARY outcome

Timeframe: day 8, day 15, day 22, day 29

Population: All part I participants were included in the analysis.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=8 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=8 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Number of Participants With at Least Partial Clinical Clearance (Part I) Day 29	8 Number of participants	1 Number of participants	—	—
Number of Participants With at Least Partial Clinical Clearance (Part I) Day 8	5 Number of participants	1 Number of participants	—	—
Number of Participants With at Least Partial Clinical Clearance (Part I) Day 15	7 Number of participants	2 Number of participants	—	—
Number of Participants With at Least Partial Clinical Clearance (Part I) Day 22	8 Number of participants	1 Number of participants	—	—

SECONDARY outcome

Timeframe: 4 weeks

Population: All part I participants were included in the analysis.

Measurement of the tumor size, volume and color by standardized digital photography, using dermatoscopic, macroscopic and 3D images of the BCCs was done by participant where if a participant had more than one tumor, for each of these tumors, the change from baseline was calculated (% change). From these values, the mean was calculated to get only one result per participant. Then for all the participants (n=8 both for LDE and vehicle), the mean was calculated. Photographic analysis was conducted by QuantifiCare. The volume of a lesion was measured with a special device, the "3D LIFEVIZ Micro system", which uses a lens splitter to produce two images of the skin surface, captured at the same time, with viewing angle differences close to human vision. A stereovision algorithm is then applied to reconstruct and quantitatively analyze the skin surface in 3D. A negative change from baseline indicates improvement.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=8 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=8 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Change From Baseline in Tumor Measurements (Part I) Height	-32.3 Percent change in tumor measurement Standard Deviation 25.51	-13.2 Percent change in tumor measurement Standard Deviation 12.47	—	—
Change From Baseline in Tumor Measurements (Part I) Redness	-4.8 Percent change in tumor measurement Standard Deviation 16.47	1.2 Percent change in tumor measurement Standard Deviation 6.99	—	—
Change From Baseline in Tumor Measurements (Part I) Total volume	-53.4 Percent change in tumor measurement Standard Deviation 30.85	-9.9 Percent change in tumor measurement Standard Deviation 17.21	—	—
Change From Baseline in Tumor Measurements (Part I) 2D surface area	-38.4 Percent change in tumor measurement Standard Deviation 24.82	9.6 Percent change in tumor measurement Standard Deviation 6.75	—	—
Change From Baseline in Tumor Measurements (Part I) 3D surface area	-35.3 Percent change in tumor measurement Standard Deviation 19.68	7.0 Percent change in tumor measurement Standard Deviation 16.86	—	—

SECONDARY outcome

Timeframe: 4 weeks, 6 weeks, 9 weeks

Population: Part II participants with evaluable data (n=3,6) were included in the analysis.

Measurement of the tumor size, volume and color by standardized digital photography, using dermatoscopic, macroscopic and 3D images of the BCCs was done by participant where if a participant had more than one tumor, for each of these tumors, the change from baseline was calculated (% change). From these values, the mean was calculated to get only one result per participant. Then for all the participants (n=8 both for LDE and vehicle), the mean was calculated.. Photographic analysis was conducted by QuantifiCare. The volume of a lesion was measured with a special device, the "3D LIFEVIZ Micro system", which uses a lens splitter to produce two images of the skin surface, captured at the same time, with viewing angle differences close to human vision. A stereovision algorithm is then applied to reconstruct and quantitatively analyze the skin surface in 3D. A negative change from baseline indicates improvement.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=3 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=6 Participants Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Change From Baseline in Tumor Measurements (Part II) 2D surface, week 6	-36.3 percent change in tumor measurement Standard Deviation 12.51	-28.5 percent change in tumor measurement Standard Deviation 43.64	—	—
Change From Baseline in Tumor Measurements (Part II) 2D surface area, week 9	NA percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-26.0 percent change in tumor measurement Standard Deviation 41.57	—	—
Change From Baseline in Tumor Measurements (Part II) 2D surface, week 4	-10.7 percent change in tumor measurement Standard Deviation 16.81	-22.2 percent change in tumor measurement Standard Deviation 40.43	—	—
Change From Baseline in Tumor Measurements (Part II) 3D surface area , week 4	-19.3 percent change in tumor measurement Standard Deviation 4.62	-26.9 percent change in tumor measurement Standard Deviation 33.29	—	—
Change From Baseline in Tumor Measurements (Part II) 3D surface area, week 6	-31.7 percent change in tumor measurement Standard Deviation 19.03	-35.2 percent change in tumor measurement Standard Deviation 30.51	—	—
Change From Baseline in Tumor Measurements (Part II) 3D surface area, week 9	NA percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-41.5 percent change in tumor measurement Standard Deviation 30.05	—	—
Change From Baseline in Tumor Measurements (Part II) Total volume, week 4	-19.3 percent change in tumor measurement Standard Deviation 27.82	-43.4 percent change in tumor measurement Standard Deviation 36.25	—	—
Change From Baseline in Tumor Measurements (Part II) Total volume, week 6	-35.2 percent change in tumor measurement Standard Deviation 37.99	-60.7 percent change in tumor measurement Standard Deviation 22.11	—	—
Change From Baseline in Tumor Measurements (Part II) Total volume, week 9	NA percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-61.3 percent change in tumor measurement Standard Deviation 31.18	—	—

