Trial Outcomes & Findings for A Phase II Trial of Combined Hydroxychloroquine and Sirolimus in Soft Tissue Sarcoma (NCT NCT01842594)

Results Overview

A baseline whole-body \[18F\]-fluorodeoxyglucose(FDG) PET was performed before therapy initiation. Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks. A second \[18F\]-FDG PET was performed after treatment completion. SUVs were calculated for all lesions. Regions of interest (ROI) were contoured to represent tumors (\>2 cm) and organs (lungs, spleen, and liver) on all transaxial and coronal slices. ROIs were normalized for injection dose and body weight, and the maximum voxel value was recorded for each region or organ. The highest SUV measured with increased uptake was considered the SUVmax. Correlative diagnostic CT examinations were used for accurate localization of the lesions. The most intense uptake at baseline was identified as the index lesion and evaluated for treatment response.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

2 Weeks

Results posted on

2015-11-03

Participant Flow

Subjects were enrolled between August 2012 and June 2014 at Department of Radiation Therapy and Oncology, Shin Kong Wu Ho-Su Memorial Hospital.

Participant milestones

Participant milestones
Measure	Sirolimus and Hydroxychloroquine Patients received 1 mg of sirolimus (rapamycin, Rapa) and 200 mg of hydroxychloroquine (HCQ) twice a day before a meal for 2 weeks.
Overall Study STARTED	13
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase II Trial of Combined Hydroxychloroquine and Sirolimus in Soft Tissue Sarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sirolimus and Hydroxychloroquine n=10 Participants Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks.
Age, Continuous	63 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 Weeks

A baseline whole-body \[18F\]-fluorodeoxyglucose(FDG) PET was performed before therapy initiation. Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks. A second \[18F\]-FDG PET was performed after treatment completion. SUVs were calculated for all lesions. Regions of interest (ROI) were contoured to represent tumors (\>2 cm) and organs (lungs, spleen, and liver) on all transaxial and coronal slices. ROIs were normalized for injection dose and body weight, and the maximum voxel value was recorded for each region or organ. The highest SUV measured with increased uptake was considered the SUVmax. Correlative diagnostic CT examinations were used for accurate localization of the lesions. The most intense uptake at baseline was identified as the index lesion and evaluated for treatment response.

Outcome measures

Outcome measures
Measure	Sirolimus and Hydroxychloroquine n=34 lesions Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks.
The Maximum Standardized Uptake Values (SUVmax) Change on PET/CT Scan	-19.6 percentage of the SUVmax Change Interval -30.1 to -9.1

SECONDARY outcome

Timeframe: 2 Weeks

Number of Participants with Adverse Events. Toxicities parameters are according to the Nation Cancer Institute Common Terminology Criteria for Adverse Event, version 3.0.

Outcome measures

Outcome measures
Measure	Sirolimus and Hydroxychloroquine n=10 Participants Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks.
The Toxicity	3 participants

Adverse Events

Sirolimus and Hydroxychloroquine

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Sirolimus and Hydroxychloroquine n=10 participants at risk Patients received 1 mg of Rapa and 200 mg of HCQ twice a day before a meal for 2 weeks.
Skin and subcutaneous tissue disorders Skin rash	20.0% 2/10 • 2 weeks
Gastrointestinal disorders Nausea	30.0% 3/10 • 2 weeks
Gastrointestinal disorders Diarrhea	10.0% 1/10 • 2 weeks
Gastrointestinal disorders Constipation	10.0% 1/10 • 2 weeks

Additional Information

Kwan-Hwa Chi

Shin Kong Wu Ho-Su Memorial Hospital

Phone: +886-28332211

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place