Clinical Trials List
Protocol NumberKOM-PAX1-001
Completed
2020-11-18 - 2024-12-31
Phase II
Recruiting2
Terminated5
An Exploratory, Randomised, Double-blind, Parallel-Group, Placebo-Controlled Phase IIa Study to Assess the Analgesic Activity of PAX-1 in Patients with Persistent Cancer Pain.
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Trial Applicant
Efficient Pharma Management Corp.
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Sponsor
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Trial scale
Taiwan Multiple Center
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Update
2026/04/01
Investigators and Locations
Principal Investigator
Co-Principal Investigator
- HUI-WEN LIU Division of Hematology & Oncology
- Yao-Yu Hsieh Division of Hematology & Oncology
- Tsu-Yi Chao Division of Hematology & Oncology
- YI-CHIEH TSAI Division of Hematology & Oncology
- Wei-Hong Cheng Division of Hematology & Oncology
- Jo-Ting Tsa Division of Hematology & Oncology
- TSU-YI CHAO Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Kuan-Der Lee Division of Hematology & Oncology
- YAORU HUANG Division of Hematology & Oncology
- Cheng-I Hsieh Division of Hematology & Oncology
- Jia-Ruey Tsai Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Terminated
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- 吳儁峰 Division of Hematology & Oncology
- 黃彥閔 Division of Hematology & Oncology
- 吳宗翰 Division of Hematology & Oncology
- 張悅詩 Division of Hematology & Oncology
- HANG HUONG LING Division of Hematology & Oncology
- 葉光揚 Division of Hematology & Oncology
- Pei-Hung Chang Division of Hematology & Oncology
- 賴建宏 Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Peng-Chan Lin Division of Hematology & Oncology
- Shang-Yin Wu Division of Hematology & Oncology
- 劉奕廷 Division of Hematology & Oncology
- 黃盈慈 Division of Hematology & Oncology
- Wei-Pang Chung Division of Hematology & Oncology
- 楊舜如 Division of Hematology & Oncology
- 顏志傑 Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- 吳志成 Division of Others
- 洪至仁 Division of Others
- 蔡昇亨 Division of Others
- YU-HSUAN SHIH Division of Hematology & Oncology
- CHENG-HSIEN LIN Division of Hematology & Oncology
- 滕傑林 Division of Hematology & Oncology
- Tsung -Chih Chen Division of Hematology & Oncology
- 謝昀叡 Division of Others
- HSIN-CHEN LIN Division of Hematology & Oncology
- 楊士杰 Division of Others
The Actual Total Number of Participants Enrolled
0 Terminated
Condition/Disease
Persistent Cancer Pain
Objectives
Primary Objective:
The primary objective of this study is to assess the efficacy of daily administration of 7.5 mg PAX-1 as an adjunctive analgesic treatment for persistent cancer pain, as measured by the number of patients experiencing ≥ 30% reduction in the mean daily pain score recorded on an 11 (0-10) point Numerical Rating Scale (NRS)
Secondary Objectives:
The secondary objectives of this study are to evaluate the clinical benefits of the daily administration of 7.5 mg PAX-1 as an opioid-sparing, adjunctive treatment for persistent cancer pain as measured by:
1. Reduction in the use of opioids measured as reduced total number of morphine equivalents (background therapy and breakthrough (rescue) medication) taken by the patient.
2. The change in average NRS pain score
3. Breakthrough pain frequency.
4. Sleep Quality: Pittsburgh Sleep Quality Index (PSQI).
5. Functional Status: Eastern Cooperative Oncology Group (ECOG) Scale of Performance Status.
6. Patient reported outcome measures: Short Form Brief Pain Inventory (BPI-SF) and the Opioid Related Symptom Distress Scale (OR-SDS).
