PTCH1 or SMO Activated Solid and Hematologic TumorsPhase 2SIGNATURENCT02002689
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
A Phase 2 study testing LDE225 in people with PTCH1 or SMO Activated Solid and Hematologic Tumors, run by Novartis Pharmaceuticals at 10 sites in United States.
Trial summary
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- PTCH1 or SMO Activated Solid and Hematologic Tumors
- What is being tested?
- LDE225
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 10 sitesUnited States
- Study dates
- Feb 2014to Mar 2015
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient has confirmed diagnosis of a select solid tumor (except medulloblastoma, basal cell carcinoma and pancreatic adenocarcinoma) or hematological malignancy (except CML, ALL and AML).
- Patient has pre-identified tumor with a PTCH1 or SMO mutation.
- Patient has received at least one prior treatment for recurrent, metastatic and /or locally advanced disease and for whom no standard therapy options are anticipated to result in a durable remission.
- Patient has progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
Who cannot join
- Patients has received prior treatment with LDE225.
- Patients has neuromuscular disorders associated with elevated CK (i.e. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy) or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
- Patients has primary CNS tumor or CNS tumor involvement
- Patient has received chemotherapy or anticancer therapy ≤ 4 weeks prior to starting study drug
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Lurie Children's Hospital of Chicago Developmental Therapeutics
Chicago, Illinois, 60611
Cleveland Clinic Foundation Cleveland Clinic (19)
Cleveland, Ohio, 44195
Rocky Mountain Cancer Centers RMCC - Aurora
Greenwood Village, Colorado
Oncology Consultants Oncology Group
Houston, Texas, 77024
MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
Houston, Texas, 77030
Minnesota Oncology Hematology, P.A. Southdate Medical Center
Minneapolis, Minnesota, 55404
Intermountain Medical Center Intermountain Healthcare
Murray, Utah, 84157
University of California Davis Cancer Center UC Davis Cancer (3)
Sacramento, California, 95817
Seattle Cancer Care Alliance Skagit Valley Hospital
Seattle, Washington, 98109-1023
Sanford Research Sanford Health
Sioux Falls, South Dakota, 57104
About this study
Official title: Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module - 5 LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Being tested
- LDE225
- Keywords
- Solid tumor malignancy,, hematologic malignancy,, mutation, translocations,, signature,, PTCH1,, SMO,, LDE225,, endometrial cancer,, colon cancer,, bladder,, NSCLC
- Medical subject headings
- Basal Cell Nevus Syndrome, Hematologic Neoplasms, Endometrial Neoplasms, Colonic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LDE225Drug
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule
| Study group | Role | What they receive |
|---|---|---|
| LDE225 | Experimental | LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule. Receives: Drug: LDE225 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Summary of Overall Response (ORR) and Clinical Benefit (CBR)
Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well.
Measured: 16 weeks
Secondary outcomes (2)
Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set
Progression-free survival (PFS) is the time from the date of start of treatment to the date of event defined as the first documented progression or death due to any cause within 30 days of last dose. If a subject has not had an event, progression-free survival is censored at the date of last adequate tumor assessment.
Measured: 4 months
Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2013 (estimated)
- Study startFeb 2014
- Primary completionMar 2015
- Study completionMar 2015
Why it stopped: Low enrollment
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 PTCH1 or SMO Activated Solid and Hematologic TumorsStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02002689
- Sponsor's protocol ID
- CLDE225XUS20
- Acronym
- SIGNATURE
- Status last verified
- Apr 2016
- First submitted
- 1 Dec 2013
- Last updated
- 2 May 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module - 5 LDE225 for Patients With PTCH1 or SMO Mutated Tumors
- ClinicalTrials.gov ID
- NCT02002689
- Acronym / sponsor ID
- SIGNATURE / CLDE225XUS20
- Keywords
- Solid tumor malignancy,, hematologic malignancy,, mutation, translocations,, signature,, PTCH1,, SMO,, LDE225,, endometrial cancer,
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2014
- Primary completion
- Mar 2015 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 6 Dec 2013 (estimated)
- Results
- Posted 2 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LDE225 | Experimental | Drug: LDE225LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patient has confirmed diagnosis of a select solid tumor (except medulloblastoma, basal cell carcinoma and pancreatic adenocarcinoma) or hematological malignancy (except CML, ALL and AML).
- Patient has pre-identified tumor with a PTCH1 or SMO mutation.
- Patient has received at least one prior treatment for recurrent, metastatic and /or locally advanced disease and for whom no standard therapy options are anticipated to result in a durable remission.
- Patient has progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
Key exclusion criteria (4 listed)
- Patients has received prior treatment with LDE225.
- Patients has neuromuscular disorders associated with elevated CK (i.e. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy) or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
- Patients has primary CNS tumor or CNS tumor involvement
- Patient has received chemotherapy or anticancer therapy ≤ 4 weeks prior to starting study drug
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 |
|---|---|---|
| LDE225LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule | Drug | LDE225 |
Primary outcome
Summary of Overall Response (ORR) and Clinical Benefit (CBR)
Time frame: 16 weeks
Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well.
2 secondary outcomes
Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set
Time frame: 4 months
Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months
Time frame: 4 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LDE225 in people with PTCH1 or SMO Activated Solid and Hematologic Tumors, run by Novartis Pharmaceuticals at 10 sites in United States.
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 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 2 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. 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. 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 2 May 2016). 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?
Summary of Overall Response (ORR) and Clinical Benefit (CBR) (time frame: 16 weeks). 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 2 May 2016.