Trial Outcomes & Findings for Baclofen-Amitriptyline Hydrochloride-Ketamine Gel in Treating Peripheral Neuropathy Caused by Chemotherapy in Patients With Cancer (NCT NCT00516503)

NCT ID: NCT00516503

Last Updated: 2017-08-24

Results Overview

The scoring algorithm for the parent instrument, the EORTC QLQ-C30, was applied for linearly converting items and subscales of CIPN-20 to 0-100 scales so that a high score corresponds to better condition or less symptom. The primary analysis was the change in sensory neuropathy subscale of the CIPN-20 from baseline to week 4. The area under the curve (AUC) from baseline to week 4 was calculated for each patient's sensory neuropathy score. The average AUC for the placebo arm was compared to the average AUC for the topical amitriptyline HCl/ baclofen/ ketamine arm using a Wilcoxon rank sum test.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

208 participants

Primary outcome timeframe

From baseline to 4 weeks

Results posted on

2017-08-24

Participant Flow

Two-hundred eight patients were enrolled to this study. Five patients cancelled prior to initiating protocol treatment and were therefore this study was analyzed using 203 remaining patients.

Participant milestones

Participant milestones
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Overall Study
STARTED
101
102
Overall Study
COMPLETED
101
102
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Baclofen-Amitriptyline Hydrochloride-Ketamine Gel in Treating Peripheral Neuropathy Caused by Chemotherapy in Patients With Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Total
n=203 Participants
Total of all reporting groups
Age, Continuous
59.5 years
n=5 Participants
62 years
n=7 Participants
61 years
n=5 Participants
Sex: Female, Male
Female
66 Participants
n=5 Participants
60 Participants
n=7 Participants
126 Participants
n=5 Participants
Sex: Female, Male
Male
35 Participants
n=5 Participants
42 Participants
n=7 Participants
77 Participants
n=5 Participants
Region of Enrollment
United States
101 participants
n=5 Participants
102 participants
n=7 Participants
203 participants
n=5 Participants

PRIMARY outcome

Timeframe: From baseline to 4 weeks

Population: There were 26 participants in the BAK arm and 27 in the placebo arm who did not provide primary endpoint data. In the BAK arm, 11 refused due to experiencing an adverse event and 15 refused for non-specified reasons. In the placebo arm, eight refused due to an adverse event, one patient died, and 18 refused for non-specified reasons.

The scoring algorithm for the parent instrument, the EORTC QLQ-C30, was applied for linearly converting items and subscales of CIPN-20 to 0-100 scales so that a high score corresponds to better condition or less symptom. The primary analysis was the change in sensory neuropathy subscale of the CIPN-20 from baseline to week 4. The area under the curve (AUC) from baseline to week 4 was calculated for each patient's sensory neuropathy score. The average AUC for the placebo arm was compared to the average AUC for the topical amitriptyline HCl/ baclofen/ ketamine arm using a Wilcoxon rank sum test.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=75 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=75 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Total Sensory Neuropathy as Measured by the European Organization for Research and Treatment of Cancer [EORTC] Quality of Life [QLQ] - Chemo-induced Peripheral Neuropathy [CIPN20]
61.0 (units on a scale) * week
Standard Deviation 16.22
60.9 (units on a scale) * week
Standard Deviation 17.9

SECONDARY outcome

Timeframe: From Baseline to week 4

Population: There were 26 participants in the BAK arm and 27 in the placebo arm who did not provide primary endpoint data. In the BAK arm, 11 refused due to experiencing an adverse\> event and 15 refused for non-specified reasons. In the placebo arm, eight refused due to an\> adverse event, one patient died, and 18 refused for non-specified reasons.

The scoring algorithm for the parent instrument, the EORTC QLQ-C30, was applied for linearly converting items and subscales of CIPN-20 to 0-100 scales so that a high score corresponds to better condition or less symptom. The secondary analysis was to compare changes from baseline at 4 weeks for the motor neuropathy subscale of the CIPN-20. To analyze this endpoint, the area under the curve (AUC) from baseline to week 4 was calculated for each patient's motor neuropathy score. The average AUC for the placebo arm was compared to the average AUC for the topical amitriptyline HCl/ baclofen/ ketamine arm using a Wilcoxon rank sum test.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=75 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=75 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Motor Neuropathy as Measured by the EORTC QLQ-CIPN20 at Baseline and Week 4
69.2 (units on a scale)* week
Standard Deviation 17.15
70.1 (units on a scale)* week
Standard Deviation 19.48

SECONDARY outcome

Timeframe: Up to 4 weeks

Population: There were 26 participants in the BAK arm and 27 in the placebo arm who did not provide primary endpoint data. In the BAK arm, 11 refused due to experiencing an adverse event and 15 refused for non-specified reasons. In the placebo arm, eight refused due to an adverse event, one patient died, and 18 refused for non-specified reasons.

