High Grade Squamous Intra-epithelial Lesion (HSIL)Phase 2SPACENCT03113942
Study of Pomalidomide in Anal Cancer Precursors
A Phase 2 study testing Pomalidomide 2 MG Oral Capsule [Pomalyst] in people with High Grade Squamous Intra-epithelial Lesion (HSIL), run by Kirby Institute at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- High Grade Squamous Intra-epithelial Lesion (HSIL)
- What is being tested?
- Pomalidomide 2 MG Oral Capsule [Pomalyst]
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteAustralia
- Study dates
- 14 Jun 2017to 31 Dec 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Persistent high grade squamous intra-epithelial lesion (HSIL) which must meet all of the following criteria:
- 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).
- 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)
Show all 12Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Being tested
- Pomalidomide 2 MG Oral Capsule [Pomalyst]
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pomalidomide 2 MG Oral Capsule [Pomalyst]Drug
Also called pomalidomide, Pomalyst
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.
| Study group | Role | What they receive |
|---|---|---|
| Pomalidomide group | Experimental | 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). Receives: Drug: Pomalidomide 2 MG Oral Capsule [Pomalyst] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy
Histological high grade squamous intra-epithelial lesion clearance
Measured: 6 months
Secondary outcomes (3)
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
Measured: 6 months
number of subjects completing of full six month course
number of subjects completing of full six month course (feasibility of polidamide in this setting)
Measured: 6 months
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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2017
- Study start14 Jun 2017
- Primary completion31 Mar 2019
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (26)Papers the sponsor has linked to this study.
- Other trials for High Grade Squamous Intra-epithelial Lesion (HSIL)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label, phase 2 trial, single-arm
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kirby Institute
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03113942
- Sponsor's protocol ID
- SPACE
- Acronym
- SPACE
- Status last verified
- Mar 2022
- First submitted
- 8 Mar 2017
- Last updated
- 31 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Munoz N, Castellsague X, Berrington de Gonzalez A, Gissmann L. Chapter 1: HPV in the etiology of human cancer. Vaccine. 2006 Aug 31;24 Suppl 3:S3/1-10. doi: 10.1016/j.vaccine.2006.05.115. Epub 2006 Jun 23.
PubMed 16949995Parkin DM, Bray F. Chapter 2: The burden of HPV-related cancers. Vaccine. 2006 Aug 31;24 Suppl 3:S3/11-25. doi: 10.1016/j.vaccine.2006.05.111.
PubMed 16949997World Health Organization International Agency for Reseach on Cancer. IARC Monograph on the Evaluation of Carcinogenic Risks to Humans: Volume 90, Human Papillomavirus. World Health Organization, Geneva 2007.
Machalek DA, Poynten M, Jin F, Fairley CK, Farnsworth A, Garland SM, Hillman RJ, Petoumenos K, Roberts J, Tabrizi SN, Templeton DJ, Grulich AE. Anal human papillomavirus infection and associated neoplastic lesions in men who have sex with men: a systematic review and meta-analysis. Lancet Oncol. 2012 May;13(5):487-500. doi: 10.1016/S1470-2045(12)70080-3. Epub 2012 Mar 23.
PubMed 22445259Palefsky JM. Anal cancer prevention in HIV-positive men and women. Curr Opin Oncol. 2009 Sep;21(5):433-8. doi: 10.1097/CCO.0b013e32832f511a.
PubMed 19587592Grulich AE, Poynten IM, Machalek DA, Jin F, Templeton DJ, Hillman RJ. The epidemiology of anal cancer. Sex Health. 2012 Dec;9(6):504-8. doi: 10.1071/SH12070.
PubMed 22958581Petoumenos K, van Leuwen MT, Vajdic CM, Woolley I, Chuah J, Templeton DJ, Grulich AE, Law MG; Australian HIV Observational Database. Cancer, immunodeficiency and antiretroviral treatment: results from the Australian HIV Observational Database (AHOD). HIV Med. 2013 Feb;14(2):77-84. doi: 10.1111/j.1468-1293.2012.01038.x. Epub 2012 Aug 30.
PubMed 22934689Yarchoan R, Tosato G, Little RF. Therapy insight: AIDS-related malignancies--the influence of antiviral therapy on pathogenesis and management. Nat Clin Pract Oncol. 2005 Aug;2(8):406-15; quiz 423. doi: 10.1038/ncponc0253.
PubMed 16130937Ajani JA, Winter KA, Gunderson LL, Pedersen J, Benson AB 3rd, Thomas CR Jr, Mayer RJ, Haddock MG, Rich TA, Willett C. Fluorouracil, mitomycin, and radiotherapy vs fluorouracil, cisplatin, and radiotherapy for carcinoma of the anal canal: a randomized controlled trial. JAMA. 2008 Apr 23;299(16):1914-21. doi: 10.1001/jama.299.16.1914.
PubMed 18430910Moscicki AB, Schiffman M, Kjaer S, Villa LL. Chapter 5: Updating the natural history of HPV and anogenital cancer. Vaccine. 2006 Aug 31;24 Suppl 3:S3/42-51. doi: 10.1016/j.vaccine.2006.06.018. Epub 2006 Jun 23.
PubMed 16950017Tong WW, Shepherd K, Garland S, Meagher A, Templeton DJ, Fairley CK, Jin F, Poynten IM, Zaunders J, Hillman RJ, Grulich AE, Kelleher AD, Carr A; Study of the Prevention of Anal Cancer (SPANC) team. Human papillomavirus 16-specific T-cell responses and spontaneous regression of anal high-grade squamous intraepithelial lesions. J Infect Dis. 2015 Feb 1;211(3):405-15. doi: 10.1093/infdis/jiu461. Epub 2014 Aug 19.
PubMed 25139018Quach H, Ritchie D, Stewart AK, Neeson P, Harrison S, Smyth MJ, Prince HM. Mechanism of action of immunomodulatory drugs (IMiDS) in multiple myeloma. Leukemia. 2010 Jan;24(1):22-32. doi: 10.1038/leu.2009.236. Epub 2009 Nov 12.
PubMed 19907437Ito T, Ando H, Suzuki T, Ogura T, Hotta K, Imamura Y, Yamaguchi Y, Handa H. Identification of a primary target of thalidomide teratogenicity. Science. 2010 Mar 12;327(5971):1345-50. doi: 10.1126/science.1177319.
PubMed 20223979Gorgun G, Calabrese E, Soydan E, Hideshima T, Perrone G, Bandi M, Cirstea D, Santo L, Hu Y, Tai YT, Nahar S, Mimura N, Fabre C, Raje N, Munshi N, Richardson P, Anderson KC. Immunomodulatory effects of lenalidomide and pomalidomide on interaction of tumor and bone marrow accessory cells in multiple myeloma. Blood. 2010 Oct 28;116(17):3227-37. doi: 10.1182/blood-2010-04-279893. Epub 2010 Jul 22.
PubMed 20651070Lentzsch S, LeBlanc R, Podar K, Davies F, Lin B, Hideshima T, Catley L, Stirling DI, Anderson KC. Immunomodulatory analogs of thalidomide inhibit growth of Hs Sultan cells and angiogenesis in vivo. Leukemia. 2003 Jan;17(1):41-4. doi: 10.1038/sj.leu.2402745.
PubMed 12529658
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03113942
- Acronym / sponsor ID
- SPACE / SPACE
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Kirby InstituteOther government
- Study start
- 14 Jun 2017 (actual)
- Primary completion
- 31 Mar 2019 (actual)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 14 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label, phase 2 trial, single-arm
| Arm | Type | Receives |
|---|---|---|
| Pomalidomide group | Experimental | Drug: Pomalidomide 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Persistent high grade squamous intra-epithelial lesion (HSIL) which must meet all of the following criteria:
- 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
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pomalidomide 2 MG Oral Capsule [Pomalyst]Also: pomalidomide, PomalystPomalidomide 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. | Drug | Pomalidomide group |
Primary outcome
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy
Time frame: 6 months
Histological high grade squamous intra-epithelial lesion clearance
3 secondary outcomes
incidence of grade 3 and 4 adverse events and therapy delays (tolerability)
Time frame: 6 months
number of subjects completing of full six month course
Time frame: 6 months
effect of pomalidomide on self-reported health related quality of life and cancer anxiety during and after therapy
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2022.
- Background: PubMed 16949995Munoz N, Castellsague X, Berrington de Gonzalez A, Gissmann L. Chapter 1: HPV in the etiology of human cancer. Vaccine. 2006 Aug 31;24 Suppl 3:S3/1-10. doi: 10.1016/j.vaccine.2006.05.115. Epub 2006 Jun 23.
- Background: PubMed 16949997Parkin DM, Bray F. Chapter 2: The burden of HPV-related cancers. Vaccine. 2006 Aug 31;24 Suppl 3:S3/11-25. doi: 10.1016/j.vaccine.2006.05.111.
- BackgroundWorld Health Organization International Agency for Reseach on Cancer. IARC Monograph on the Evaluation of Carcinogenic Risks to Humans: Volume 90, Human Papillomavirus. World Health Organization, Geneva 2007.
- Background: PubMed 22445259Machalek DA, Poynten M, Jin F, Fairley CK, Farnsworth A, Garland SM, Hillman RJ, Petoumenos K, Roberts J, Tabrizi SN, Templeton DJ, Grulich AE. Anal human papillomavirus infection and associated neoplastic lesions in men who have sex with men: a systematic review and meta-analysis. Lancet Oncol. 2012 May;13(5):487-500. doi: 10.1016/S1470-2045(12)70080-3. Epub 2012 Mar 23.
- Background: PubMed 19587592Palefsky JM. Anal cancer prevention in HIV-positive men and women. Curr Opin Oncol. 2009 Sep;21(5):433-8. doi: 10.1097/CCO.0b013e32832f511a.
- Background: PubMed 22958581Grulich AE, Poynten IM, Machalek DA, Jin F, Templeton DJ, Hillman RJ. The epidemiology of anal cancer. Sex Health. 2012 Dec;9(6):504-8. doi: 10.1071/SH12070.
- Background: PubMed 22934689Petoumenos K, van Leuwen MT, Vajdic CM, Woolley I, Chuah J, Templeton DJ, Grulich AE, Law MG; Australian HIV Observational Database. Cancer, immunodeficiency and antiretroviral treatment: results from the Australian HIV Observational Database (AHOD). HIV Med. 2013 Feb;14(2):77-84. doi: 10.1111/j.1468-1293.2012.01038.x. Epub 2012 Aug 30.
- Background: PubMed 16130937Yarchoan R, Tosato G, Little RF. Therapy insight: AIDS-related malignancies--the influence of antiviral therapy on pathogenesis and management. Nat Clin Pract Oncol. 2005 Aug;2(8):406-15; quiz 423. doi: 10.1038/ncponc0253.
- Background: PubMed 18430910Ajani JA, Winter KA, Gunderson LL, Pedersen J, Benson AB 3rd, Thomas CR Jr, Mayer RJ, Haddock MG, Rich TA, Willett C. Fluorouracil, mitomycin, and radiotherapy vs fluorouracil, cisplatin, and radiotherapy for carcinoma of the anal canal: a randomized controlled trial. JAMA. 2008 Apr 23;299(16):1914-21. doi: 10.1001/jama.299.16.1914.
- Background: PubMed 16950017Moscicki AB, Schiffman M, Kjaer S, Villa LL. Chapter 5: Updating the natural history of HPV and anogenital cancer. Vaccine. 2006 Aug 31;24 Suppl 3:S3/42-51. doi: 10.1016/j.vaccine.2006.06.018. Epub 2006 Jun 23.
- Background: PubMed 25139018Tong WW, Shepherd K, Garland S, Meagher A, Templeton DJ, Fairley CK, Jin F, Poynten IM, Zaunders J, Hillman RJ, Grulich AE, Kelleher AD, Carr A; Study of the Prevention of Anal Cancer (SPANC) team. Human papillomavirus 16-specific T-cell responses and spontaneous regression of anal high-grade squamous intraepithelial lesions. J Infect Dis. 2015 Feb 1;211(3):405-15. doi: 10.1093/infdis/jiu461. Epub 2014 Aug 19.
- Background: PubMed 19907437Quach H, Ritchie D, Stewart AK, Neeson P, Harrison S, Smyth MJ, Prince HM. Mechanism of action of immunomodulatory drugs (IMiDS) in multiple myeloma. Leukemia. 2010 Jan;24(1):22-32. doi: 10.1038/leu.2009.236. Epub 2009 Nov 12.
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Trial to Evaluate Nonavalent HPV Vaccine as a Secondary Prevention in Patients Treated for Human Papilloma Virus Related High-grade Squamous Intraepithelial Lesions
Patients With Primary HPV High-grade Squamous Intra-epithelial Lesions (HSIL) at Vulvar Site, Patients With Primary HPV High-grade Squamous Intra-epithelial Lesions (HSIL) at Vaginal Site
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pomalidomide 2 MG Oral Capsule [Pomalyst] in people with High Grade Squamous Intra-epithelial Lesion (HSIL), run by Kirby Institute at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 31 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Histological High Grade Squamous Intraepithelial Lesions (HSIL) clearance at 6 months of therapy (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03113942, which also lists 26 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2022.