7. Patient Global Impression of Change (PGIC).
8. Evaluation of the pharmacodynamic activity of PAX-1 when administered to patients with persistent cancer pain.
9. Evaluating the safety and tolerability of PAX-1 when administered to patients with persistent cancer pain.
Test Drug
PAX-1
Active Ingredient
Dosage Form
tablet
Dosage
2.5 mg/tablet
Endpoints
Primary Endpoints:
The number of patients with a ≥ 30% reduction in the Mean Daily Pain Score when recorded on an 11 (0 – 10) point Numerical Rating Scale (NRS) at the end of the 4-week double-blind treatment period (Treatment Period 1) when compared to the baseline value recorded over 7 consecutive days during the Screening Period. The Mean Daily Pain Score will be the mean of the seven days preceding the clinic visit day as recorded in the Patient Daily Pain Diary.
Secondary Endpoints:
1. The number of patients on daily 7.5 mg PAX-1 with a ≥ 25% reduction in opioid use at the end of the 4-week double-blind
treatment period (Treatment Period 1) when compared to the baseline value recorded over 7 consecutive days during the
Screening Period. The amount of opioid, when used, is the total amount of prescribed morphine equivalents (background and
breakthrough (rescue) medication) actually taken by the patient.
2. The change in the mean NRS pain score from baseline (recorded during the Screening Period) during Treatment Period 1.
3. Breakthrough pain frequency: total number of days with breakthrough pain during the entire Treatment Period 1 and
change in total number of days with breakthrough pain from the baseline during week 4 of treatment period 1.
4. Sleep quality (PSQI).
5. Functional status (ECOG Scale of Performance Status).
6. Patient reported outcome (BPI-SF and OR-SDS).
7. Patients Global Impression of Change (PGIC).
8. The pharmacodynamic activity of PAX-1 will be evaluated by the quantitative assessment of IL-1, IL-6, IL-8 and TNF-alpha in the blood.
The number of patients with a ≥ 30% reduction in the Mean Daily Pain Score when recorded on an 11 (0 – 10) point Numerical Rating Scale (NRS) at the end of the 4-week double-blind treatment period (Treatment Period 1) when compared to the baseline value recorded over 7 consecutive days during the Screening Period. The Mean Daily Pain Score will be the mean of the seven days preceding the clinic visit day as recorded in the Patient Daily Pain Diary.
Secondary Endpoints:
1. The number of patients on daily 7.5 mg PAX-1 with a ≥ 25% reduction in opioid use at the end of the 4-week double-blind
treatment period (Treatment Period 1) when compared to the baseline value recorded over 7 consecutive days during the
Screening Period. The amount of opioid, when used, is the total amount of prescribed morphine equivalents (background and
breakthrough (rescue) medication) actually taken by the patient.
2. The change in the mean NRS pain score from baseline (recorded during the Screening Period) during Treatment Period 1.
3. Breakthrough pain frequency: total number of days with breakthrough pain during the entire Treatment Period 1 and
change in total number of days with breakthrough pain from the baseline during week 4 of treatment period 1.
4. Sleep quality (PSQI).
5. Functional status (ECOG Scale of Performance Status).
6. Patient reported outcome (BPI-SF and OR-SDS).
7. Patients Global Impression of Change (PGIC).
8. The pharmacodynamic activity of PAX-1 will be evaluated by the quantitative assessment of IL-1, IL-6, IL-8 and TNF-alpha in the blood.
Inclution Criteria
1. Male or female patients 20 years of age and over.
2. Patients with advanced solid tumour with life expectancy >= 3 months.
3. Patients with persistent cancer-related pain who are receiving stable opioid treatment in the 7 days prior to the screening.
4. Level of pain measured on NRS is >=4 on at least one occasion per day for at least 2 out of 7 consecutive days during the screening period.
5. If currently on adjuvant pain treatment, having a stable regimen (i.e. dose unchanged) of existing adjuvant pain treatment for a minimum of 7 days prior to screening.
6. If currently on anti-cancer therapy (hormonal therapy, biological, chemotherapy, targeted therapy or radiotherapy), having a stable regimen of existing anti-cancer therapy for a minimum of 2 weeks prior to screening.
7. Patients with a functional status of 0 - 3 as measured using the ECOG Scale of Performance Status.
8. Patients must be competent to understand the nature of the study & capable of giving written informed consent. Be willing to report for the scheduled study visits, complete the study questionnaires, daily Diary Card and communicate to study personnel about adverse events and concomitant medication use.
