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Nevoid Basal Cell Carcinoma Syndrome

Basal Cell Carcinoma, Multiple

Dermatologic Agents

All Drugs and Chemicals

Verteporfin

Photosensitizing Agents

verteporfin PDT

Herma Neyndorff

The purpose of the two studies is to determine whether an experimental therapy using a photoactive drug, verteporfin, in combination with direct light exposure of basal cell carcinoma of the skin can safely eliminate these skin tumors.

Basal cell carcinoma (BCC) of the skin is the most common type of cancer that can be treated with various therapies including surgical removal. A number of factors can lead to the development of multiple BCC of the skin, including genetic disorders (e.g., nevoid basal cell carcinoma syndrome). Treatment of multiple BCC becomes much more challenging. In these trials, the experimental therapy: verteporfin PDT is compared to placebo PDT. PDT or photodynamic therapy in these studies is the combination of the photoactive drug verteporfin (given intravenously) and red light exposure of skin tumors.

The primary objective is to assess whether verteporfin PDT can completely eliminate multiple BCCs. Eligible subjects will have at least 3 BCCs. Study subjects won't know which treatment they have been been assigned to - this is done randomly (like flipping a coin). After study treatment, the BCCs in each subject will be closely observed and toward the end of the study all will be surgically removed and examined to verify response to study treatment. Safety will be assessed by testing blood samples before and after study treatment, and analyzing adverse events and skin reactions to the study treatment.

Two Studies to Determine if Verteporfin PDT is Effective & Safe in Treating Multiple Basal Cell Carcinoma of the Skin.

A Randomized, Placebo-Controlled, Masked, Multicenter Phase III Study Of Photodynamic Therapy With Verteporfin For Injection (VFI) For The Treatment Of Multiple Basal Cell Carcinoma

Novartis

QLT Inc.

Eye Diseases

Nervous System Diseases

Neoplasm Metastasis

Myopia

Neovascularization, Pathologic

Vision, Low

Choroidal Neovascularization

Vision Disorders

Neoplastic Processes

Refractive Errors

Metaplasia

Choroid Diseases

Uveal Diseases

Sensation Disorders

Neurologic Manifestations

Antineoplastic Agents

Ranibizumab

Angiogenesis Inhibitors

Growth Inhibitors

0.5 mg ranibizumab driven by visual acuity stability criteria

0.5 mg ranibizumab driven by disease activity criteria

0.5 mg ranibizumab (intravitreal injections)

Ranibizumab 0.5mg

Ranibizumab 0.5 mg

Verteporfin for intravenous injection delivered by intravenous infusion followed by the light application

Verteporfin PDT

Novartis Pharmaceuticals

This study was designed to evaluate the efficacy and safety of two different dosing regimens of 0.5 mg ranibizumab given as intravitreal injection in comparison to verteporfin PDT in patients with visual impairment due to choroidal neovascularization secondary to pathologic myopia (PM)

This was a phase III, multi-center, randomized, double-masked, active-controlled study comparing 0.5 mg ranibizumab vs. vPDT therapy. The study included 15 scheduled visits over 12 months, and there were to be two additional visits (2a, 3a) for subset of patients in whom PK analysis were performed.

There were 3 periods in this study: Screening period-from Day -14 to Baseline; Treatment period-from Baseline to Month 11; Follow-up period-from Month 11 to Month 12 Patients entered the 11 months Treatment period at Visit 2 (Day 1) if eligibility criteria were met and were randomized in three treatment groups Group I ranibizumab 0.5 mg driven by VA stability criteria or Group II ranibizumab 0.5 mg driven by disease activity criteria or Group III vPDT (randomization ratio of 2:2:1) and received first treatment of either a ranibizumab injection and sham vPDT or sham injection and active vPDT and will return to the clinical center within 7 days to undergo safety assessments as well as assessments of the effect of treatment by the evaluating investigator. The following visits were performed at one month intervals starting at Visit 4 and continuing through Visit 14. At all monthly visits (at/from Month 2 for group I, at/from Month 1 for group II and at/from Month 3 for group III) the decision for treatment were made by the evaluating investigator based on the VA stability criteria and on the disease activity criteria. At Month 3 (visit 6) and at all following monthly visits for all three groups one of the three options can recommended by evaluating investigator: a) ranibizumab 0.5 mg, b) ranibizumab 0.5 mg + vPDT; c) vPDT. The treating investigator were then perform treatment based on randomization and masking requirements.

