Trial Outcomes & Findings for Everolimus in Treating Cutaneous T-cell Lymphoma (NCT NCT01637090)

NCT ID: NCT01637090

Last Updated: 2017-01-12

Results Overview

Determine the efficacy of everolimus in the treatment of CTCL as overall response rate (ORR)

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: 12 months after beginning treatment
• Results posted on: 2017-01-12

Participant Flow

This study was actively recruiting from within the investigator's own praceice from June 2012 through May 2015 in the hematology/oncology multidisciplinary clinic.

Participant milestones

Measure	All Patients on Study 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Everolimus in Treating Cutaneous T-cell Lymphoma

Baseline characteristics by cohort

Measure	All Patients on Study n=3 Participants 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Gender Female	0 Participants n=5 Participants
Gender Male	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months after beginning treatment

Population: Study was pre-maturely terminated. No data were collected for the Outcome Measure

Determine the efficacy of everolimus in the treatment of CTCL as overall response rate (ORR)

Outcome measures

Measure

All Patients on Study 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.

Efficacy of Treatment

0

SECONDARY outcome

Timeframe: three months

Population: Study was pre-maturely terminated. No data were collected for the Outcome Measure

Determine time to response (TTR)/duration of objective response (DOR)

Outcome measures

Measure

All Patients on Study 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.

Time to Response

0

SECONDARY outcome

Timeframe: two years after discontinuing study treatment

Population: Study was pre-maturely terminated. No data were collected for the Outcome Measure

Determine progression-free survival of CTCL patients treated with everolimus

Outcome measures

Measure

All Patients on Study 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.

Progression-free Survival

0

SECONDARY outcome

Timeframe: Up to one year

Determine the adverse event profile and tolerability of everolimus in patients with CTCL

Outcome measures

Measure

All Patients on Study n=3 Participants 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

3 participants

SECONDARY outcome

Timeframe: one year

Population: Study was pre-maturely terminated. No data were collected for the Outcome Measure

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

Outcome measures

Measure

All Patients on Study 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.

Effect of mTOR on Tumors

0

Adverse Events

All Patients on Study

Serious events: 3 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	All Patients on Study n=3 participants at risk 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. Everolimus will be administered orally as once daily dose of 10 mg continuously from study day 1 until progression of disease or unacceptable toxicity.
General disorders fatigue	33.3% 1/3 • Number of events 1 • Adverse event reporting was to be collected for up to one year.
Skin and subcutaneous tissue disorders pruritis	33.3% 1/3 • Number of events 1 • Adverse event reporting was to be collected for up to one year.
Skin and subcutaneous tissue disorders Rash	33.3% 1/3 • Adverse event reporting was to be collected for up to one year.
Infections and infestations skin infection	33.3% 1/3 • Number of events 1 • Adverse event reporting was to be collected for up to one year.

Other adverse events

Adverse event data not reported

Additional Information

Adam Lerner, MD

Boston Medical Center

Phone: 617-638-8265

Email: adam.lerner@bmc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place