9. Patients must have adequate haematological, hepatic and renal functions:
a. Absolute neutrophil count (ANC) > 1.5 x10^9/L
b. Platelet count > 100 x10^9/L
c. Haemoglobin > 90 g/L (> 9.0 g/dL)
d. Total bilirubin < 1.5 ULN (< 2.5 ULN if patient has Gilbert’s syndrome)
e. AST/ALT ≤ 2.5 x ULN; ≤ 5 x ULN for patients with liver metastases or liver cancer
f. Serum creatinine within normal range or calculated creatinine clearance > 50 ml/min
g. Serum potassium and magnesium levels within normal range (sodium and magnesium replacement strategies may be used in case of deficiency)
h. Serum electrolyte levels including sodium, calcium, phosphorus, and chloride within normal range or deemed not clinically significant by the Investigator
Patients whose serum electrolyte levels (including those listed in (g) and (h) above) are not within normal range can be re-tested once during the screening period.
10. The patient is able to take oral medication.
11. Female patients must be of either:
a. Non-childbearing potential, post-menopausal, provided there is a laboratory confirmed serum follicle stimulating hormone (FSH) level >= 40mIU/mL at screening (the test is waived for females >= 70 years old), or there is a confirmed clinical history of sterility (e.g. the patient is without a uterus)
b. Childbearing potential, provided there are pregnancy test results prior to study treatment, to rule out pregnancy.
12. Male and female patients of childbearing potential must agree not to become pregnant and be willing to consent to using effective contraception, starting at screening and continuing throughout the clinical study period and for 90 days after the last study visit. Refer to Section 8.1.4 for effective contraceptive methods.
13. Patients agrees not to participate in another interventional study while participating in the present clinical study.
2. Patients with advanced solid tumour with life expectancy >= 3 months.
3. Patients with persistent cancer-related pain who are receiving stable opioid treatment in the 7 days prior to the screening.
4. Level of pain measured on NRS is >=4 on at least one occasion per day for at least 2 out of 7 consecutive days during the screening period.
5. If currently on adjuvant pain treatment, having a stable regimen (i.e. dose unchanged) of existing adjuvant pain treatment for a minimum of 7 days prior to screening.
6. If currently on anti-cancer therapy (hormonal therapy, biological, chemotherapy, targeted therapy or radiotherapy), having a stable regimen of existing anti-cancer therapy for a minimum of 2 weeks prior to screening.
7. Patients with a functional status of 0 - 3 as measured using the ECOG Scale of Performance Status.
8. Patients must be competent to understand the nature of the study & capable of giving written informed consent. Be willing to report for the scheduled study visits, complete the study questionnaires, daily Diary Card and communicate to study personnel about adverse events and concomitant medication use.
9. Patients must have adequate haematological, hepatic and renal functions:
a. Absolute neutrophil count (ANC) > 1.5 x10^9/L
b. Platelet count > 100 x10^9/L
c. Haemoglobin > 90 g/L (> 9.0 g/dL)
d. Total bilirubin < 1.5 ULN (< 2.5 ULN if patient has Gilbert’s syndrome)
e. AST/ALT ≤ 2.5 x ULN; ≤ 5 x ULN for patients with liver metastases or liver cancer
f. Serum creatinine within normal range or calculated creatinine clearance > 50 ml/min
g. Serum potassium and magnesium levels within normal range (sodium and magnesium replacement strategies may be used in case of deficiency)
h. Serum electrolyte levels including sodium, calcium, phosphorus, and chloride within normal range or deemed not clinically significant by the Investigator
Patients whose serum electrolyte levels (including those listed in (g) and (h) above) are not within normal range can be re-tested once during the screening period.
10. The patient is able to take oral medication.
11. Female patients must be of either:
a. Non-childbearing potential, post-menopausal, provided there is a laboratory confirmed serum follicle stimulating hormone (FSH) level >= 40mIU/mL at screening (the test is waived for females >= 70 years old), or there is a confirmed clinical history of sterility (e.g. the patient is without a uterus)
b. Childbearing potential, provided there are pregnancy test results prior to study treatment, to rule out pregnancy.
