The purpose of this research study is to determine if the new drug resminostat will be able to delay or prevent worsening of disease in patients with advanced stage mycosis fungoides or Sézary Syndrome that have achieved disease control with previous systemic therapy, recently

• Register: EUCTR
• Last refreshed on: 26 February 2024
• Main ID: EUCTR2016-000807-99-DE
• Date of registration: 18/07/2016
• Prospective Registration: Yes
• Primary sponsor: 4SC AG
• Public title: The purpose of this research study is to determine if the new drug resminostat will be able to delay or prevent worsening of disease in patients with advanced stage mycosis fungoides or Sézary Syndrome that have achieved disease control with previous systemic therapy, recently
• Scientific title: A multicentre, double blind, randomised, placebo controlled, Phase II trial to evaluate Resminostat for maintenance treatment of patients with advanced stage (Stage IIB-IVB) mycosis fungoides (MF) or Sézary Syndrome (SS) that have achieved disease control with systemic therapy – the RESMAIN Study - RESMAIN Study
• Date of first enrolment: 07/12/2016
• Target sample size: 201
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000807-99
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: yes Other trial design description: open label phase to follow the blinded phase, see protocol for details If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, France, Germany, Greece, Italy, Japan, Netherlands, Poland, Spain, Switzerland, United Kingdom

Contacts

• Name: Julia Lion
• Affiliation: ICON Clinical Research Germany GmbH

• Name: Julia Lion
• Affiliation: ICON Clinical Research Germany GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patients with histologically confirmed MF (Stage IIB IVB) or SS in an ongoing CR, PR or SD after at least one prior systemic therapy according to local standards (including but not limited to a-interferon, bexarotene, extracorporeal photopheresis, chemotherapy) or total skin electron beam irradiation,
o the most recent systemic therapy must have been completed as planned or stopped due to unacceptable toxicity 2 12 weeks prior to randomisation, i.e. patients should not be withdrawn from a treatment from which they derive benefit
2.Male or female = 18 years
3.Written informed consent obtained prior to any trial specific procedure
4.Eastern Cooperative Oncology Group (ECOG) status score 0-2
5.Adequate haematological, hepatic and renal function, as demonstrated by:
a)haemoglobin = 9.0 g/dl (International System [SI] of Units: 5.6 mmol/L)
b)absolute neutrophil count = 1,000/mm3
c)platelets = 75 × 109/L
d)alanine aminotransferase and aspartate amino-transferase = 2 times upper limit of normal
e)total bilirubin = 2 mg/dL (SI units: 34.2 µmol/L) (unless known Gilbert syndrome)
f)serum creatinine = 1.5 mg/dL (SI units: 132 µmol/L)
g)prothrombin time International Normalised Ratio = 2.3
6. Women of childbearing potential (not post-menopausal for 1 year and not surgically sterile) and males with partners of childbearing potential must be sexually abstinent (i.e. refraining from heterosexual intercourse) or must use a highly effective contraception (at least one of the following: combined (oral, intravaginal or transdermal) or progestegen-only (oral, injectable or implantable) hormonal contraceptives, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomy of the partner from the time of screening to 30 days (female patients) or 3 months (male patients) after the last dose of trial treatment
7.Adequate recovery from precedent non-haematological toxicities, excluding alopecia, to = National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade 1
8.Able to comply with all the requirements of the protocol

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 100

Exclusion criteria:
1.Patients with PD
2.Known central nervous system involvement
3.History and current cardiovascular complications, including unstable angina pectoris, uncontrolled hypertension, congestive heart failure (New York Heart Association [NYHA] Class III or IV) related to primary cardiac disease, a condition requiring anti arrhythmic therapy, ischemic or severe valvular heart disease, or a myocardial infarction within 6 months prior to randomisation
4.Baseline corrected QT (QTc) interval > 500 milliseconds, [NOTE: QTcF is relevant]
5.History of additional risk factors for Torsade de Pointes (e.g., heart failure, hypokalaemia, family history of Long QT Syndrome)
6.Use of concomitant medications that are known to prolong the QT/QTc interval
7.Concurrent use of any other specific anti tumour therapy including psoralen photo chemotherapy (PUVA), chemotherapy, immunotherapy, hormonal therapy, radiation therapy, or experimental medications
8.Previous or concurrent cancer that is distinct in primary site or histology from CTCL, except curatively treated squamous-cell carcinoma
of the skin stage 0-1 and curatively treated melanoma stage 0-1A with a
low risk of recurrence/metastasis as per assessment of the investigator, cervical carcinoma in situ, treated basal cell carcinoma, superficial bladder tumours (Ta, Tis and T1); any cancer curatively treated > 3 years prior to randomisation will be allowed
9.Current evidence of any uncontrolled clinically significant internal, psychiatric or neurologic disease
10.Altered mental status precluding understanding of the informed consent process and/or completion of the necessary trial procedures
11.Pregnant or breast feeding women
12.History of allergic reactions attributed to compounds of similar chemical or biological composition to the trial drugs
13.Active alcohol and/or drug abuse
14.Any other acute or chronic condition that, in the investigator’s opinion, would limit the patient’s ability to complete or participate in this trial



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

Advanced stage (Stage IIB-IVB) mycosis fungoides (MF) or Sézary Syndrome (SS) that have achieved disease control with systemic therapy
MedDRA version: 22.0 Level: LLT Classification code 10028508 Term: Mycosis fungoides/Sezary syndrome System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Intervention(s)

Product Name: Resminostat
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Resminostat
CAS Number: 864814-88-0
Current Sponsor code: 4SC-201
Other descriptive name: BYK 408740
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: PFS (progression free survival) is defined as the time from date of
randomisation to first date that criteria for progressive disease (PD)
have been met according to the global response score or death due to
any cause in the absence of documented PD.

Main Objective: The primary objective is to determine if maintenance treatment with resminostat increases progression free survival (PFS) compared to placebo in patients with advanced stage (Stage IIB-IVB) MF or SS that have achieved disease control (complete response [CR], partial response [PR] or stable disease [SD]) with previous systemic therapy.

Primary end point(s): Progression free survival (PFS) is defined as the time from date of randomisation to first date that criteria for PD have been met according to the global response score or death due to any cause in the absence of documented PD.

Secondary Objective: Key Secondary Objective
•To determine if maintenance treatment with resminostat increases time to symptom (pruritus) worsening (TTSW) compared to placebo.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: TTSW (pruritus), the key secondary endpoint, is defined as the time from date of randomisation to first date that criteria for symptom (pruritus) worsening have been met.

Secondary end point(s): Key Secondary Endpoint
•TTSW (pruritus)

Secondary Endpoints
•TTP
•TTNT
•PFS2
•PFS3
•ORR (CR, PR)
•DOR
•OS
•HrQoL
oVAS (itching)
oFACT-G
oSkindex-29
•PK analysis (peripheral blood)

Secondary ID(s)

2016-000807-99-GB

4SC-201-6-2015

Source(s) of Monetary Support

4SC AG

Ethics review

Status: Approved
Approval date: 07/12/2016
Contact: