Back to resultsStudy of Pomalidomide in Anal Cancer Precursors (SPACE)
Primary Purpose
High Grade Squamous Intra-epithelial Lesion (HSIL)
Status
Active
Phase
Phase 2
Locations
Australia
Study Type
Interventional
Intervention
Pomalidomide 2 MG Oral Capsule [Pomalyst]
Sponsored by
About this trial
This is an interventional treatment trial for High Grade Squamous Intra-epithelial Lesion (HSIL)
Eligibility Criteria
Inclusion Criteria:
Persistent high grade squamous intra-epithelial lesion (HSIL) which must meet all of the following criteria:
i. Pathologically confirmed grade 2 or 3 AIN demonstrated by high resolution anoscopy with grade on each occasion re-confirmed at screening by nominated study pathologist from Douglas Hanly Moir (DHM) (pathology case review to be conducted prior to enrolment) ii. Lesion must have been visualised on at least three sequential occasions over at least 12 months, including the pre enrolment screening high resolution anoscopy (HRA).
iii. Lesion must have persistent geographical characteristics consistent with a single lesion observed over time (as defined in the Manual of Operations).
- No history of thromboembolic disease
- No evidence of anal cancer or Superficially Invasive Squamous Cell Carcinoma of the Anus (SISCCA)
- Willingness to use appropriate contraception (including refraining from sperm donation)
- Age 18 years or older
Provision of written informed consent
In addition, for subjects with HIV:
- Adherence to a stable suppressive antiretroviral therapy (ART) regimen, unchanged for at least two months
- CD4+ count ≥ 200 cells/µl
- HIV viral load < 200 copies/mL for at least six months
Exclusion Criteria:
- Absolute neutrophil count (ANC) <1000 cells/μL
- Haemoglobin <10.0 g/dL
- Platelet count <75,000 cells/μL
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > three times upper limit of normal
- Calculated or measured creatinine clearance (CLCr) ≤ 50 mL/min (calculated by Cockcroft-Gault formula)
- Patients with significant cardiac dysfunction including congestive heart failure, NY Heart Association Class II; Myocardial infarction within 12 months of starting study; unstable of poorly controlled angina
- Current pregnancy or breastfeeding
- Any condition not already outlined above which, in the opinion of the clinical investigator, would place the subject at risk if they participated or would jeopardise adherence or follow up
Sites / Locations
- St Vincent's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Pomalidomide group
Arm Description
Open label - all participants will receive pomalidomide 2mg orally once a day for 6 cycles (21 days on treatment and a 7 day rest period constitutes a cycle).
Outcomes
Primary Outcome Measures
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy
Histological high grade squamous intra-epithelial lesion clearance
Secondary Outcome Measures
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
number of subjects completing of full six month course
number of subjects completing of full six month course (feasibility of polidamide in this setting)
effect of pomalidomide on self-reported health related quality of life and cancer anxiety during and after therapy
individual patient change in quality of life questionnaire (SF12) from baseline to mid therapy and end therapy
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03113942
Brief Title
Study of Pomalidomide in Anal Cancer Precursors
Acronym
SPACE
Official Title
Study of Pomalidomide in Anal Cancer Precursors (SPACE): a Phase 2 Study of Immunomodulation in People With Persistent HPV-associated High Grade Squamous Intraepithelial Lesions
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 14, 2017 (Actual)
Primary Completion Date
March 31, 2019 (Actual)
Study Completion Date
December 31, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kirby Institute
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a single centre open label phase II trial to determine the antitumor efficacy of the oral immunomodulatory agent pomalidomide in persistent human papillomavirus (HPV) -associated high grade squamous intra-epithelial lesions (HSIL) in patients with and without human immunodeficiency virus (HIV) infection.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
High Grade Squamous Intra-epithelial Lesion (HSIL)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open label, phase 2 trial, single-arm
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Pomalidomide group
Arm Type
Experimental
Arm Description
Open label - all participants will receive pomalidomide 2mg orally once a day for 6 cycles (21 days on treatment and a 7 day rest period constitutes a cycle).
Intervention Type
Drug
Intervention Name(s)
Pomalidomide 2 MG Oral Capsule [Pomalyst]
Other Intervention Name(s)
pomalidomide, Pomalyst
Intervention Description
Pomalidomide is an oral immunomodulatory derivative of thalidomide. Thalidomide and its derivatives are small molecules with broad effects on immune activation, including T-cell activation and responsiveness. Pomalidomide augments T cell responsiveness and proliferation by several mechanisms, many mediated by transcriptional regulation downstream of its primary target, cereblon. Effects include increased production of IL-2 and interferon-γ (IFN-γ), enhanced CD4+ and CD8+ T cell co-stimulation.
Primary Outcome Measure Information:
Title
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy
Description
Histological high grade squamous intra-epithelial lesion clearance
Time Frame
6 months
Secondary Outcome Measure Information:
Title
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
Description
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
Time Frame
6 months
Title
number of subjects completing of full six month course
Description
number of subjects completing of full six month course (feasibility of polidamide in this setting)
Time Frame
6 months
Title
effect of pomalidomide on self-reported health related quality of life and cancer anxiety during and after therapy
Description
individual patient change in quality of life questionnaire (SF12) from baseline to mid therapy and end therapy
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Persistent high grade squamous intra-epithelial lesion (HSIL) which must meet all of the following criteria:
i. Pathologically confirmed grade 2 or 3 AIN demonstrated by high resolution anoscopy with grade on each occasion re-confirmed at screening by nominated study pathologist from Douglas Hanly Moir (DHM) (pathology case review to be conducted prior to enrolment) ii. Lesion must have been visualised on at least three sequential occasions over at least 12 months, including the pre enrolment screening high resolution anoscopy (HRA).
iii. Lesion must have persistent geographical characteristics consistent with a single lesion observed over time (as defined in the Manual of Operations).
No history of thromboembolic disease
No evidence of anal cancer or Superficially Invasive Squamous Cell Carcinoma of the Anus (SISCCA)
Willingness to use appropriate contraception (including refraining from sperm donation)
Age 18 years or older
Provision of written informed consent
In addition, for subjects with HIV:
Adherence to a stable suppressive antiretroviral therapy (ART) regimen, unchanged for at least two months
CD4+ count ≥ 200 cells/µl
HIV viral load < 200 copies/mL for at least six months
Exclusion Criteria:
Absolute neutrophil count (ANC) <1000 cells/μL
Haemoglobin <10.0 g/dL
Platelet count <75,000 cells/μL
Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > three times upper limit of normal
Calculated or measured creatinine clearance (CLCr) ≤ 50 mL/min (calculated by Cockcroft-Gault formula)
Patients with significant cardiac dysfunction including congestive heart failure, NY Heart Association Class II; Myocardial infarction within 12 months of starting study; unstable of poorly controlled angina
Current pregnancy or breastfeeding
Any condition not already outlined above which, in the opinion of the clinical investigator, would place the subject at risk if they participated or would jeopardise adherence or follow up
Facility Information:
Facility Name
St Vincent's Hospital
City
Darlinghurst
State/Province
New South Wales
ZIP/Postal Code
2010
Country
Australia
12. IPD Sharing Statement
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Study of Pomalidomide in Anal Cancer Precursors
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