Trial Outcomes & Findings for RAD001(Everolimus) in Treating Patients With Myelodysplastic Syndromes (NCT NCT00809185)

Last Updated: 2019-03-07

Results Overview

Major erythroid response: (1) For patients with a baseline hemoglobin less than 11 g/dL, a major erythroid response is defined as a \> 2 g/dL increase in hemoglobin from baseline; or (2) 100% decrease in red blood cell transfusion requirements. Minor erythroid response: (1) For patients with baseline hemoglobin less than 11 g/dL, a minor erythroid response is defined as an increase in hemoglobin greater than 1 g/dL but less than 2 g/dL from baseline; or (2) \> 50% decrease in red blood cell transfusion requirements.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

7 participants

Primary outcome timeframe

2 years of treatment

Results posted on

2019-03-07

Participant Flow

Patients were recruited from Cleveland Clinic medical hospital from April 2006-March 2009

Participant milestones

Participant milestones
Measure	RAD001(Everolimus) drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

RAD001(Everolimus) in Treating Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RAD001(Everolimus) n=7 Participants drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Age, Continuous	67 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years of treatment

Population: All patients enrolled and given treatment.

Outcome measures

Outcome measures
Measure	RAD001(Everolimus) n=7 Participants drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Number of Patients With Either a Major or Minor Erythroid Response (Hemoglobin Change From Baseline Measure) Number of Patients with a Major Response	0 participants
Number of Patients With Either a Major or Minor Erythroid Response (Hemoglobin Change From Baseline Measure) Number of Patients with a Minor Response	0 participants

SECONDARY outcome

Timeframe: at end of one cycle (28 days)

Population: All patients enrolled and given treatment.

Number of Dose- and Non-dose-limiting Toxicities at the end of cycle 1 associated with RAD001 (see AE/SAE section for details).

Outcome measures

Outcome measures
Measure	RAD001(Everolimus) n=7 Participants drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Number of Dose- and Non-dose-limiting Toxicities	26 events

SECONDARY outcome

Timeframe: at 2 years of treatment

Population: All patients enrolled and given treatment.

Number of Participants with change in bone marrow morphology and cytogenetics

Outcome measures

Outcome measures
Measure	RAD001(Everolimus) n=7 Participants drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Number of Participants With Bone Marrow Morphology and Cytogenetics Pre- and Post-therapy	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: at 2 years of treatment

T-cell populations in patients pre- and post-treatment

Outcome measures

Outcome data not reported

Adverse Events

RAD001(Everolimus)

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	RAD001(Everolimus) n=7 participants at risk drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
Infections and infestations infection	14.3% 1/7 • Adverse events were measure during treatment which ranges from 5-196 days.

Other adverse events

Other adverse events
Measure	RAD001(Everolimus) n=7 participants at risk drug will be administered at 10mg by mouth for 21 days followed by a 7 day rest period
General disorders bleeding	28.6% 2/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Respiratory, thoracic and mediastinal disorders dyspnea	42.9% 3/7 • Adverse events were measure during treatment which ranges from 5-196 days.
General disorders fatigue	42.9% 3/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Infections and infestations fever/infection	57.1% 4/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Blood and lymphatic system disorders neutropenia	42.9% 3/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Respiratory, thoracic and mediastinal disorders pneumonitis	14.3% 1/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Skin and subcutaneous tissue disorders rash	14.3% 1/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Renal and urinary disorders renal insufficiency	14.3% 1/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Infections and infestations stomatitis	28.6% 2/7 • Adverse events were measure during treatment which ranges from 5-196 days.
Blood and lymphatic system disorders thrombocytopenia	42.9% 3/7 • Adverse events were measure during treatment which ranges from 5-196 days.
General disorders weight loss	28.6% 2/7 • Adverse events were measure during treatment which ranges from 5-196 days.

Additional Information

Anjali Advani

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Phone: 216-445-9354

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place