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Trial registered on ANZCTR


Registration number
ACTRN12615000987561
Ethics application status
Approved
Date submitted
18/08/2015
Date registered
22/09/2015
Date last updated
4/11/2016
Type of registration
Prospectively registered

Titles & IDs
Public title
Study of PAX-1 in Patients With Persistent Cancer Pain in the Palliative Setting
Scientific title
A Randomised, Double-Blind, Parallel-Group, Placebo Controlled Phase II Study to Assess the Clinical Benefit of 3 Doses of PAX-1 as Adjunctive Treatment for Persistent Cancer Pain
Secondary ID [1] 287224 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Moderate or Severe, Persistent Cancer Related Pain 288864 0
Condition category
Condition code
Cancer 289205 289205 0 0
Any cancer
Anaesthesiology 296269 296269 0 0
Pain management

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a multi-centre double-blind, four arm, parallel, randomised Phase 2 comparison study evaluating the efficacy and safety of PAX 1 verses Placebo in patients with persistent cancer pain.
Patients will be randomly assigned in a 1:1:1:1 ratio (7.5mg, 10mg, 12.5mg or placebo).
In addition to continuing with their usual background pain therapy, patients will take 5 study tablets daily for 28 days. 2 tablets before breakfast, 1 tablet before lunch and 2 tablets before dinner.
Patients will be asked to return unused study medication and the empty packaging at each weekly visit to ensure compliance to the study and to conduct patient drug accountability.
Intervention code [1] 286937 0
Treatment: Drugs
Comparator / control treatment
Placebo microcellulose tablet. Patients will also take their regular background pain medication.
Control group
Placebo

Outcomes
Primary outcome [1] 289319 0
Pain intensity as measured by mean daily pain intensity on an 11 (0-10) point Numerical Rating Scale.
Timepoint [1] 289319 0
Daily pain scores recorded in a dairy from the Screening visit until Day 36.
Secondary outcome [1] 302232 0
Efficacy of PAX 1 compared to placebo as assessed by pain. Assessed by the 11 point numerical pain scale.
Timepoint [1] 302232 0
Weekly review through 14 days following the last administration of study medication.
Secondary outcome [2] 302233 0
To evaluate the tolerability and safety of PAX1 as adjunctive treatment for persistent cancer pain. This will be evaluated by assessing ECG results, laboratory values, PK studies to assess the blood concentration of arsenic and metabolites of PAX-1 at weekly study visits from the Screening visit until Day 36.
Adverse events will be reviewed until Day 59.
Common side effects (which may be mild, moderate or severe) are nausea, vomiting, neutropenia, reduced appetite and swelling in the face.
Timepoint [2] 302233 0
Lab assessments including PK studies, and ECG's will be evaluated through 14 days following the last administration of study medication. There are 7 weekly blood tests and ECG's from the Screening visit until the Day 36 follow up visit.