At each monthly visit, patients had a safety evaluation by the evaluating investigator prior to study treatment, consisting of visual acuity measurements, ophthalmic examinations and evaluation of adverse events and vital signs. Routine hematology, chemistry, and urinalysis profiles were obtained at Visit 6, 9 and 12 (Month 3, 6 and 9). At Month 12 several procedures and assessments were performed which are required at study completion visit.

Efficacy and Safety of Ranibizumab 0.5 vs Verteporfin PDT in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia

A 12-month, Phase III, Randomized, Double-masked, Multicenter, Active-controlled Study to Evaluate the Efficacy and Safety of Two Individualized Regimens of 0.5mg Ranibizumab vs. Verteporfin PDT in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Best corrected visual acuity (BCVA) was tested using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity (VA) charts testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

Best corrected visual acuity (BCVA) was tested using the early treatment diabetic retinopathy study (ETDRS) VA testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

Between treatment comparison of 0.5 mg ranibizumab intravitreal injections driven by disease activity re-treatment criteria (Group II) versus 0.5 mg ranibizumab intravitreal injections driven by visual acuity stability criteria (Group I) based on the average BCVA change from Baseline to Month 1 through Month 6.

Best corrected visual acuity (BCVA) was tested using the ETDRS VA testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

Between treatment comparison of 0.5 mg ranibizumab intravitreal injections driven by disease activity re-treatment criteria (Group II) versus 0.5 mg ranibizumab intravitreal injections driven by visual acuity stability criteria (Group I) based on the average BCVA change from Baseline to Month 1 through Month 12

Best corrected visual acuity (BCVA) was tested using the ETDRS VA testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

ETDRS VA testing protocol. VA measurements were taken in a sitting position at an initial test distance of 4 meters using ETDRS charts. The overall BCVA score was calculated using the BCVA worksheet, which was kept in the source data and the score was recorded in the eCRF.

Central sub-field thickness (CSFT) is a variable assessed via Optical Coherence Tomography (OCT). OCT was performed prior to any study drug administration to assess presence of intra, subretinal fluid, or increase of CSFT.

CNV leakage is assessed via fluorescein angiography (center involvement) category: definite, questionable, absent, can't grade, and missing.

The VFQ-25 consists of 25 vision related questions across 11 vision related subscales, including general vision, ocular pain, near activities, distance activities, social function, mental health, role difficulties, dependency, driving, color vision and peripheral vision, and a general health rating. Items are converted to a 0-100 scale on each subscale and for the composite score where higher scores represents better functioning.

To assess treatment pattern with ranibizumab

Analysis was done in the safety set: consisted of all patients who received at least one application of study treatment and had at least one post-Baseline safety assessment. Patients were analyzed according to treatment received. The statement that a patient had no AEs also constitutes a safety assessment.

One patient randomized to vPDT Group III received one ranibizumab injection prior to Month 3; this patient was analyzed under Group II in Safety Set"

Group I

Group II

Visudyne PDT with 0.5mg ranibizumab from month 3

Group III With 0.5mg Ranibizumab

Visudyne PDT without 0.5mg ranibizumab from month 3

Group III Without 0.5mg Ranibizumab

verteporfin PDT (driven by disease activity). Patients received vPDT on Day 1. From Month 3, based on disease activity vPDT, Ranibizumab 0.5 mg, or combo of Ranibizumab and vPDT was selected for treatment

Group III

Total

Age, Continuous

Sex: Female, Male

Randomized Set: consisted of all randomized patients. Patients were considered randomized when they had been given a randomization number.

Study Director

2 one-sided hypotheses were tested under the Type I error rate of 0.025, using the Hochberg procedure: H01: μRanibizumab-I - μvPDT ≤ 0 vs. HA1: μRanibizumab-I - μvPDT \> 0 and H02: μRanibizumab-II - μvPDT ≤ 0 vs. HA2: μRanibizumab-II - μvPDT \> 0, where μRanibizumab-I, μRanibizumab-II and μvPDT were the means of the primary efficacy variable for ranibizumab treatment Group I, ranibizumab treatment Group II, and vPDT treatment Group III, respectively.

