Trial Outcomes & Findings for Methylphenidate for Cancer-Related Fatigue (NCT NCT01164956)

NCT ID: NCT01164956

Last Updated: 2019-04-26

Results Overview

The feasibility of conducting an N-1-T to evaluate MPH for cancer-related fatigue will be determined by the completion rate of two MPH-placebo pairs.

• Recruitment status: TERMINATED
• Study phase: PHASE1
• Target enrollment: 3 participants
• Primary outcome timeframe: 18 days
• Results posted on: 2019-04-26

Participant Flow

3 participants were enrolled between July 2011 and January 2012. Only the arms of these participants are presented.

Participant milestones

Measure	M-P, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, M-P, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Overall Study STARTED	1	1	1
Overall Study Evaluable for pedsFACIT-F Score Change	0	1	0
Overall Study COMPLETED	0	1	0
Overall Study NOT COMPLETED	1	0	1

Reasons for withdrawal

Measure	M-P, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, M-P, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Overall Study Side Effects	1	0	1

Baseline Characteristics

Methylphenidate for Cancer-Related Fatigue

Baseline characteristics by cohort

Measure	All Participants n=3 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	16 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 18 days

Population: The analysis dataset is comprised of all enrolled patients. Based on the definition of the outcome being evaluated, aggregation of data for all participants is appropriate.

The feasibility of conducting an N-1-T to evaluate MPH for cancer-related fatigue will be determined by the completion rate of two MPH-placebo pairs.

Outcome measures

Measure	All Participants n=3 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, M-P, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Completion Rate of Two Treatment Pairs Using the N-1-T Design	1 participants	—	—

SECONDARY outcome

Timeframe: 18 days

Population: The analysis dataset is comprised of all enrolled patients.

Efficacy of the N-1-T design is defined as patient providing a clinically definite answer regarding the ability of MPH to reduce fatigue.Based on the definition of the outcome being evaluated, aggregation of data for all participants is appropriate.

Outcome measures

Measure	All Participants n=1 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, P-M, M-P n=1 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, M-P, M-P n=1 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Rate of Receiving Clinically Definite Answer Regarding the Ability of Experimental Treatment to Reduce Fatigue Using the N-1-T Design	0 participants	1 participants	0 participants

SECONDARY outcome

Timeframe: The pedsFACIT-Fwas administered at baseline and at the end of each treatment pair (TP) and related change in score was calculated for each period: baseline to end of TP 1 (day 6); end of TP 1 to end of TP 2 (day 12); end of TP 2 to end of TP 3 (day 18).

Population: The analysis dataset is comprised of all enrolled patients with data at baseline and end of all treatment pairs.

The pediatric Functional Assessment of Chronic Illness Therapy-Fatigue (pedsFACIT-F) is an 11-item instrument derived from a comprehensive pediatric item bank that assesses fatigue. (Lai et al. Pediatr Hematol Oncol 2007) Measuring fatigue over the past 7 days in a population of pediatric cancer patients, the pedsFACIT-F instrument has a score ranging from 0-44, with a higher score meaning more fatigue. A minimally important difference (MID) was established as 4.7 points.

Outcome measures

Measure	All Participants n=1 Participants Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, P-M, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.	P-M, M-P, M-P Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Change Over Treatment Pairs in pedsFACIT-F Score Change from Baseline to end of Treatment Pair 1	4 units on a scale	—	—
Change Over Treatment Pairs in pedsFACIT-F Score Change from end of Treatment Pair 1 to Pair 2	2 units on a scale	—	—
Change Over Treatment Pairs in pedsFACIT-F Score Change from end of Treatment Pair 2 to Pair 3	0 units on a scale	—	—

Adverse Events

All Participants

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	All Participants n=3 participants at risk Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Blood and lymphatic system disorders hemoglobin	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Psychiatric disorders Mood alteration - anxiety	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations Metabolic/Laboratory-Other	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
General disorders Rigors/chills	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
General disorders Constitutional Symptoms-Other	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Dehydration	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Gastrointestinal disorders diarrhea	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations Creatinine	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations Alkaline phosphatase	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations ALT, SGPT (serum glutamic pyruvic transaminase)	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations AST, SGOT (serum glutamic oxaloacetic transaminase)	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Nervous system disorders Neuropathy: cranial - CN VII Motor-face; Sensory-taste	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
General disorders fatigue	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
General disorders Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Injury, poisoning and procedural complications fracture	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Cardiac disorders Cardiac General-Other	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Albumin, serum-low (hypoalbuminemia)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Potassium, serum-low (hypokalemia)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Magnesium, serum-low (hypomagnesemia)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Metabolism and nutrition disorders Phosphate, serum-low (hypophosphatemia)	66.7% 2/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Vascular disorders hypotension	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Psychiatric disorders Insomnia	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Musculoskeletal and connective tissue disorders Pain - Muscle	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Investigations PTT (Partial thromboplastin time)	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Renal and urinary disorders Proteinuria	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Ear and labyrinth disorders Tinnitus	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Infections and infestations Infection-Other	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Nervous system disorders Tremor	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.
Renal and urinary disorders Urine color change	33.3% 1/3 • Adverse event data was collected continuously during treatment. As noted in limitations, the study did not meet the accrual target due to lack of resources. AE data by individual is not accessible and therefore AE outcomes for the 3 participants are aggregated. The metabolic/laboratory-other cases were elevated lactate dehydrogenase (LDH), ketonuria and bilirubin in urinalysis. The cardiac-other case was hypertensionsinus tachycardia. The constitutional symptoms-other case was decreased appetite.

Additional Information

Christina K. Ullrich, MD, MPH

Dana-Farber Cancer Institute

Phone: 617.632.4997

Email: Christina_Ullrich@dfci.harvard.edu

Results disclosure agreements

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