Adverse Events will be followed for 30 days after the last administration of study medication. These are reviewed at the weekly visit with the study doctor.
Secondary outcome [3] 317406 0
Change in pain intensity (Change of mean pain scale from baseline, obtained from daily diary entries where the 11 Point Numerical Pain Rating Scale (NRS) is recorded)
Timepoint [3] 317406 0
Daily diary entry from the Screening visit until Day 36, (Visit 7).
Secondary outcome [4] 317407 0
Responder rate (proportion of participants who achieve >= 30% reduction in mean pain scale) obtained from daily diary entries where the 11 Point Numerical Pain Rating Scale (NRS) is recorded)
Timepoint [4] 317407 0
Daily diary entry from the Screening visit until Day 36, (Visit 7).
Secondary outcome [5] 317408 0
Breakthrough pain frequency obtained from daily diary entries where the 11 Point Numerical Pain Rating Scale (NRS) is recorded)
Timepoint [5] 317408 0
Daily diary entry from the Screening visit until Day 36 (Visit 7).
Secondary outcome [6] 317409 0
Background therapy utilisation, including oral morphine equivalents obtained from daily diary entries.
Timepoint [6] 317409 0
Daily diary entry from the Screening visit until Day 36 (Visit 7).
Secondary outcome [7] 317410 0
Constipation (Adverse Events) obtained from daily diary entries.
Timepoint [7] 317410 0
Daily diary entry from the Screening visit until Day 36 (Visit 7).
Secondary outcome [8] 317411 0
Appetite (NRS) obtained from daily diary entries.
Timepoint [8] 317411 0
Daily diary entry from the Screening visit until Day 36 (Visit 7).
Secondary outcome [9] 317412 0
Sleep quality (Sleep Interference NRS) obtained from daily diary entries.
Timepoint [9] 317412 0
Daily diary entry from the Screening visit until Day 36 (Visit 7).
Secondary outcome [10] 317413 0
Functional status (Australian-modified Karnofsky Performance Scale (AKPS) and Life Space Questionnaire(LSQ)
Timepoint [10] 317413 0
Obtained at weekly visit with the study doctor from the Screening visit to Day 36.
Secondary outcome [11] 317414 0
Participant reported outcome measures (BPI-SF and NPQ)
Timepoint [11] 317414 0
Obtained at weekly visit with the study doctor from the Screening visit to Day 36.
Secondary outcome [12] 317415 0
Patient global impression of change (PGIC).
Timepoint [12] 317415 0
Obtained in weekly visit with the study doctor from Day 8 (Visit 3) until Day 36 (Visit 7).

Eligibility
Key inclusion criteria
1) Participants with moderate or severe, persistent cancer-related pain who are receiving WHO Step 3 cancer pain treatment. The type of cancer is non specific and the patient should be in palliative care.

2) Pain generator (source of pain) must be primarily due to underlying cancer or cancer treatment

3) Pain intensity must be moderate or severe defined by an average daily pain score of >= 4 achieved over a minimum of 2 days within the 7 days preceding randomisation.

4) Australian-modified Karnofsky Performance Scale Score of >= 50

5) Ability to complete study questionnaires and provide written informed consent

6) Adequate haematological, hepatic and renal functions required

7) Participant is able to take medication orally

8) Women of childbearing potential must have a negative pregnancy test and agree to adequate birth control if conception is possible during the study and up to 6 months after the discontinuation of study medications; and males must agree to adequate birth control during the study and up to 6 months after the discontinuation of study medication.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1) Unstable pain at study Visit 1 (screening visit) and Visit 2 (randomisation visit)


2) Use of prohibited adjuvant pain treatment (including interventional pain procedures) in the 7 days preceding Visit 1 or plan to use these medications/procedures during the study,

3) Participants without a functional digestive system. Participants with percutaneous endoscopic gastrostomy (PEG), colectomy, colostomy, and conditions such as irritable bowel syndrome

4) Antitumour treatments (chemotherapy and targeted therapies) within 2 weeks prior to randomisation or planned to take place during the study.

5) HIV or AIDS, autoimmune disorders (including Crohn's Disease and Inflammatory Bowel Disease) or history of organ transplantation who require immunosuppressive therapy.

6) Has had certain other recent treatment e.g. extended field radiotherapy or limited field radiotherapy within 4 weeks prior to randomisation, or radiotherapy planned to take place during the study for the purpose of relieving pain

7) Planned major surgery during the study

8) Is a Lactating or pregnant female

9) Participants who required regular medications for a chronic pain syndrome prior to their diagnosis of cancer

10) Certain cardiac medical conditions

11) Has received any investigational product in the past 4 weeks prior to starting the study

12) known or suspected hypersensitivities, allergies to sodium meta-arsenite, related compounds or any of the excipients of the study drug (calcium phosphate dibasic anhydrous, silicified microcrystalline cellulose, sodium starch glycolate, colloidal silicon dioxide, and sodium stearyl fumarate)

13) Has any unresolved toxicity to <= CTCAE grade 2 attributed to any prior therapies

14) Taking an anti-platelet and anticoagulant combination or two or more anti-platelet agents.

