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Immunotherapy After Transplantation for Skin Cancer Prevention in Organ Transplant Recipients

Phase 2
Withdrawn
Conditions
Immunotherapy
Cutaneous Squamous Cell Carcinoma
Actinic Keratoses
Organ Transplant Recipients
Skin Cancer
Interventions
Registration Number
NCT04642287
Lead Sponsor
Massachusetts General Hospital
Brief Summary

This clinical trial aims to investigate the efficacy of Calcipotriol ointment combined with 5-FU cream in Organ Transplant Recipients (OTRs) to determine if it can stimulate the immune cells against actinic keratoses precancerous skin lesions after transplantation and prevent cutaneous squamous cell carcinoma (SCC) in long-term.

Detailed Description

The main goal of this investigator-initiated clinical trial is to determine the efficacy of topical calcipotriol combined with 5-fluorouracil (5-FU) treatment in OTRs on immunosuppressive medications with precancerous skin lesions called actinic keratoses (AKs) and a history of non-melanoma skin cancer in order to eliminate AKs and prevent squamous cell carcinoma (SCC) development. SCC is the most common cutaneous malignancy seen after transplantation, with a 65-250fold greater incidence in organ transplant recipients (OTRs) compared to the general population. This increased risk is due to the systemic immunosuppression caused by anti-rejection medications, which are indispensable for protecting against allograft loss. Our previous findings have established the efficacy of calcipotriol in combination with 5-FU in inducing an antitumor immunity against AKs in immunocompetent patients. This SCC risk reduction is accompanied by the induction of robust T cell immunity and TRM cell formation against AKs. Calcipotriol is a FDA-approved low calcemic vitamin D analogue for the treatment of psoriasis. Topical 5-FU is a standard chemotherapy for AKs. Based on our previous findings demonstrating the synergistic impact of TSLP induction by calcipotriol in combination with the cytotoxic effects of 5-FU that leads to a robust T cell immunity against early skin carcinogenesis in immunocompetent patients, we aim to determine whether this efficacy is maintained in OTRs on immunosuppressive therapy and its effect on SCC prevention in long-term after transplantation.

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
64
Inclusion Criteria
  • Solid organ transplant recipients with AKs and a history of non-melanoma skin cancer in one year prior to enrollment into the study. The target population includes post-transplant OTRs.
  • Presence of four to fifteen clinically typical, visible, and discrete AKs in 25 cm2 on any of the four anatomical sites: scalp, face, right upper extremity and left upper extremity.
  • The period between the first visit and transplantation is minimum 4 weeks and maximum 12 months.
  • Age of at least 18 years
  • Ability and willingness of the patient to participate in the study (Informed consent will be obtained)
Exclusion Criteria
  • Treatment area is within 5 cm of an incompletely healed wound or a suspected basal cell or squamous cell carcinoma.
  • Treatment area contained hypertrophic and hyperkeratotic lesions, cutaneous horns, or lesions that had not responded to repeated cryotherapy.
  • Patients with history of hypercalcemia or vitamin D toxicity.
  • Female participants must be either of non-reproductive potential (i.e., post-menopausal by history of age > 50 years old and no menses for >1 year without an alternative medical cause; OR history of hysterectomy, history of bilateral tubal ligation, or history of bilateral oophorectomy) OR must have a negative serum pregnancy test within 7 days prior to study registration.
  • Patients with DPD (Dihydropyrimidine Dehydrogenase) deficiency (due to their higher risk of 5-FU toxicity).

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Topical Calcipotriol ointment plus 5-Fluorouracil creamCalcipotriol Only Product in Cutaneous Dose FormTopical Calcipotriol 0.0025% ointment plus 5-Fluorouracil 2.5% cream will be administered by the participants to their face, scalp and upper extremities twice a day for 6 consecutive days.
Topical Calcipotriol ointment plus 5-Fluorouracil creamTopical 5FUTopical Calcipotriol 0.0025% ointment plus 5-Fluorouracil 2.5% cream will be administered by the participants to their face, scalp and upper extremities twice a day for 6 consecutive days.
Topical vaseline plus 5-Fluorouracil 2.5% creamVaselineTopical Vaseline plus 5-Fluorouracil 2.5% cream will be administered by the participants to their face, scalp and upper extremities twice a day for 6 consecutive days.
Topical vaseline plus 5-Fluorouracil 2.5% creamTopical 5FUTopical Vaseline plus 5-Fluorouracil 2.5% cream will be administered by the participants to their face, scalp and upper extremities twice a day for 6 consecutive days.
Primary Outcome Measures
NameTimeMethod
The changes in baseline number of AKs on treated anatomical sites in post-transplant OTRs8 weeks after treatment

The changes in baseline number of AKs on treated anatomical sites in post-transplant OTRs quantified based on participants' medical records and photographs in test versus control group

Secondary Outcome Measures
NameTimeMethod
The changes in the number of SCC on treated anatomical sites in post-transplant OTRs1, 2 and 4 years after treatment

The changes in number of SCC on treated anatomical sites in post-transplant OTRs quantified based on participants' medical records, photographs and pathology results in test versus control group

The changes in the magnitude of TSLP, CD3+, CD4+ and CD8+ TRM cell infiltrates in in the AK and normal skin after transplantationat one day after 6-day treatment and at one year post-treatment

The changes in the magnitude of TSLP, CD3+, CD4+ and CD8+ TRM cell infiltrates in OTRs after transplantation compared to before transplantation in test versus control group.

The changes in immune infiltrate (CD3+, CD4+ and CD8+ TRM cell) in any SCC that develops after treatmentup to 4 years after treatment

The changes in immune infiltrate in any SCC that develops after calcipotriol plus 5-FU versus Vaseline plus 5-FU treatment for up to 4 years post-transplant.

Number of Participants with Treatment Related Adverse EventsFrom the start of treatment until 30 days after the end of treatment, up to 2 months

Adverse events will be assessed including any local skin reactions like itching and rash

Number of participants with any proven rejection of the graft in OTRsFrom the start of treatment until 30 days after the end of treatment

Number of participants with any biopsy proven acute rejection of the graft after treatment with calcipotriol plus 5-FU compared to test group.

The changes in erythema extent and intensity scores (0-4) of the treated anatomical sitesat one day after the completion of a 6-day treatment

The changes in erythema extent and intensity scores of the treated anatomical sites in test versus control group in post-transplant OTRs. Treated skin will be evaluated for any sign of irritation including erythema, crusting or ulceration using a clinical erythema scale. (No erythema=0, mild erythema=1, sever erythema with minimal scaling=2, sever erythema with significant scaling=3, sever erythema with scaling, crusting, itching and burning=4)

The changes in response to treatment (AKs number) between treated anatomical sitesat one day after treatment and one year after treatment

The changes in response to topical calcipotriol plus 5-FU versus Vaseline plus 5-FU between treated anatomical sites

The changes in SCC prevention (number of SCC) on the untreated anatomical sites (i.e., trunk and lower extremities) of OTRsat one, two and four years post-transplant.

The changes in efficacy of a twice daily 6-day treatment with topical calcipotriol plus 5-FU (test) versus Vaseline plus 5-FU (control) before transplantation in preventing SCC on the untreated anatomical sites (i.e., trunk and lower extremities) of OTRs.

Trial Locations

Locations (2)

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Barnes-Jewish Hospital

🇺🇸

Saint Louis, Missouri, United States

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