12. Male and female patients of childbearing potential must agree not to become pregnant and be willing to consent to using effective contraception, starting at screening and continuing throughout the clinical study period and for 90 days after the last study visit. Refer to Section 8.1.4 for effective contraceptive methods.
13. Patients agrees not to participate in another interventional study while participating in the present clinical study.
Exclusion Criteria
1. Patients with any small intestinal diseases.
2. Patients with a documented history of HIV or AIDS, autoimmune disorders (including Crohn’s Disease and Inflammatory Bowel Disease) or history of organ transplantation who require immunosuppressive therapy.
3. Unplanned palliative radiotherapy during the study for the purpose of relieving pain.
4. Planned major surgery during the study.
5. If females, is breastfeeding.
6. Patients with ECG evidence of a QTcF > 450 milliseconds in men and > 470 milliseconds in women. Patients with cardiac arrhythmia including bradycardia, history of atrial fibrillation or paroxysmal atrial fibrillation, history of atrial flutter, supraventricular tachycardia (atrial fibrillation, paroxysmal supraventricular tachycardia, atrial flutter or Wolff-Parkinson-White syndrome), ventricular tachycardia, history of ventricular fibrillation, secondary and third degree AV block, or patients who are receiving or plan to receive medications which affect QTc. A list of these medications is provided in Appendix 7.
7. Patients with uncontrolled cardiac disease (e.g. uncontrolled hypertension (diastolic blood pressure (DBP) >100, or systolic blood pressure (SBP) >180), severe and unstable angina, recent myocardial infarction (within the 12 months prior to screening).
8. Patients with moderate to severe heart failure - New York Heart Association (NYHA) Class III or IV.
9. Patients with known hypersensitivities, allergies to sodium meta-arsenite, related compounds or any of the excipients of the study drug (for details see Section 6.1).
10. Treatment with any investigational therapy within the 4 weeks prior to screening.
11. Unresolved toxicity to > Grade 2 Common Terminology Criteria for Adverse Events (CTCAE version 5.0), attributed to any prior therapies (excluding haemoglobin, alopecia, pigmentation, and chemotherapy-induced neurotoxicity).
12. Patients, who in the opinion of the Investigator, are inappropriate for enrolment into the study.
2. Patients with a documented history of HIV or AIDS, autoimmune disorders (including Crohn’s Disease and Inflammatory Bowel Disease) or history of organ transplantation who require immunosuppressive therapy.
3. Unplanned palliative radiotherapy during the study for the purpose of relieving pain.
4. Planned major surgery during the study.
5. If females, is breastfeeding.
6. Patients with ECG evidence of a QTcF > 450 milliseconds in men and > 470 milliseconds in women. Patients with cardiac arrhythmia including bradycardia, history of atrial fibrillation or paroxysmal atrial fibrillation, history of atrial flutter, supraventricular tachycardia (atrial fibrillation, paroxysmal supraventricular tachycardia, atrial flutter or Wolff-Parkinson-White syndrome), ventricular tachycardia, history of ventricular fibrillation, secondary and third degree AV block, or patients who are receiving or plan to receive medications which affect QTc. A list of these medications is provided in Appendix 7.
7. Patients with uncontrolled cardiac disease (e.g. uncontrolled hypertension (diastolic blood pressure (DBP) >100, or systolic blood pressure (SBP) >180), severe and unstable angina, recent myocardial infarction (within the 12 months prior to screening).
8. Patients with moderate to severe heart failure - New York Heart Association (NYHA) Class III or IV.
9. Patients with known hypersensitivities, allergies to sodium meta-arsenite, related compounds or any of the excipients of the study drug (for details see Section 6.1).
10. Treatment with any investigational therapy within the 4 weeks prior to screening.
11. Unresolved toxicity to > Grade 2 Common Terminology Criteria for Adverse Events (CTCAE version 5.0), attributed to any prior therapies (excluding haemoglobin, alopecia, pigmentation, and chemotherapy-induced neurotoxicity).
12. Patients, who in the opinion of the Investigator, are inappropriate for enrolment into the study.
The Estimated Number of Participants
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Taiwan
64 participants
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Global
participants