The scoring algorithm for the parent instrument, the EORTC QLQ-C30, was applied for linearly converting items and subscales of CIPN-20 to 0-100 scales so that a high score corresponds to better condition or less symptom. The secondary analysis was to compare changes from baseline at 4 weeks for the autonomic neuropathy subscale of the CIPN-20. To analyze this endpoint, the area under the curve (AUC) from baseline to week 4 was calculated for each patient's motor neuropathy score. The average AUC for the placebo arm was compared to the average AUC for the topical amitriptyline HCl/ baclofen/ ketamine arm using a Wilcoxon rank sum test.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=75 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=75 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Autonomic Symptoms and Functioning as Measured by the EORTC QLQ-CIPN20 at Baseline and Week 4
85.0 (units on a scale)*week
Standard Deviation 15.47
86.8 (units on a scale)*week
Standard Deviation 15.03

SECONDARY outcome

Timeframe: At 4 weeks

Each mood scale (0 - 100, higher is better mood) will be analyzed as an endpoint along with the total mood disturbance score.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS Tension-Anxiety Subscale
83.2 units on a scale
Standard Deviation 17.09
83.0 units on a scale
Standard Deviation 15.73
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS VA Subscale
31.1 units on a scale
Standard Deviation 18.42
33.0 units on a scale
Standard Deviation 19.61
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS Anger-Hostility Score
87.8 units on a scale
Standard Deviation 14.48
87.7 units on a scale
Standard Deviation 12.98
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS Confusion-Bewilderment
76.9 units on a scale
Standard Deviation 17.58
77.9 units on a scale
Standard Deviation 11.75
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS Depression-Dejection Score
86.4 units on a scale
Standard Deviation 16.09
85.8 units on a scale
Standard Deviation 15.23
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
POMS Fatigue-Inertia Score
67.2 units on a scale
Standard Deviation 21.69
63.6 units on a scale
Standard Deviation 22.22
Mood States and Total Mood Disturbance as Measured by the Profile of Mood States (POMS)
Mean POMS Score
71.5 units on a scale
Standard Deviation 13.27
71.8 units on a scale
Standard Deviation 13.14

SECONDARY outcome

Timeframe: Up to 4 weeks

Pain severity, defined by the four items addressing worst, least, and average pain and pain right now as measured by the BPI will be analyzed identical to the primary endpoint. Additionally, total pain interference as measured by the BPI will be transformed onto a 0-100 ( higher is less pain) point scale. The area under the curve (AUC) from baseline to week 4 was calculated for each patient's score. The average AUC for the placebo arm and the topical amitriptyline HCl/ baclofen/ ketamine arm are reported.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Pain Severity and Interference as Measured by the Brief Pain Inventory (BPI) at Baseline and Week 4
Least Pain
78.0 units on a scale * week
Standard Deviation 20.28
77.3 units on a scale * week
Standard Deviation 22.01
Pain Severity and Interference as Measured by the Brief Pain Inventory (BPI) at Baseline and Week 4
Average Pain
66.4 units on a scale * week
Standard Deviation 21.99
65.4 units on a scale * week
Standard Deviation 24.55
Pain Severity and Interference as Measured by the Brief Pain Inventory (BPI) at Baseline and Week 4
BPI Total Interference
76.5 units on a scale * week
Standard Deviation 20.16
77.3 units on a scale * week
Standard Deviation 21.16
Pain Severity and Interference as Measured by the Brief Pain Inventory (BPI) at Baseline and Week 4
Worst Pain
58.1 units on a scale * week
Standard Deviation 25.48
58.7 units on a scale * week
Standard Deviation 27.39

SECONDARY outcome

Timeframe: Up to 4 weeks

The Peripheral Neuropathy Questionnaire was used to analyze this endpoint. Patient neuropathy symptoms were scored on a 0 - 100 scale (higher score represents less symptomatic). The area under the curve (AUC) from baseline to week 4 was calculated for each patient's score. The average AUC for the placebo arm and the topical amitriptyline HCl/ baclofen/ ketamine arm are reported.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Numbness, Tingling, and Pain as Measured by the Peripheral Neuropathy Questionnaire at Baseline and Weekly for 4 Weeks
172.6 units on a scale * week
Standard Deviation 82.09
175.7 units on a scale * week
Standard Deviation 85.38

SECONDARY outcome

Timeframe: Up to 4 weeks

Population: All patients that were assessed for adverse events are used in this analysis.

Frequency and severity of adverse events reported by patients in weekly diary and evaluated through clinical assessment by NCI CTCAE v3.0. The number of patients reporting grade 3 or higher events are reported in this outcome measure. For a full list of all events, please refer to the Adverse Events section of this report.

Outcome measures

Outcome measures
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 Participants
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 Participants
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Adverse Event Profile of Topical Amitriptyline HCl/ Baclofen/Ketamine > Frequency and Severity of Adverse Events Reported by the Patient in the > Symptom Experience Diary and Evaluated Through Clinical Assessment by NCI CTCAE v3.0
Grade 3+ Adverse Event
8 Participants
5 Participants
Adverse Event Profile of Topical Amitriptyline HCl/ Baclofen/Ketamine > Frequency and Severity of Adverse Events Reported by the Patient in the > Symptom Experience Diary and Evaluated Through Clinical Assessment by NCI CTCAE v3.0
Grade 4+ Adverse Event
1 Participants
1 Participants

Adverse Events

Baclofen-amitriptyline Hydrochloride-ketamine

Serious events: 1 serious events
Other events: 48 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 45 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 participants at risk
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 participants at risk
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
General disorders
Disease progression
0.00%
0/101
0.98%
1/102 • Number of events 1
Vascular disorders
Thrombosis
0.99%
1/101 • Number of events 1
0.00%
0/102

Other adverse events

Other adverse events
Measure
Baclofen-amitriptyline Hydrochloride-ketamine
n=101 participants at risk
Patients apply 1 spoonful of baclofen-amitriptyline hydrochloride-ketamine gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Baclofen/amitriptyline/ketamine gel: Applied topically.
Placebo
n=102 participants at risk
Patients apply 1 spoonful of placebo gel topically to each area of pain, numbness, and/or tingling on the feet and/or hands twice daily for 4 weeks. Placebo: Applied topically
Ear and labyrinth disorders
Tinnitus
0.00%
0/101
0.98%
1/102 • Number of events 1
Gastrointestinal disorders
Abdominal pain
0.00%
0/101
2.0%
2/102 • Number of events 5
Gastrointestinal disorders
Constipation
23.8%
24/101 • Number of events 62
27.5%
28/102 • Number of events 71
Gastrointestinal disorders
Diarrhea
0.99%
1/101 • Number of events 1
0.98%
1/102 • Number of events 1
Gastrointestinal disorders
Dry mouth
17.8%
18/101 • Number of events 55
22.5%
23/102 • Number of events 55
Gastrointestinal disorders
Ileal obstruction
0.00%
0/101
0.98%
1/102 • Number of events 1
Gastrointestinal disorders
Nausea
0.99%
1/101 • Number of events 1
0.00%
0/102
Gastrointestinal disorders
Small intestinal obstruction
0.00%
0/101
2.0%
2/102 • Number of events 4
Gastrointestinal disorders
Stomach pain
0.00%
0/101
0.00%
0/102
Gastrointestinal disorders
Vomiting
0.99%
1/101 • Number of events 1
0.00%
0/102
General disorders
Edema limbs
0.99%
1/101 • Number of events 1
0.98%
1/102 • Number of events 1
General disorders
Fatigue
0.99%
1/101 • Number of events 1
0.98%
1/102 • Number of events 1
General disorders
Pain
0.00%
0/101
0.00%
0/102
Immune system disorders
Hypersensitivity
0.00%
0/101
0.98%
1/102 • Number of events 1
Infections and infestations
Bladder infection
0.00%
0/101
0.00%
0/102
Infections and infestations
Skin infection
0.99%
1/101 • Number of events 2
0.00%
0/102
Injury, poisoning and procedural complications
Fracture
0.00%
0/101
0.00%
0/102
Investigations
Alkaline phosphatase increased
0.00%
0/101
0.98%
1/102 • Number of events 1
Investigations
Aspartate aminotransferase increased
0.00%
0/101
0.98%
1/102 • Number of events 1
Investigations
Platelet count decreased
0.00%
0/101
0.98%
1/102 • Number of events 1
Musculoskeletal and connective tissue disorders
Back pain
0.00%
0/101
0.00%
0/102
Musculoskeletal and connective tissue disorders
Chest wall pain
0.00%
0/101
0.00%
0/102
Musculoskeletal and connective tissue disorders
Joint pain
0.99%
1/101 • Number of events 1
0.00%
0/102
Musculoskeletal and connective tissue disorders
Pain in extremity
0.99%
1/101 • Number of events 1
0.00%
0/102
Nervous system disorders
Ataxia
0.00%
0/101
0.98%
1/102 • Number of events 1
Nervous system disorders
Depressed level of consciousness
2.0%
2/101 • Number of events 2
2.0%
2/102 • Number of events 2
Nervous system disorders
Dizziness
0.00%
0/101
0.00%
0/102
Nervous system disorders
Neuralgia
0.99%
1/101 • Number of events 1
0.00%
0/102
Nervous system disorders
Peripheral sensory neuropathy
0.00%
0/101
2.0%
2/102 • Number of events 2
Nervous system disorders
Syncope vasovagal
0.00%
0/101
0.00%
0/102
Psychiatric disorders
Anxiety
0.00%
0/101
0.00%
0/102
Psychiatric disorders
Confusion
5.0%
5/101 • Number of events 5
3.9%
4/102 • Number of events 4
Skin and subcutaneous tissue disorders
Dry skin
0.99%
1/101 • Number of events 1
0.00%
0/102
Skin and subcutaneous tissue disorders
Hand-and-foot syndrome
15.8%
16/101 • Number of events 26
11.8%
12/102 • Number of events 25
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/101
0.00%
0/102
Vascular disorders
Thrombosis
0.00%
0/101
0.98%
1/102 • Number of events 2

Additional Information

Debra Barton, R.N., Ph.D.

Mayo Clinic

Results disclosure agreements

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