RVU120 for Treatment of Anemia in Patients With Lower-risk Myelodysplastic Neoplasms

NCT06243458 | Active Not Recruiting Phase 2

• 2 other identifiers: REMARK_001EUCT Number 2023-509947-29
• Study Type: interventional
• Target: 41
• Locations: 5 countries
• Sites: 25

Brief Summary

This study will evaluate orally administered RVU120, a novel small molecule Cyclin-dependent Kinase (CDK) 8/19 inhibitor, in terms of erythroid hematologic improvement (HI-E) and safety in participants with lower-risk myelodysplastic syndrome (MDS). Responding patients are eligible to continue treatment until loss of response/disease progression.

Conditions

Low Risk Myelodysplastic Syndromes

Keywords

CDK8/19 Inhibitor; RVU120 (SEL120); Clinical Trial, Phase II

Outcome Measures

Primary Outcomes (1)

• Erythroid Response (HI-E) Rate of Participants after Treatment with RVU120 (dose of 150 mg)

  The International Working Group (IWG) 2018 Hematologic Improvement Criteria for MDS will be used to define responders.

  Response Assessment measured after 8 full Cycles of RVU120 administration. Each cycle is 21 days.

Study Arms (1)

Single-arm RVU120

EXPERIMENTAL

Drug: RVU120 (SEL120)

Interventions

RVU120 (SEL120) DRUG

RVU120 will be taken orally every other day (q.o.d). in a 21-day treatment cycle. Doses of RVU120 will be administered from day 1 to day 13 (total of 7 doses per cycle).

Single-arm RVU120

Eligibility Criteria

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

You may qualify if:

• Written informed consent provided prior to any study-related procedure
• Age ≥18 years
• Diagnosis of de novo myelodysplastic neoplasms (MDS) according to World Health Organization (WHO) 2022 criteria.
• Diagnosis will be confirmed during screening assessment.
• Very low, low or intermediate risk disease MDS with up to 3.5 points according to International Prognostic Scoring System Score Revised (IPSS-R) classification (to be confirmed during screening assessment).
• Patients with del(5q) and max. one further abnormality (excluding monosomy 7, del(7q), TP53mut) are eligible.
• Patients should be registered only if it is expected at time of registration that
• the Hb Mean over the baseline period will be less than 10 g/dL OR three or more red blood cell (RBC)-transfusions will have been given during the baseline period documenting transfusion dependence.
• No available option of an approved MDS therapy according to decision of the treating physician and based on the following:
• Patients must be
• ESA exposed (and refractory or intolerant) or ESA naïve and serum erythropoietin level \>200 U/L AND/OR
• Luspatercept exposed (and refractory or intolerant) or luspatercept naïve and not eligible for treatment (e.g. not approved) AND/OR
• Lenalidomide exposed (and refractory or intolerant) or lenalidomide naïve and not eligible for treatment (e.g. due to non-presence of del(5q))
• Eastern Cooperative Oncology Group (ECOG) performance score of 0-2
• Patients must have been off anti-cancer treatment for 2 weeks or 5 half-lives, whichever is longer

You may not qualify if:

• Inability to swallow and retain oral medications.
• Patient does not accept bone marrow sampling during screening and after the treatment.
• Prior treatment with azacitidine (injectable or oral) or decitabine.
• The patient medically requires treatment with the following drugs that are forbidden during the trial or was exposed to one of these 14 days before the first dose of the IMP:
• Erythropoiesis stimulating agent (ESA) or luspatercept
• Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony stimulating factor (GM-CSF)
• Lenalidomide
• Another investigational drug or device, or approved therapy for investigational use
• Iron chelation therapy NOTE: if therapy was initiated 56 days or more prior to the first dose of the IMP, patient can be included. Recently initiated iron chelation \[\< 56 days prior to registration\] might influence interpretation of hematological response after start of trial medication.
• Previous treatment with CDK8-targeted therapy(s).
• Active central nervous system (CNS) involvement.
• Patients who have undergone major surgery within 28 days prior to first dose of study drug.
• Evidence of ongoing and uncontrolled systemic bacterial, fungal, or viral infection and acute inflammatory conditions (including pancreatitis)
• Hematopoietic stem cell transplant within 120 days prior to first dose of study drug.
• Active Grade 2-4 acute graft versus host disease (GVHD), active moderate-to-severe chronic GVHD, or requirement for systemic immunosuppressive medications for GVHD.

