Trial Outcomes & Findings for Phase II Study to Evaluate Fenofibrate Therapy in Patients With Smoldering or Symptomatic Multiple Myeloma (NCT NCT01965834)

NCT ID: NCT01965834

Last Updated: 2017-06-14

Results Overview

To determine response rate (Strict Complete Response (sCR), Complete response (CR), Very Good Partial Response (VgPR), and Partial Response (PR)) in multiple myeloma patients receiving oral fenofibrate therapy. Response will be measured by serum and urine protein electrophoresis and immunofixation, as well as by percentage of plasma cells present on bone marrow biopsy.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

After two cycles, about 2 months

Results posted on

2017-06-14

Participant Flow

Participant milestones

Participant milestones
Measure
Fenofibrate Therapy
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Overall Study
STARTED
6
Overall Study
COMPLETED
2
Overall Study
NOT COMPLETED
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Fenofibrate Therapy
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Overall Study
Lack of Efficacy
4

Baseline Characteristics

Phase II Study to Evaluate Fenofibrate Therapy in Patients With Smoldering or Symptomatic Multiple Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Fenofibrate Therapy
n=6 Participants
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
4 Participants
n=99 Participants
Age, Categorical
>=65 years
2 Participants
n=99 Participants
Sex: Female, Male
Female
3 Participants
n=99 Participants
Sex: Female, Male
Male
3 Participants
n=99 Participants
Region of Enrollment
United States
6 Participants
n=99 Participants

PRIMARY outcome

Timeframe: After two cycles, about 2 months

Population: No participants achieved response to protocol therapy (Strict Complete Response (sCR), Complete response (CR), Very Good Partial Response (VgPR), or Partial Response (PR)). Two (2) patients had stable disease (SD); one (1) patient achieved Stable Disease/clinical Progressive Disease, and three (3) patients had progressive disease (PD).

To determine response rate (Strict Complete Response (sCR), Complete response (CR), Very Good Partial Response (VgPR), and Partial Response (PR)) in multiple myeloma patients receiving oral fenofibrate therapy. Response will be measured by serum and urine protein electrophoresis and immunofixation, as well as by percentage of plasma cells present on bone marrow biopsy.

Outcome measures

Outcome measures
Measure
Fenofibrate Therapy
n=6 Participants
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Rate of Response in Participants Receiving Fenofibrate Therapy
Strict Complete Response (sCR)
0 participants
Rate of Response in Participants Receiving Fenofibrate Therapy
Complete response (CR)
0 participants
Rate of Response in Participants Receiving Fenofibrate Therapy
Very Good Partial Response (VgPR)
0 participants
Rate of Response in Participants Receiving Fenofibrate Therapy
Partial Response (PR)
0 participants

SECONDARY outcome

Timeframe: Up to 8 months

To evaluate safety and tolerability of fenofibrate therapy in patients with multiple myeloma.

Outcome measures

Outcome measures
Measure
Fenofibrate Therapy
n=6 Participants
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Number of Subjects Experiencing Adverse Events
5 participants

SECONDARY outcome

Timeframe: 6 months, 12 months

Proportion of participants achieving progression-free survival. Measured from date of initiation of treatment (Day 1) to the earliest occurrence of any of the following events: documented disease progression, or death from any cause. Patients who are alive and progression-free will be censored at the date of last documented progression-free status.

Outcome measures

Outcome measures
Measure
Fenofibrate Therapy
n=6 Participants
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Proportion of Participants Achieving Progression-Free Survival
6 months
0 participants
Proportion of Participants Achieving Progression-Free Survival
12 months
0 participants

Adverse Events

Fenofibrate Therapy

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Fenofibrate Therapy
n=6 participants at risk
Fenofibrate orally daily for each 28 day cycle, per study protocol. Fenofibrate: Upon screening, registration and enrollment, all subjects will receive Fenofibrate 160 mg orally daily for at least 2 months and may continue receiving study medication for as long as in the opinion of the investigator there is clinical benefit in doing so. Patients with calculated creatinine clearance \< 50 mL/min will receive a reduced dose of 54 mg orally daily.
Gastrointestinal disorders
Abdominal Pain
16.7%
1/6 • Number of events 1
Blood and lymphatic system disorders
Anemia
50.0%
3/6 • Number of events 3
Investigations
Aspartate aminotransferase increased
16.7%
1/6 • Number of events 1
Gastrointestinal disorders
Constipation
16.7%
1/6 • Number of events 1
Metabolism and nutrition disorders
Creatinine Increased
33.3%
2/6 • Number of events 2
Respiratory, thoracic and mediastinal disorders
Dyspnea
16.7%
1/6 • Number of events 2
General disorders
Fatigue
33.3%
2/6 • Number of events 2
General disorders
Fever
16.7%
1/6 • Number of events 1
General disorders
Flu-like Symptoms
16.7%
1/6 • Number of events 1
Metabolism and nutrition disorders
Hypokalemia
16.7%
1/6 • Number of events 1
Infections and infestations
Upper respiratory infection
33.3%
2/6 • Number of events 2
General disorders
Localized edema
16.7%
1/6 • Number of events 1
Nervous system disorders
Peripheral sensory neuropathy
16.7%
1/6 • Number of events 1
General disorders
Non-cardiac chest pain
16.7%
1/6 • Number of events 1
General disorders
Back pain
16.7%
1/6 • Number of events 1
General disorders
Bone pain
16.7%
1/6 • Number of events 1
General disorders
Pain
33.3%
2/6 • Number of events 2
Cardiac disorders
Palpitations
33.3%
2/6 • Number of events 2
Skin and subcutaneous tissue disorders
Rash maculo-papular
16.7%
1/6 • Number of events 1

Additional Information

Denise Pereira MD

University of Miami

Phone: 305-243-4909

Results disclosure agreements

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