15) Taking any medication containing arsenic trioxide.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
The study will be stratified by Centre only and a randomisation list will be prepared for each centre. A randomised block design will be used for each randomisation list. The randomisation number will be used for the medication kits and the numbering system for the case report form.
An independent statistician will be assigned to prepare the final randomisation list.
This study is double-blind; therefore the Sponsor personnel, Investigator, site staff, and patient will not know which treatment has been assigned.
If an emergency treatment code break is required then the Sponsors Medical Monitor and the site pharmacist will have access to the treatment assignment for each randomization number.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
An independent statistician will be assigned to prepare the final randomisation list.
A block randomisation schedule will be used.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
30/10/2016
Actual
Date of last participant enrolment
Anticipated
31/07/2018
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA

Funding & Sponsors
Funding source category [1] 287080 0
Commercial sector/Industry
Name [1] 287080 0
Bio And Gene Pty Ltd
Country [1] 287080 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Bio And Gene Pty Ltd
Address
Level 6, 23 Berry Street,
North Sydney NSW 2060
Country
Australia
Secondary sponsor category [1] 285855 0
None
Name [1] 285855 0
Address [1] 285855 0
Country [1] 285855 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 289094 0
Hunter New England Local Health District
Ethics committee address [1] 289094 0
Locked Bag No 1
New Lambton NSW 2305
Ethics committee country [1] 289094 0
Australia
Date submitted for ethics approval [1] 289094 0
Approval date [1] 289094 0
02/04/2013
Ethics approval number [1] 289094 0
13/02/20/3.03
Ethics committee name [2] 289095 0
Bellberry Limited
Ethics committee address [2] 289095 0
229 Greenhill Road
Dulwich South Australia 5065
Ethics committee country [2] 289095 0
Australia
Date submitted for ethics approval [2] 289095 0
Approval date [2] 289095 0
11/02/2013
Ethics approval number [2] 289095 0
2012-12-1230
Ethics committee name [3] 289096 0
Southern Adelaide Clinical HREC
Ethics committee address [3] 289096 0
The Flats G5 – Rooms 3 and 4
Flinders Drive
Flinders Medical Centre, Bedford Park SA 5042
Ethics committee country [3] 289096 0
Australia
Date submitted for ethics approval [3] 289096 0
04/02/2013
Approval date [3] 289096 0
12/05/2015
Ethics approval number [3] 289096 0
26.13

Summary
Brief summary
The purpose of this study is to assess if the drug, PAX-1, can reduce cancer pain when used in addition to usual pain medication. It will also assess the side effects it may cause.

Who is it for? You may be eligible to join this study if you are aged 18 years or above and are experiencing moderate or severe, persistent cancer-related pain are in the palliative setting.
Study details: Participants in this study will be randomly (by chance) allocated to one of four groups. Participants in three of the groups will receive the study drug, PAX-1, at a dose of either 7.5mg, 10mg, or 12.5mg . Participants in the fourth group will receive a placebo (inactive) tablet. The tablets are taken orally 3 times a day for 28 days. All participants will continue to take their usual pain medication in addition to the study medication. They will not know to which group they have been allocated until after the study. Participants will be asked to record their daily pain intensity on a numerical rating scale. They will also be asked to complete questionnaires on appetite, sleep quality, functional status, and quality of life and will undergo safety and tolerability evaluations involving blood tests, ECGs and monitoring of adverse events. This will enable us to determine the clinical benefit of PAX-1 for cancer patients in the palliative setting.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 39218 0
Prof David Currow
Address 39218 0
Palliative and Supportive Services
Flinders University
Flinders Drive
Bedford Park
South Australia 5042
Country 39218 0
Australia
Phone 39218 0
+61 8 8275 1732
Fax 39218 0
Email 39218 0
[email protected]
Contact person for public queries
Name 39219 0
Andrew Han
Address 39219 0
Bio And Gene Pty. Ltd,
Level 6, 23 Berry Street,
North Sydney NSW 2060 Australia
Country 39219 0
Australia
Phone 39219 0
+61 2 8021 1521
Fax 39219 0
Email 39219 0
[email protected]
Contact person for scientific queries
Name 39220 0
Andrew Han
Address 39220 0
Bio And Gene Pty. Ltd,
Level 6, 23 Berry Street,
North Sydney NSW 2060 Australia
Country 39220 0
Australia
Phone 39220 0
+61 2 8021 1521
Fax 39220 0
Email 39220 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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