Sponsors & Collaborators

• GCP-Service International West GmbH: lead
• Universitätsklinikum Leipzig: collaborator
• Saint-Louis Hospital, Paris, France: collaborator
• University of Florence: collaborator
• University of Salamanca: collaborator
• Medical University of Warsaw: collaborator
• Ryvu Therapeutics SA: collaborator
• European Myelodysplastic Neoplasms Cooperative Group: collaborator

Study Sites (25)

CHU Grenoble Alpes - Hôpital Michallon

Grenoble, 38700, France

Location

CHU de Nice - Hôpital l'Archet 1

Nice, 06202, France

Location

Hôpital Saint-Louis, Service d'Hématologie Séniors

Paris, 75010, France

Location

CHU de Bordeaux - Centre François Magendie - Groupe Hospitalier Sud

Pessac, 33604, France

Location

CHU de Toulouse - Institut Universitaire du Cancer de Toulouse - Oncopole

Toulouse, 31059, France

Location

Marien Hospital Düsseldorf

Düsseldorf, 40479, Germany

Location

Universität Leipzig, Med. Fak., Klinik und Poliklinik für Hämatologie, Zelltherapie, Hämostaseologie und Infektiologie

Leipzig, 04103, Germany

Location

Universitätsklinikum Münster (UKM)

Münster, 48149, Germany

Location

AOU delle Marche

Ancona, 60126, Italy

Location

AOU Consorziale Policlinico, Università degli Studi Aldo Moro

Bari, 70124, Italy

Location

Candiolo Cancer Institute, IRCCS Fondazione del Piemonte per l'Oncologia

Candiolo, 10060, Italy

Location

AOU Careggi

Florence, 50134, Italy

Location

IRCCS Humanitas Research Hospital

Milan, Italy

Location

Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

Reggio Calabria, 89133, Italy

Location

Samodzielny Publiczny Szpital Kliniczny im.Andrzeja Mielęckiego Śląskiego Uniwersytetu Medycznego w Katowicach

Katowice, 40-027, Poland

Location

Pratia Hematologia Sp. z o. o. , Pratia Onkologia Katowice

Katowice, 40-519, Poland

Location

Wojewódzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M.Kopernika w Łodzi

Lodz, 93-523, Poland

Location

MTZ Clinical Research powered by Pratia

Warsaw, 02-172, Poland

Location

Uniwersytecki Szpital Kliniczny im.Jana Mikulacza-Radeckiego

Wroclaw, 50-367, Poland

Location

Hematology Department Vall d'Hebron Institute of Oncology (VHIO)

Barcelona, 08035, Spain

Location

Hospital Universitario de León

León, 24008, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Navarra university clinic

Pamplona, 31008, Spain

Location

Complejo Asistencial Universitario de Salamanca

Salamanca, 37007, Spain

Location

Hospital Clínico Universitario de Valencia

Valencia, 46010, Spain

Location

Study Officials

• Uwe Platzbecker, Prof.

  Universitätsklinikum Leipzig

  PRINCIPAL INVESTIGATOR

• Lionel Adès, Prof.

  Service d'hématologie seniors Hôpital Saint-Louis, Paris

  PRINCIPAL INVESTIGATOR

• Valeria Santini, Prof.

  University of Careggi, Italy

  PRINCIPAL INVESTIGATOR

• Krzysztof Mądry, Dr.

  Medical University of Warsaw

  PRINCIPAL INVESTIGATOR

• Diez Campelo, Dr.

  University of Salamanca

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2024
• First Posted: February 6, 2024
• Study Start: September 10, 2024
• Primary Completion: May 1, 2026
• Study Completion (Estimated): May 1, 2027
• Last Updated: May 22, 2025

Record last verified: 2025-05

Locations

ES (6); IT (6); PL (5); DE (3); FR (5)