Therapeutic Efficacy of Topical Sirolimus in Early Stage Cutaneous T-cell Lymphoma (CTCL)

NCT01843998 | Withdrawn Phase 2 DMC

• 1 other identifier: PRO#00019812
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether sirolimus reduces the symptoms of cutaneous T-cell lymphoma (CTCL) and whether it causes any side effects.

Conditions

Cutaneous T-cell Lymphoma (CTCL)

Keywords

Early stage; Stage 1A; Cutaneous T-cell Lymphoma; CTCL

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate

  Determine the efficacy of topical sirolimus in the treatment of early stage CTCL as overall response rate (ORR) Response criteria: Complete response (CR) will be defined as no evidence of clinical skin disease. Partial response (PR) will be defined as a marked improvement in skin disease of \> 50% from baseline without new lesions. Stable disease (SD) will be defined as \< 25% increase and \< 50% clearance in skin disease from baseline without new lesions. Progressive disease (PD) will be defined as \> 25% increase in skin disease from baseline or new tumors or loss of response in those with CR or PR (increase in skin score of greater than the sum of nadir plus 50% baseline score). The assessment will be based on Composite Assessment of Index Lesion Severity (CAILS) and, in case of more extensive disease, Modified Severity-Weighed Assessment Tool (mSWAT) scores.

  6 months

Secondary Outcomes (3)

• Duration of objective response (DOR)

  6 months

• Adverse event profile and tolerability of topical sirolimus in patients with CTCL

  6 months

• Correlative biomarkers such as mTOR pathway activation at baseline and during treatment

  6 months

Study Arms (1)

Sirolimus 0.1% ointment

EXPERIMENTAL

Sirolimus 0.1% ointment

Drug: Sirolimus 0.1% Ointment

Interventions

Sirolimus 0.1% Ointment DRUG

Sirolimus 0.1% ointment will be applied topically to all affected areas of the skin twice daily for 6 months or until progression or unacceptable toxicity.

Also known as: Rapamune, sirolimus, rapamycin

Sirolimus 0.1% ointment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinically and histologically confirmed diagnosis of CTCL (early stage disease with patches and/or thin plaques covering up to 10%, stage IA)
• Relapsed or refractory disease after at least one standard skin directed treatment including corticosteroids, topical bexarotene, phototherapy
• All subjects must be 18 years of age or older
• Life expectancy ≥ 6 months, determined by the treating physician
• Signed informed consent

You may not qualify if:

• Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of study drug (including topical or systemic glucocorticosteroids, chemotherapy, radiation therapy, antibody based therapy, etc.)
• Prior treatment with any investigational drug within the preceding 4 weeks
• Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent.
• Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. Adequate contraception (oral contraceptives ("the pill"), intrauterine devices (IUDs), contraceptive implants under the skin, or contraceptive injections, diaphragms with spermicide and condoms with foam) must be used throughout the trial and for 8 weeks after the last dose of study drug (women of childbearing potential must have a negative urine within 7 days prior to administration of sirolimus).
• Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus, temsirolimus, everolimus).
• Patients with a known hypersensitivity to sirolimus or other rapamycin (e.g., everolimus, temsirolimus) or to its excipient
• History of noncompliance to medical regimens
• Patients unwilling to or unable to comply with the protocol

Sponsors & Collaborators

• Stefan Schieke MD: lead

Study Sites (1)

Froedtert Hospital and the Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Lymphoma, T-Cell, Cutaneous

Interventions

Sirolimus; Ointments

Condition Hierarchy (Ancestors)

Lymphoma, T-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Dosage Forms; Pharmaceutical Preparations

Study Officials

• Stefan Schieke, MD

  Medical College of Wisconsin

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: April 24, 2013
• First Posted: May 1, 2013
• Study Start: February 1, 2014
• Primary Completion: May 1, 2015
• Study Completion: December 1, 2015
• Last Updated: June 4, 2015

Record last verified: 2015-06

Locations

US (1)