SECONDARY outcome

Timeframe: 4 weeks

Population: All part I participants were included in this analysis.

Measurement of the tumor size, volume and color by standardized digital photography, using dermatoscopic, macroscopic and 3D images of the BCCs. Photographic analysis was conducted by QuantifiCare. The volume of a lesion was measured with a special device, the "3D LIFEVIZ Micro system", which uses a lens splitter to produce two images of the skin surface, captured at the same time, with viewing angle differences close to human vision. A stereovision algorithm is then applied to reconstruct and quantitatively analyze the skin surface in 3D. A negative change from baseline indicates improvement.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=13 tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=14 tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Change From Baseline in Tumor Measurements (by Tumor) (Part I) 3D surface area	-34.5 Percent change in tumor measurement Standard Deviation 21.36	8.7 Percent change in tumor measurement Standard Deviation 20.41	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part I) Total volume	-49.8 Percent change in tumor measurement Standard Deviation 33.68	-9.1 Percent change in tumor measurement Standard Deviation 35.24	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part I) 2D surface area	-40.8 Percent change in tumor measurement Standard Deviation 24.85	10.6 Percent change in tumor measurement Standard Deviation 10.52	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part I) Height	-32.0 Percent change in tumor measurement Standard Deviation 26.97	-12.7 Percent change in tumor measurement Standard Deviation 18.93	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part I) Redness	-2.0 Percent change in tumor measurement Standard Deviation 22.50	1.2 Percent change in tumor measurement Standard Deviation 7.42	—	—

SECONDARY outcome

Timeframe: 4 weeks, 6 weeks, 9 weeks

Population: Part II participants with evaluable data (n=3,6) were included in the analysis.

Outcome measures

Outcome measures
Measure	LDE225 (Applied in Parallel With Vehicle) [Part I] n=12 Tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. LDE225 0.75%	Vehicle Cream (Applied in Parallel With LDE225 [Part I] n=22 Tumors (BCCs) Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant. Vehicle: Placebo cream	LDE225 0.25% [Part II] Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks. LDE225 0.25%	LDE225 0.75% [Part II] Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks. LDE225 0.75%
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 2D surface area, week 9 (LDE 0.75%, n=10)	NA Percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-25.5 Percent change in tumor measurement Standard Deviation 35.71	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 3D surface area, week 4	-19.3 Percent change in tumor measurement Standard Deviation 30.58	-23.1 Percent change in tumor measurement Standard Deviation 28.41	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) Total volume, week 9 (LDE 0.75%, n=10)	NA Percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-58.0 Percent change in tumor measurement Standard Deviation 37.24	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 2D surface area, week 4	-10.7 Percent change in tumor measurement Standard Deviation 24.23	-24.1 Percent change in tumor measurement Standard Deviation 30.69	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 2D surface area, week 6 (LDE 0.75%, n=21)	-36.3 Percent change in tumor measurement Standard Deviation 21.52	-33.3 Percent change in tumor measurement Standard Deviation 32.42	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 3D surface area, week 6 (LDE 0.75%, n=21)	-31.7 Percent change in tumor measurement Standard Deviation 31.19	-33.2 Percent change in tumor measurement Standard Deviation 23.63	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) 3D surface area, week 9 (LDE 0.75%, n=10)	NA Percent change in tumor measurement Standard Deviation NA Treatment duration for the LDE225 0.25% arm was 6 weeks only.	-38.0 Percent change in tumor measurement Standard Deviation 27.97	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) Total volume, week 4	-19.3 Percent change in tumor measurement Standard Deviation 57.85	-38.3 Percent change in tumor measurement Standard Deviation 39.75	—	—
Change From Baseline in Tumor Measurements (by Tumor) (Part II) Total volume, week 6 (LDE 0.75%, n=21)	-35.2 Percent change in tumor measurement Standard Deviation 60.52	-56.8 Percent change in tumor measurement Standard Deviation 23.70	—	—