The following 2 one-sided hypotheses were tested under the Type I error rate of 0.025, using the Hochberg procedure:

H01: μRanibizumab-I - μvPDT ≤ 0 vs. HA1: μRanibizumab-I - μvPDT \> 0 and H02: μRanibizumab-II - μvPDT ≤ 0 vs. HA2: μRanibizumab-II - μvPDT \> 0, where μRanibizumab-I, μRanibizumab-II and μvPDT were the means of the primary efficacy variable for ranibizumab treatment Group I, ranibizumab treatment Group II, and vPDT treatment Group III, respectively.

Baseline

Average Month 1 to Month 3

Full Analysis Set (FAS): consisted of all patients to whom study treatment were assigned. Following the intent-to-treat principle, patients were analyzed according to the treatment group they were assigned to at randomization.

Change From Baseline BCVA to the Average Level of BCVA Over All Monthly Assessments From Month 1 to Month 3

Average Month 1 to Month 6

Full Analysis Set (FAS): Following the intent-to-treat principle, patients were analyzed according to the treatment group they were assigned to at randomization.

FAS, modified last observation carried forward (LOCF), for comparison between ranibizumab treatment groups.): consisted of all patients to whom study treatment were assigned.

Change From Baseline BCVA to the Average Level of BCVA Over All Monthly Assessments From Month 1 to Month 6

Average Month 1 to Month 12

verteporfin PDT (driven by disease activity). Patients received vPDT on Day 1. From Month 3, based on disease activity vPDT, Ranibizumab 0.5 mg, or combo of Ranibizumab and vPDT was selected for treatment.

FAS, modified LOCF for comparison between ranibizumab treatment groups.

The Average Change in BCVA Score From Baseline to Month 1 Through Month 12

Month 3

Month 6

Month 12

Mean Change From Baseline in Visual Acuity Over Time

Gained≥15 letters or reached 84 letters-Month 3

Gained≥10 letters or reached 84 letters-Month 3

Gained≥15 letters or reached 84 letters-Month 6

Gained≥10 letters or reached 84 letters-Month 6

Gained≥15 letters or reached 84 letters-Month 12

Gained≥10 letters or reached 84 letters-Month 12

Categorized BCVA Changes at Months 3, 6 and 12 Compared With Baseline for the Study Eye

Mean Change From Baseline Over Time in Central Sub-field Thickness (CSFT)

Baseline - Definite

Baseline - Questionable

Baseline - Absent

Baseline - Can't Grade

Baseline - Missing

Month 12 - Definite

Month 12 - Questionable

Month 12 - Absent

Month 12 - Can't Grade

Month 12 - Missing

Number of Patients With CNV Leakage (Center Involvement) in the Study Eye at Baseline and Month 12

NEI-VFQ-25 - Change From Baseline to Month 3, 6 and 12

verteporfin PDT with 0.5 mg ranibizumab from month 3

verteporfin PDT without 0.5 mg ranibizumab from month 3

Safety Set: all patients who received at least one application of study treatment \& had at least one post-Baseline safety assessment. Patients were analyzed according to treatment received. One patient randomized to vPDT Group III received one ranibizumab injection prior to Month 3; this patient was analyzed under Group II in Safety Set

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Two Studies to Determine if Verteporfin PDT is Effective & Safe in Treating Multiple Basal Cell Carcinoma of the Skin.	NCT00049959	Basal Cell Carc... Nevoid Basal Ce... Gorlin Syndrome	verteporfin PDT	18 Years - 90 Years	QLT Inc.
Two Studies to Determine if Verteporfin PDT is Effective & Safe in Treating Multiple Basal Cell Carcinoma of the Skin.	NCT00049959	Basal Cell Carc... Nevoid Basal Ce... Gorlin Syndrome	verteporfin PDT	18 Years - 90 Years	QLT Inc.
Efficacy and Safety of Ranibizumab 0.5 vs Verteporfin PDT in Patients With Visual Impairment Due to Choroidal Neovascularization Secondary to Pathologic Myopia	NCT01922102	Visual Impairme...	Ranibizumab 0.5... Ranibizumab 0.5... Verteporfin PDT	18 Years -	Novartis

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