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Cutaneous T-cell Lymphoma (CTCL)Phase 2NCT01843998

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

A Phase 2 study testing Sirolimus 0.1% Ointment in people with Cutaneous T-cell Lymphoma (CTCL), run by Stefan Schieke MD at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cutaneous T-cell Lymphoma (CTCL)
What is being tested?
Sirolimus 0.1% Ointment
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
Feb 2014to Dec 2015
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

  • 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

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Froedtert Hospital and the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

Other trials in United States

About this study

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.

Read the detailed description

This will be a prospective, non-randomized, open label study of topical sirolimus for the treatment of CTCL recurrent or refractory to at least one previous skin directed treatment. The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin examinations.

Study duration:

For subjects with at least partial remission, treatment will be continued for a maximum of 6 months. All subjects will be followed for 6 months from the time of discontinuation of the study drug or until progression of disease or until a new treatment for CTCL will be started.

Being tested
Sirolimus 0.1% Ointment
Keywords
Early stage, Stage 1A, Cutaneous T-cell Lymphoma, CTCL
Medical subject headings
Lymphoma, T-Cell, Cutaneous

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sirolimus 0.1% OintmentDrug

    Also called Rapamune, sirolimus, rapamycin

    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.

Study groups in NCT01843998
Study groupRoleWhat they receive
Sirolimus 0.1% ointmentExperimentalSirolimus 0.1% ointment
Receives: Drug: Sirolimus 0.1% Ointment

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 6 months

Secondary outcomes (3)

  1. Duration of objective response (DOR)

    Duration of objective response (DOR) - time from the first date of response until progression of disease or death due to the underlying lymphoma.

    Measured: 6 months

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

    The safety and tolerability of the study treatment regimen will be evaluated. Analyses will be descriptive and no formal hypothesis testing will be performed.

    Safety endpoints will include treatment related adverse events. All reported adverse events will be graded using version 4.0 of the Common Terminology Criteria for Adverse Events (CTCAE v4.0).

    All adverse events recorded during the study will be summarized. The incidence of adverse events will be summarized by body system, severity (based on CTCAE grades), type of adverse event, and relation to the study drug. Deaths reportable as SAEs and non-fatal serious adverse events will be listed by patient and type of adverse event. Adverse events will be summarized by presenting the number and percentage of patients having any adverse event in each body system and having each individual adverse event.

    Measured: 6 months

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

    • Activation level of the mammalian target of rapamycin (mTOR) pathway, quantification of regulatory T cells (Tregs), cytokine profile in tissue samples. This part of the study will characterize the molecular events and mechanisms involved in the effect of sirolimus on CTCL. These studies will be performed in tissues samples from skin biopsies of patients upon study entry and during the study.
    1. mTOR pathway activity in skin: Assessment of phosphorylation status and expression level of downstream targets of mTOR with direct effects on cellular proliferation and growth. Those may include:
    • phospho-S6
    • phospho-4-EBP1
    • cyclin D1 levels
    1. Number of skin resident Tregs: Quantification of regulatory T cells characterized by surface marker expression pattern (CD4+, CD25+, FOXP3+) in tissue samples by immunohistochemistry and/or PCR/flow cytometry.
    2. Cytokine profile in skin:

    Characterization of cytokine profile in lesional skin will be done by cytokine PCR arrays.

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 May 2013 (estimated)
  2. Study startFeb 2014
  3. Primary completionMay 2015 (estimated)
  4. Study completionDec 2015 (estimated)

Resources

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
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01843998
Sponsor's protocol ID
PRO#00019812
Status last verified
Jun 2015
First submitted
24 Apr 2013
Last updated
4 Jun 2015
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sirolimus 0.1% Ointment in people with Cutaneous T-cell Lymphoma (CTCL), run by Stefan Schieke MD at 1 site in United States.

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 5 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: withdrawn, as of the sponsor's last update on 4 Jun 2015. 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 United States. 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?

The study was withdrawn before enrolling participants, so there are no results.

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?

Overall Response Rate (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 NCT01843998.

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 4 Jun 2015.

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