Adverse Events

All Part I Participants

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

0.25% LDE225 [Part II]

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

0.75% LDE225 [Part II]

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Part I Participants n=8 participants at risk Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant.	0.25% LDE225 [Part II] n=3 participants at risk Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks.	0.75% LDE225 [Part II] n=7 participants at risk Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks.
Investigations HEPATIC ENZYME INCREASED	0.00% 0/8	0.00% 0/3	14.3% 1/7

Other adverse events

Other adverse events
Measure	All Part I Participants n=8 participants at risk Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant.	0.25% LDE225 [Part II] n=3 participants at risk Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks.	0.75% LDE225 [Part II] n=7 participants at risk Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were treated for 6 weeks and some BCCs were treated for 9 weeks.
Ear and labyrinth disorders EXTERNAL EAR INFLAMMATION	0.00% 0/8	0.00% 0/3	14.3% 1/7
Eye disorders CONJUNCTIVITIS	0.00% 0/8	0.00% 0/3	14.3% 1/7
General disorders FATIGUE	0.00% 0/8	0.00% 0/3	14.3% 1/7
Infections and infestations INFECTION	0.00% 0/8	0.00% 0/3	14.3% 1/7
Infections and infestations NASOPHARYNGITIS	12.5% 1/8	0.00% 0/3	0.00% 0/7
Injury, poisoning and procedural complications ARTHROPOD STING	12.5% 1/8	0.00% 0/3	0.00% 0/7
Injury, poisoning and procedural complications INJURY CORNEAL	0.00% 0/8	0.00% 0/3	14.3% 1/7
Injury, poisoning and procedural complications LIMB INJURY	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations BLOOD CHOLESTEROL INCREASED	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations BLOOD GLUCOSE INCREASED	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations BLOOD TRIGLYCERIDES INCREASED	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations GAMMA-GLUTAMYLTRANSFERASE INCREASED	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations GLUCOSE URINE PRESENT	12.5% 1/8	0.00% 0/3	0.00% 0/7
Investigations HAEMATOCRIT DECREASED	0.00% 0/8	33.3% 1/3	0.00% 0/7
Investigations HAEMOGLOBIN DECREASED	0.00% 0/8	33.3% 1/3	0.00% 0/7
Investigations WHITE BLOOD CELLS URINE POSITIVE	25.0% 2/8	0.00% 0/3	0.00% 0/7
Metabolism and nutrition disorders IRON DEFICIENCY	0.00% 0/8	33.3% 1/3	0.00% 0/7
Musculoskeletal and connective tissue disorders ARTHRALGIA	12.5% 1/8	0.00% 0/3	0.00% 0/7
Respiratory, thoracic and mediastinal disorders ASTHMA	0.00% 0/8	33.3% 1/3	0.00% 0/7
Skin and subcutaneous tissue disorders SKIN FISSURES	12.5% 1/8	0.00% 0/3	0.00% 0/7
Skin and subcutaneous tissue disorders SKIN IRRITATION	50.0% 4/8	0.00% 0/3	14.3% 1/7
Skin and subcutaneous tissue disorders URTICARIA	0.00% 0/8	0.00% 0/3	14.3% 1/7

Additional Information

Study Director

Novartis

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 or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER