Cutaneous T-Cell LymphomaPhase 2CTCLNCT01637090
Everolimus in Treating Cutaneous T-cell Lymphoma
A Phase 2 study testing Everolimus in people with Cutaneous T-Cell Lymphoma, run by Adam Lerner at 1 site in United States.
Trial summary
CTCL is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemo. This study will investigate the effect of everolimus as treatment for recurrent or refractory CTCL. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cutaneous T-Cell Lymphoma
- What is being tested?
- Everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2012to May 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
- Clinically and histologically confirmed diagnosis of CTCL (at least stage IB for mycosis fungoides and Sézary syndrome, and T2 and/or refractory to at least one prior treatment for CTCL other than mycosis fungoides/Sézary syndrome)
- Relapsed or refractory disease after at least one standard systemic treatment including extracorporeal photopheresis (ECP), oral bexarotene, interferon, HDAC inhibitors
- ≥18 years old
- WHO performance status ≤ 2
- Life expectancy ≥ 6 months
- ANC ≥ 1.5 x 109/L, Platelets ≥ 100 x 109/L, Hb >9 g/dL
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- Serum bilirubin ≤ 1.5 x ULN
- ALT and AST ≤ 2.5x ULN (≤ 5x ULN in patients with liver metastases)
- INR ≤1.5 (Anticoagulation is allowed if target INR ≤ 1.5 on a stable dose of warfarin or on a stable dose of LMW heparin for >2 weeks at time of randomization)
- Serum creatinine ≤ 1.5 x ULN
- Fasting serum cholesterol ≤300 mg/dL OR ≤7.75 mmol/L
- Fasting triglycerides ≤ 2.5 x ULN.
Who cannot join
- Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of stating study drug
- Treatment with any investigational drug within the past 4 weeks
- Patients, who have had major surgery or significant traumatic injury within 4 weeks of starting study drug, patients who have not recovered from the side effects of any major surgery, or patients that may require major surgery during the study
- Patients receiving chronic, systemic treatment with corticosteroids or any immunosuppressive agent other than topical or inhaled corticosteroids
- Patients receiving immunization with attenuated live vaccines within one week of study entry or during study period
- Uncontrolled brain or leptomeningeal metastases, including patients who continue to require glucocorticoids for brain or leptomeningeal metastases
Show all 23Show fewer
- Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
- Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
- Symptomatic congestive heart failure of New York heart Association Class III or IV
- unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease
- severely impaired lung function as defined as spirometry and DLCO that is 50% of the normal predicted value and/or O2 saturation that is 88% or less at rest on room air
- uncontrolled diabetes as defined by fasting serum glucose >1.5 x ULN (Note: Optimal glycemic control should be achieved before starting trial therapy.)
- active (acute or chronic) or uncontrolled severe infections
- liver disease such as cirrhosis or severe hepatic impairment (Child-Pugh class C).
- A known history of HIV seropositivity
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus
- Patients with an active, bleeding diathesis
- Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. Adequate contraception must be used throughout the trial and for 8 weeks after the last dose of study drug, by both sexes.
- Male patient whose sexual partner(s) are WOCBP who are not willing to use adequate contraception, during the study and for 8 weeks after the end of treatment
- Patients who have received prior treatment with an mTOR inhibitor
- Patients with a known hypersensitivity to everolimus or other rapamycins or to its excipient
- History of noncompliance to medical regimens
- Patients unwilling to or unable to comply with the protocol
Trial locations and contacts
1 site in 1 country.
About this study
Official title: PHASE II TRIAL OF THE mTOR INHIBITOR EVEROLIMUS IN RELAPSED OR REFRACTORY CUTANEOUS T-CELL LYMPHOMA (CTCL)
CTCL is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemo. This study will investigate the effect of everolimus as treatment for recurrent or refractory CTCL. Participation in this study will last as long as the study doctor believes disease has not gotten worse, and patients continue to tolerate the study medication for a maximum of 1 year. Once off the treatment, patients will be followed for two years.
Read the detailed description
Cutaneous T-cell lymphoma is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemotherapy. This study will investigate the effect of everolimus as a treatment for patients diagnosed with CTCL that has either not responded to previous treatments or has recurred despite previous treatments. Everolimus is the common name for the commercial drug Afinitor® (Novartis). It is approved by the U.S. Food and Drug Administration (FDA) for use in kidney and brain cancer. In several different forms of lymphomas, everolimus is used as an investigational drug, which means it has not been approved by the FDA for this group of diseases.
Everolimus blocks a protein (mTOR) that helps cells and tumors to grow. Earlier studies have indicated that the drug everolimus may work against lymphomas including cutaneous T-cell lymphomas. Participation in this study will last as long as the study doctor believes disease has not gotten worse, and patients continue to tolerate the study medication for a maximum of 1 year. Once off the treatment, patients will be followed for two years.
- Conditions
- Cutaneous T-Cell Lymphoma
- Being tested
- Everolimus
- Medical subject headings
- Lymphoma, T-Cell, Cutaneous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EverolimusDrug
Also called Afinitor
The study drug everolimus will be self-administered (by the patients themselves). The investigator will instruct the patient to take the study drug exactly as specified in the protocol. Everolimus should be administered orally once daily, preferably in the morning, at the same time every day with our without food. Everolimus tablets should be swallowed whole with a glass of water. The tablets must not be chewed or crushed.
Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.
If vomiting occurs, no attempt should be made to replace the vomited dose. All dosages prescribed and dispensed to the patient and all dose changes during the study must be recorded.
| Study group | Role | What they receive |
|---|---|---|
| all patients on study | Experimental | This will be a prospective, phase II non-randomized, open label study of single agent everolimus for the treatment of CTCL recurrent or refractory to at least one previous treatment other than topical medication. The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin examinations and assessment of tumor burden in tissue and blood.
This study will be conducted in 2 stages. During stage 1 we will enroll a maximum of 11 subjects to evaluate response rate and will only continue to stage 2, if we observe two or more responses. During stage 2, we will expand the study to an overall N of 28 patients. Receives: Drug: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of Treatment
Determine the efficacy of everolimus in the treatment of CTCL as overall response rate (ORR)
Measured: 12 months after beginning treatment
Secondary outcomes (4)
Time to Response
Determine time to response (TTR)/duration of objective response (DOR)
Measured: three months
Progression-free Survival
Determine progression-free survival of CTCL patients treated with everolimus
Measured: two years after discontinuing study treatment
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Determine the adverse event profile and tolerability of everolimus in patients with CTCL
Measured: Up to one year
Effect of mTOR on Tumors
Determine mTOR (mammilian target of rapamycin) pathway activation and number of regulatory T cells (Tregs) in pre-treated tumor tissue and evaluate changes following treatment
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2012 (estimated)
- Study startJun 2012
- Primary completionMay 2015
- Study completionMay 2015
Why it stopped: Poor 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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cutaneous T-Cell LymphomaStudies of the same condition, open and under way.
- 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
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Adam Lerner
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Sponsor-investigator, Adam Lerner, Boston Medical Center
- Investigators
- Adam Lerner, MD, Principal investigator, Boston University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01637090
- Sponsor's protocol ID
- H-30213
- Acronym
- CTCL
- Status last verified
- Nov 2016
- First submitted
- 2 Apr 2012
- Last updated
- 12 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Banchi M, Cox MC, Bandini A, Orlandi P, Tacchi C, Stefanelli F, Chericoni S, Bocci G. Linifanib alone and in combination with metronomic chemotherapy is active on cutaneous T-cell lymphoma cells by targeting the AKT/mTOR signaling pathway. Invest New Drugs. 2025 Feb;43(1):135-146. doi: 10.1007/s10637-024-01501-8. Epub 2025 Jan 17.
PubMed 39821757
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PHASE II TRIAL OF THE mTOR INHIBITOR EVEROLIMUS IN RELAPSED OR REFRACTORY CUTANEOUS T-CELL LYMPHOMA (CTCL)
- ClinicalTrials.gov ID
- NCT01637090
- Acronym / sponsor ID
- CTCL / H-30213
- Conditions
- Cutaneous T-Cell Lymphoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Adam LernerUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Jun 2012
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 10 Jul 2012 (estimated)
- Results
- Posted 31 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
CTCL is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemo. This study will investigate the effect of everolimus as treatment for recurrent or refractory CTCL. Participation in this study will last as long as the study doctor believes disease has not gotten worse, and patients continue to tolerate the study medication for a maximum of 1 year. Once off the treatment, patients will be followed for two years.
Detailed description
Cutaneous T-cell lymphoma is a rare form of lymphoma of the skin. While early stages are usually confined to the skin, later stages may spread to blood, lymph nodes and other organs. At this point, patients usually require systemic chemotherapy. This study will investigate the effect of everolimus as a treatment for patients diagnosed with CTCL that has either not responded to previous treatments or has recurred despite previous treatments. Everolimus is the common name for the commercial drug Afinitor® (Novartis). It is approved by the U.S. Food and Drug Administration (FDA) for use in kidney and brain cancer. In several different forms of lymphomas, everolimus is used as an investigational drug, which means it has not been approved by the FDA for this group of diseases.
Everolimus blocks a protein (mTOR) that helps cells and tumors to grow. Earlier studies have indicated that the drug everolimus may work against lymphomas including cutaneous T-cell lymphomas. Participation in this study will last as long as the study doctor believes disease has not gotten worse, and patients continue to tolerate the study medication for a maximum of 1 year. Once off the treatment, patients will be followed for two years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| all patients on study | Experimental | Drug: EverolimusThis will be a prospective, phase II non-randomized, open label study of single agent everolimus for the treatment of CTCL recurrent or refractory to at least one previous treatment other than topical medication. The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin examinations and assessment of tumor burden in tissue and blood. This study will be conducted in 2 stages. During stage 1 we will enroll a maximum of 11 subjects to evaluate response rate and will only continue to stage 2, if we observe two or more responses. During stage 2, we will expand the study to an overall N of 28 patients. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Clinically and histologically confirmed diagnosis of CTCL (at least stage IB for mycosis fungoides and Sézary syndrome, and T2 and/or refractory to at least one prior treatment for CTCL other than mycosis fungoides/Sézary syndrome)
- Relapsed or refractory disease after at least one standard systemic treatment including extracorporeal photopheresis (ECP), oral bexarotene, interferon, HDAC inhibitors
- ≥18 years old
- WHO performance status ≤ 2
- Life expectancy ≥ 6 months
Key exclusion criteria (23 listed)
- Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of stating study drug
- Treatment with any investigational drug within the past 4 weeks
- Patients, who have had major surgery or significant traumatic injury within 4 weeks of starting study drug, patients who have not recovered from the side effects of any major surgery, or patients that may require major surgery during the study
- Patients receiving chronic, systemic treatment with corticosteroids or any immunosuppressive agent other than topical or inhaled corticosteroids
- Patients receiving immunization with attenuated live vaccines within one week of study entry or during study period
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 |
|---|---|---|
| EverolimusAlso: AfinitorThe study drug everolimus will be self-administered (by the patients themselves). The investigator will instruct the patient to take the study drug exactly as specified in the protocol. Everolimus should be administered orally once daily, preferably in the morning, at the same time every day with our without food. Everolimus tablets should be swallowed whole with a glass of water. The tablets must not be chewed or crushed. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity. If vomiting occurs, no attempt should be made to replace the vomited dose. All dosages prescribed and dispensed to the patient and all dose changes during the study must be recorded. | Drug | all patients on study |
Primary outcome
Efficacy of Treatment
Time frame: 12 months after beginning treatment
Determine the efficacy of everolimus in the treatment of CTCL as overall response rate (ORR)
4 secondary outcomes
Time to Response
Time frame: three months
Progression-free Survival
Time frame: two years after discontinuing study treatment
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: Up to one year
Effect of mTOR on Tumors
Time frame: one year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39821757Banchi M, Cox MC, Bandini A, Orlandi P, Tacchi C, Stefanelli F, Chericoni S, Bocci G. Linifanib alone and in combination with metronomic chemotherapy is active on cutaneous T-cell lymphoma cells by targeting the AKT/mTOR signaling pathway. Invest New Drugs. 2025 Feb;43(1):135-146. doi: 10.1007/s10637-024-01501-8. Epub 2025 Jan 17.
- Individual participant dataSharing planned: No
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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.
Adam Lerner, MD principal investigator
Boston University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Everolimus in people with Cutaneous T-Cell Lymphoma, run by Adam Lerner at 1 site 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 12 inclusion and 23 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 12 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 31 Oct 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?
Efficacy of Treatment (time frame: 12 months after beginning treatment). 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 NCT01637090, which also lists 1 linked publication.
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 12 Jan 2017.