Sporadic Superficial and Nodular Skin Basal Cell CarcinomasPhase 2NCT01033019

To Evaluate the Safety, Local Tolerability, PK and PD of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas (sBCC)

A Phase 2 study testing LDE225 0.75% in people with Sporadic Superficial and Nodular Skin Basal Cell Carcinomas, run by Novartis Pharmaceuticals at 4 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This was a double-blinded, randomized, vehicle-controlled study in sporadic superficial BCC (sBCC) and nodular BCC (nBCC) patients which consisted of a 21-day screening period, a treatment period of 6 weeks (topical 0.75% LDE225 cream application b.i.d) ending with post treatment biopsies, as safety visit one week… Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Sporadic Superficial and Nodular Skin Basal Cell Carcinomas
What is being tested?: LDE225 0.75%
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 25 participants
Where: 4 sitesin 2 countries
Study dates: Dec 2009to Feb 2011
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients with one histologically confirmed superficial and nodular basal cell carcinoma (8-20 mm) eligible for surgical excision on selected body areas (scalp, arm, frontal trunk, posterior trunk, upper legs)

Who cannot join

Previous treatment of the sBCC that are selected for treatment.
Any systemic treatment which is known to affect BCCs esp. cytostatic treatments, retinoids and photodynamic treatments.
Dark-skinned persons whose skin color prevents readily assessment of skin reactions
Other protocol defined Incl./Excl. criteria may apply.

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

Australia2 sites

Novartis Investigative Site
Benowa, Queensland, 4217
Novartis Investigative Site
Woolloongabba, Queensland, 4102

Other trials in Australia

Austria2 sites

Novartis Investigative Site
Graz, A-8036
Novartis Investigative Site
Vienna, 1090

Other trials in Austria

About this study

Official title: Double-blind, Randomized, Vehicle-controlled Proof of Concept Study on the Efficacy, Safety, Local Tolerability, PK and Pharmacodynamics of Multiple Topical Administrations of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas

Conditions: Sporadic Superficial and Nodular Skin Basal Cell Carcinomas
Being tested: LDE225 0.75%, Vehicle
Keywords: Basal cell carcinomas,, sporadic,, superficial and nodular,, skin
Medical subject headings: Carcinoma, Basal Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

LDE225 0.75%Drug
0.75% cream
VehicleDrug
matching placebo cream to 0.75% LDE225 cream

Study groups in NCT01033019
Study group	Role	What they receive
LDE225 0.75%	Experimental	Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks. Receives: Drug: LDE225 0.75%
Vehicle	Placebo comparator	Participants topically applied matching placebo cream twice daily for 6 weeks. Receives: Drug: Vehicle

What is being measured

The primary outcome is the main question the study is designed to answer.

Clinical Evaluation of sBCCs Tumors
The 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).
Measured: Day 43

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2009Feb 2011

First posted16 Dec 2009 (estimated)
Study startDec 2009
Primary completionFeb 2011
Study completionFeb 2011

Why it stopped: The data from participants with sBCCs showed insufficient efficacy with current formulation and treatment conditions.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Sporadic Superficial and Nodular Skin Basal Cell CarcinomasStudies 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
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01033019
Sponsor's protocol ID: CLDE225B2204
Other IDs: 2009-013665-26, EudraCT 2009-013665-26, 2009
Status last verified: Oct 2015
First submitted: 15 Dec 2009
Last updated: 30 Oct 2015
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Study at a glance

From the registry record, last updated by the sponsor on 30 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Double-blind, Randomized, Vehicle-controlled Proof of Concept Study on the Efficacy, Safety, Local Tolerability, PK and Pharmacodynamics of Multiple Topical Administrations of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas
ClinicalTrials.gov ID: NCT01033019
Acronym / sponsor ID: CLDE225B2204
Conditions: Sporadic Superficial and Nodular Skin Basal Cell Carcinomas
Keywords: Basal cell carcinomas,, sporadic,, superficial and nodular,, skin
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The data from participants with sBCCs showed insufficient efficacy with current formulation and treatment conditions.
Enrolment: 25 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Dec 2009
Primary completion: Feb 2011 (actual)
Study completion: Feb 2011 (actual)
First posted: 16 Dec 2009 (estimated)
Results: Posted 30 Oct 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
LDE225 0.75%	Experimental	Drug: LDE225 0.75%Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
Vehicle	Placebo comparator	Drug: VehicleParticipants topically applied matching placebo cream twice daily for 6 weeks.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

Patients with one histologically confirmed superficial and nodular basal cell carcinoma (8-20 mm) eligible for surgical excision on selected body areas (scalp, arm, frontal trunk, posterior trunk, upper legs)

Key exclusion criteria (4 listed)

Previous treatment of the sBCC that are selected for treatment.
Any systemic treatment which is known to affect BCCs esp. cytostatic treatments, retinoids and photodynamic treatments.
Dark-skinned persons whose skin color prevents readily assessment of skin reactions
Other protocol defined Incl./Excl. criteria may apply.

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
LDE225 0.75%0.75% cream	Drug	LDE225 0.75%
Vehiclematching placebo cream to 0.75% LDE225 cream	Drug	Vehicle

Primary outcome

Clinical Evaluation of sBCCs Tumors
Time frame: Day 43
The 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).

Results and safety

Results posted to the registry on 30 Oct 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
LDE225 0.75%	16	15
Vehicle	8	8

Primary outcome: Clinical Evaluation of sBCCs Tumors

Time frame: Day 43. Number. Unit: Participants

Measure	LDE225 0.75% n = 16	Vehicle n = 8
Complete response	3	1
Partial response	9	4
No response	4	3

Population analysed: Efficacy analysis set: the efficacy analysis set included all randomized participants with evaluable data.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
LDE225 0.75% - sBCC	0 of 16 (0%)	8 of 16 (50%)	Not reported
Vehicle - sBCC	0 of 8 (0%)	5 of 8 (62.5%)	Not reported
LDE225 0.75% - nBCC	0 of 1 (0%)	1 of 1 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2015.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related InfoLink provided by the sponsor.

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

4 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LDE225 0.75% in people with Sporadic Superficial and Nodular Skin Basal Cell Carcinomas, run by Novartis Pharmaceuticals at 4 sites in 2 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 1 inclusion and 4 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: stopped early, as of the sponsor's last update on 30 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Australia, Austria. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Oct 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Clinical Evaluation of sBCCs Tumors (time frame: Day 43). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01033019.

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 30 Oct 2015.