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Study of Pomalidomide in Anal Cancer Precursors

Phase 2
Conditions
High Grade Squamous Intra-epithelial Lesion (HSIL)
Interventions
Registration Number
NCT03113942
Lead Sponsor
Kirby Institute
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
26
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Pomalidomide groupPomalidomide 2 MG Oral Capsule [Pomalyst]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).
Primary Outcome Measures
NameTimeMethod
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy6 months

Histological high grade squamous intra-epithelial lesion clearance

Secondary Outcome Measures
NameTimeMethod
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)6 months

incidence of grade 3 and 4 adverse events and therapy delays (tolerability)

number of subjects completing of full six month course6 months

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 therapy6 months

individual patient change in quality of life questionnaire (SF12) from baseline to mid therapy and end therapy

Trial Locations

Locations (1)

St Vincent's Hospital

🇦🇺

Darlinghurst, New South Wales, Australia

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