Basal Cell CarcinomaPhase 2/3NCT03070691
Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
A Phase 2/3 study testing LDE225B in people with Basal Cell Carcinoma, run by Novartis Pharmaceuticals at 35 sites in 10 countries.
Trial summary
This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Carcinoma
- What is being tested?
- LDE225B
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 35 sitesin 10 countries
- Study dates
- Start not givento not given
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult greater than 18 years old, male or female.
- Patient must be able to understand and communicate with the investigator and comply with the requirements of the study.
- Typical presentation of NBCCS in the opinion of the investigator
- At least one of the major clinical criteria of NBCCS.
- Multiple BCCs during the screening period
Who cannot join
- Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator.
- Prior exposure to LDE225.
- Use of systemic treatment for BCC in the 4 weeks prior to Baseline.
- Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline.
- Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer.
- Clinically significant medical condition, as per judgment of the investigator.
Show all 10Show fewer
- History of hypersensitivity to any of the ingredient of the study drug.
- Pregnant or nursing (lactating) women
- Women of child-bearing potential and fertile males, UNLESS they are using two birth control methods
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
35 sites in 10 countries.
35 sites in 10 countries
Germany15 sites
Novartis Investigative Site
Berlin
Novartis Investigative Site
Bonn
Novartis Investigative Site
Dresden
Novartis Investigative Site
Essen
Novartis Investigative Site
Frankfurt
Novartis Investigative Site
Heidelberg
Novartis Investigative Site
Jena
Novartis Investigative Site
Kiel
Novartis Investigative Site
Lübeck
Novartis Investigative Site
Mainz
Novartis Investigative Site
Munich
Novartis Investigative Site
Münster
Novartis Investigative Site
Recklinghausen
Novartis Investigative Site
Tübingen
Novartis Investigative Site
Würzburg
Italy5 sites
Novartis Investigative site
Catania
Novartis Investigative site
L’Aquila
Novartis Investigative site
Naples
Novartis Investigative site
Padova
Novartis Investigative site
Siena
United Kingdom3 sites
Novartis Investigative site
Glasgow
Novartis Investigative site
London
Novartis Investigative site
Manchester
Belgium2 sites
Novartis Investigative site
Brussels
Novartis Investigative site
Leuven
Canada2 sites
Novartis Investigative site
Sainte-Foy, Quebec
Novartis Investigative site
Waterloo, Ontario
Greece2 sites
Novartis Investigative Site
Athens
Novartis Investigative site
Thessaloniki
Spain2 sites
Novartis Investigative site
Barcelona
Novartis Investigative site
Madrid
Switzerland2 sites
Novartis Investigative site
Lausanne
Novartis Investigative site
Zurich
About this study
Official title: A Randomized, Double-blind, Vehicle-controlled, Multicenter Trial of Topically Administered LDE225 Cream (0.75% Bid) to Evaluate Clearance of Basal Cell Carcinoma in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome
This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Treatment success is defined as complete clinical clearance and complete histological clearance in BCCs.
- Conditions
- Basal Cell Carcinoma
- Being tested
- LDE225B, Vehicle
- Keywords
- Basal cell carcinoma, Nevoid Basal Cell Carcinoma Syndrome, NBCCS, Gorlin syndrome, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, BCNS, Hedgehog pathway inhibitor, topical, Smoothened
- Medical subject headings
- Carcinoma, Basal Cell, Basal Cell Nevus Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LDE225BDrug
Cream, 2x daily
VehicleDrug
cream, 2x daily
| Study group | Role | What they receive |
|---|---|---|
| LDE225 0.75% cream | Active comparator | Not described Receives: Drug: LDE225B |
| Vehicle | Placebo comparator | Not described Receives: Drug: Vehicle |
What is being measured
The primary outcome is the main question the study is designed to answer.
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs
Measured: 4 weeks after LDE225 treatment
Secondary outcomes (4)
To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS
Measured: Treatment phase and up to 4 weeks after LDE225 treatment
To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS
Measured: Treatment phase and up to 4 weeks after LDE225 treatment
To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma
Measured: 4 weeks after LDE225 treatment
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue
Measured: 4 weeks after LDE225 treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Basal Cell CarcinomaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03070691
- Sponsor's protocol ID
- CLDE225B2307
- Status last verified
- Feb 2017
- First submitted
- 6 Jul 2010
- Last updated
- 5 May 2017
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Vehicle-controlled, Multicenter Trial of Topically Administered LDE225 Cream (0.75% Bid) to Evaluate Clearance of Basal Cell Carcinoma in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome
- ClinicalTrials.gov ID
- NCT03070691
- Acronym / sponsor ID
- CLDE225B2307
- Conditions
- Basal Cell Carcinoma
- Keywords
- Basal cell carcinoma, Nevoid Basal Cell Carcinoma Syndrome, NBCCS, Gorlin syndrome, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, BCNS, Hedgehog pathway inhibitor, topical, Smoothened
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 3 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Treatment success is defined as complete clinical clearance and complete histological clearance in BCCs.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LDE225 0.75% cream | Active comparator | Drug: LDE225B |
| Vehicle | Placebo comparator | Drug: Vehicle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adult greater than 18 years old, male or female.
- Patient must be able to understand and communicate with the investigator and comply with the requirements of the study.
- Typical presentation of NBCCS in the opinion of the investigator
- At least one of the major clinical criteria of NBCCS.
- Multiple BCCs during the screening period
Key exclusion criteria (10 listed)
- Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator.
- Prior exposure to LDE225.
- Use of systemic treatment for BCC in the 4 weeks prior to Baseline.
- Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline.
- Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LDE225BCream, 2x daily | Drug | LDE225 0.75% cream |
| Vehiclecream, 2x daily | Drug | Vehicle |
Primary outcome
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs
Time frame: 4 weeks after LDE225 treatment
4 secondary outcomes
To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS
Time frame: Treatment phase and up to 4 weeks after LDE225 treatment
To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS
Time frame: Treatment phase and up to 4 weeks after LDE225 treatment
To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma
Time frame: 4 weeks after LDE225 treatment
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue
Time frame: 4 weeks after LDE225 treatment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
35 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing LDE225B in people with Basal Cell Carcinoma, run by Novartis Pharmaceuticals at 35 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 5 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in Germany, Italy, United Kingdom, Belgium, Canada and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs (time frame: 4 weeks after LDE225 treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2017.