Trial Outcomes & Findings for Study of DCA (Dichloroacetate) in Combination With Cisplatin and Definitive Radiation in Head and Neck Carcinoma (NCT NCT01386632)

Last Updated: 2020-10-27

Results Overview

Percentage of Participants Who Experienced Adverse Events During Treatment including but are not limited to mucositis, leucopenia, neuropathy, and treatment breaks. This will be done according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.0

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

Adverse events (AE) will be assessed from the time the subject begins the study until the 30-days after receiving the last dose of the study medication.

Results posted on

2020-10-27

Participant Flow

Participant milestones

Participant milestones
Measure	DCA (Dichloroacetate) Treatment DCA orally 12.5mg/kg or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60 minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions) DCA (dichloroacetate): DCA orally 12.5mg/kg PO or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions)	Placebo Placebo: Placebo PO or per G-tube twice a day for 8 weeks given in combination with Cisplatin.
Overall Study STARTED	25	25
Overall Study COMPLETED	20	21
Overall Study NOT COMPLETED	5	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of DCA (Dichloroacetate) in Combination With Cisplatin and Definitive Radiation in Head and Neck Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	DCA (Dichloroacetate) Treatment n=25 Participants DCA orally 12.5mg/kg or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60 minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions) DCA (dichloroacetate): DCA orally 12.5mg/kg PO or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions)	Placebo n=25 Participants Placebo: Placebo PO or per G-tube twice a day for 8 weeks given in combination with Cisplatin.	Total n=50 Participants Total of all reporting groups
Age, Continuous	60.89 years STANDARD_DEVIATION 9.06 • n=5 Participants	58.35 years STANDARD_DEVIATION 8.33 • n=7 Participants	59.62 years STANDARD_DEVIATION 8.71 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	6 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants	19 Participants n=7 Participants	43 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	24 Participants n=5 Participants	24 Participants n=7 Participants	48 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	25 Participants n=5 Participants	24 Participants n=7 Participants	49 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	25 participants n=5 Participants	25 participants n=7 Participants	50 participants n=5 Participants

PRIMARY outcome

Timeframe: Adverse events (AE) will be assessed from the time the subject begins the study until the 30-days after receiving the last dose of the study medication.

Outcome measures

Outcome measures
Measure	DCA (Dichloroacetate) Treatment n=25 Participants DCA orally 12.5mg/kg or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60 minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions) DCA (dichloroacetate): DCA orally 12.5mg/kg PO or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions)	Placebo n=25 Participants Placebo: Placebo PO or per G-tube twice a day for 8 weeks given in combination with Cisplatin.
Percentage of Participants Who Experienced Adverse Events During Treatment.	96 percentage of patients Interval 79.7 to 99.9	96 percentage of patients Interval 79.7 to 99.9

SECONDARY outcome

Timeframe: Year 2

Outcome of tumor change will be compared in two separate ways. First, change in measured tumor size at 8 weeks and 3 months will be compared using standard linear models methods (using appropriate transformation to reduce statistical skew). Progression was determined using RECIST 1.1 definition of 20% increase in the sum of the diameters of target lesions, in either primary or nodal lesions or the appearance of one or more new lesion(s) and/or unequivocal progression of existing non-target lesions.

Outcome measures

Outcome measures
Measure	DCA (Dichloroacetate) Treatment n=25 Participants DCA orally 12.5mg/kg or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60 minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions) DCA (dichloroacetate): DCA orally 12.5mg/kg PO or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions)	Placebo n=25 Participants Placebo: Placebo PO or per G-tube twice a day for 8 weeks given in combination with Cisplatin.
Two-year Progression-free Survival Rate in Locally Advanced Head and Neck Squamous Cell Carcinoma in Patients Receiving Concurrent Cisplatin, Radiation Therapy, and DCA.	86.5 percentage of patients Interval 72.3 to 100.0	87.5 percentage of patients Interval 74.3 to 100.0

SECONDARY outcome

Timeframe: Year 5

Progression will be determined using RECIST 1.1 definition of 20% increase in the sum of the diameters of target lesions, in either primary or nodal lesions or the appearance of one or more new lesion(s) and/or unequivocal progression of existing non-target lesions.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 5 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Completion of Treatment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 5 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: End of treatment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 5 years

Outcome measures

Outcome data not reported

Adverse Events

DCA (Dichloroacetate) Treatment

Serious events: 8 serious events

Other events: 25 other events

Deaths: 0 deaths

Placebo

Serious events: 6 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Steven Powell

Sanford Clinical Research

Phone: 605-328-8000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	DCA (Dichloroacetate) Treatment n=25 participants at risk DCA orally 12.5mg/kg or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60 minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions) DCA (dichloroacetate): DCA orally 12.5mg/kg PO or per G-tube BID daily for 8 weeks in conjunction with Cisplatin 100 mg/m\^2 IV over 30-60minutes every 3wks X 3(Days 1, 22, and 43 of RT)and RT 70 Gy/35 -200 cGy/d x 7 weeks (35 Fractions)	Placebo n=25 participants at risk Placebo: Placebo PO or per G-tube twice a day for 8 weeks given in combination with Cisplatin.
Gastrointestinal disorders Vomiting	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	12.0% 3/25 • Number of events 3 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Gastrointestinal disorders Nausea	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	8.0% 2/25 • Number of events 2 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Metabolism and nutrition disorders dehydration	8.0% 2/25 • Number of events 2 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Cardiac disorders Atrial fibrillation	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Nervous system disorders Dizziness	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Vascular disorders Hypotension	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
General disorders Fatigue	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Investigations creatinine increased	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Infections and infestations Infection and infestation-Other	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Psychiatric disorders Delirium	4.0% 1/25 • Number of events 2 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
General disorders Fever	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Gastrointestinal disorders Mucositis Oral	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Blood and lymphatic system disorders Febrile neutropenia	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Blood and lymphatic system disorders Anemia	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.
Investigations Neutrophil count decreased	4.0% 1/25 • Number of events 1 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.	0.00% 0/25 • Adverse event data collected from first day of treatment to 30 days post last day of treatment, up to 2 years post treatment.