Clinical trial NCT03113942: Study of Pomalidomide in Anal Cancer Precursors
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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: High Grade Squamous Intra-epithelial Lesion (HSIL)
Lead sponsor: Kirby Institute
Sponsor class: OTHER_GOV
First posted: 2017-04-14
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label, phase 2 trial, single-arm
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. 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).
2. No history of thromboembolic disease
3. No evidence of anal cancer or Superficially Invasive Squamous Cell Carcinoma of the Anus (SISCCA)
4. Willingness to use appropriate contraception (including refraining from sperm donation)
5. Age 18 years or older
6. Provision of written informed consent

   In addition, for subjects with HIV:
7. Adherence to a stable suppressive antiretroviral therapy (ART) regimen, unchanged for at least two months
8. CD4+ count ≥ 200 cells/µl
9. HIV viral load \< 200 copies/mL for at least six months

Exclusion Criteria:

1. Absolute neutrophil count (ANC) \<1000 cells/μL
2. Haemoglobin \<10.0 g/dL
3. Platelet count \<75,000 cells/μL
4. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> three times upper limit of normal
5. Calculated or measured creatinine clearance (CLCr) ≤ 50 mL/min (calculated by Cockcroft-Gault formula)
6. 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
7. Current pregnancy or breastfeeding
8. 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

Primary outcomes:
measure: Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy
timeFrame: 6 months
Secondary outcomes:
measure: incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
timeFrame: 6 months
measure: number of subjects completing of full six month course
timeFrame: 6 months
measure: effect of pomalidomide on self-reported health related quality of life and cancer anxiety during and after therapy
timeFrame: 6 months
Other outcomes:
Locations:
city: Darlinghurst
country: Australia
facility: St Vincent's Hospital
state: New South Wales
Study officials:
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