Clinical trial NCT02546440: Study on Therapy With Dimethylfumarate (DMF) in Patients With Cutaneous T Cell Lymphoma (CTCL)
Official title: Phase IIA Study on Therapy With the NF-κB Inhibiting and Apoptosis Inducing Drug Dimethylfumarate (DMF) in Patients With Cutaneous T Cell Lymphoma (CTCL)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cutaneous T Cell Lymphoma
Lead sponsor: Universitätsmedizin Mannheim
Sponsor class: OTHER
Collaborators: name: Klinikum Minden
name: Klinikum Krefeld
name: Wuerzburg University Hospital
name: KKS Netzwerk
name: Klinikum Ludwigshafen
name: Universitätsklinikum Kiel
First posted: 2015-09-10
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main objective of the trial is to investigate whether oral treatment of patients suffering from cutaneous T cell lymphoma with dimethylfumarate is leading to a significant improvement of modified severity assessment tool (mSWAT) values in the skin after 24 weeks of treatment (primary endpoint). Secondary endpoints are dermatologic life quality index, itching and pain measured by a NRS and the blood involvement if applicable.

Primary: safety and efficacy of DMF treatment in CTCL Secondary: Dermatologic Life Quality index, NRS for itching and pain, blood involvement if appl.


Eligibility criteria: Inclusion criteria:

* Histopathologically confirmed Mycosis fungoides or Sézary syndrome (CTCL stage ≥ Ib according to EORTC-ISCL consensus classification) at study entry with progressive, persistant or recurrent disease
* Pretreatment with at least one topical or systemic CTCL therapy or UV therapy, if the prior therapy is not possible anymore or if there is new contraindication or unsatisfactory response
* Karnofsky index ≥70 % (according to Karnofsky DA, Burchenal JH. (1949). "The Clinical Evaluation of Chemotherapeutic Agents in Cancer." In: MacLeod CM (Ed), Evaluation of Chemotherapeutic Agents. Columbia Univ Press. Page 196)
* Life expectancy \> 3 months
* Age ≥ 18 years
* Adequate organ function:
* differential blood count: hemoglobin ≥ 10 g/dl without transfusions, leukocyte count \> 3000/µl, lymphocyte count \> 700/µl
* liver enzymes ≤ 2 x upper limit of normal (ULN)
* serum creatinine ≤ 1.5 mg/dl or calculate creatinine clearance ≥ 50 ml/min,
* Negative Pregnancy test from blood, agreement for efficient contraception in male and female patients unless infertility is documented (DMF is not approved during pregnancy)
* Ability to understand character and individual consequences of the clinical trial and to provide written informed consent to participate in the study
* written informed consent must be given according to ICH/GCP, and national/local regulations, before patient registration and prior to any study specific procedures.

Exclusion criteria:

* Another active malignant disease with the following exceptions:
* Basal or squamous cell carcinoma of the skin
* In situ carcinoma of the cervix or the skin
* Topical chemotherapy, superficial radiotherapy, photopheresis or systemic CTCL treatment within 28 days before study therapy initiation
* Severe systemic disease or infection at study therapy initiation
* Prior treatment with DMF or simultaneous topical DMF treatment
* Contraindications for treatment with DMF (known hypersensibility to the drug, severe gastrointestinal disease (like ulcerations), Alcohol abuse, other obligately liver- or nephrotoxic medication, known clinically apparent renal or hepatic insufficiency)
* Any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial
* Participation in other clinical studies within 14 days before study therapy initiation
* Pregnant or lactating patients

Primary outcomes:
measure: safety (via occurrence of AE/SAE) of DMF treatment in CTCL
timeFrame: every 2 weeks until 24 weeks of treatment are finished
measure: efficacy (via improvement of Skin involvement measured by the standardized modified severity weighted assessment tool (mSWAT))of DMF treatment in CTCL
timeFrame: every 2 weeks until 24 weeks of treatment are finished
Secondary outcomes:
measure: changes in dermatologic life quality index
timeFrame: every 2 weeks until 24 weeks of treatment are finished
measure: changes in pruritus intensity measured by a visual analog scale
timeFrame: every 2 weeks until 24 weeks of treatment are finished
measure: changes in blood involvement measured by Sezary cell count (if applicable, only in stage IV patients)
timeFrame: every 2 weeks until 24 weeks of treatment are finished
Other outcomes:
Locations:
city: Mannheim
country: Germany
facility: University Medical Center
Study officials:
References: