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NCT04910685Recrutement en cours

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

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Résumé

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.

Critères d'inclusion

All Participants: -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. Part 1 and PK groups: • Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review • Participant must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab. • Participants must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures. • For participants receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days. Part K: -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review Part S: -Participant has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria. Part 2: -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review

Critères d'exclusion

• Participant has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non-MC lineage (SM-AHN), aggressive SM, mast cell leukemia, or mast cell sarcoma. • Participant has been diagnosed with another myeloproliferative disorder. • Participant has organ damage attributable to SM. • Participant has clinically significant, uncontrolled, cardiovascular disease • Participant has a QT interval corrected using Fridericia's formula (QTcF) \> \> 470 milliseconds (msec) (for females) or \> 450 msec (for males). • Participant has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site. • Time since any cytoreductive therapy including masitinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy \< 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening period. • Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \< 14 days before beginning the screening period. Other protocol-defined criteria apply.

Contacts

Les coordonnées de l'équipe de l'essai s'affichent sur demande.

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Statut
Recrutement en cours
Phase
Phase 2 · Phase 3
Type d'étude
Interventionnelle
Pays
Suisse, France, Allemagne, Italie, Autriche, États-Unis, Argentine, Australie, Belgique, Brésil, Chili, Colombie, Tchéquie, Danemark, Grèce, Irlande, Pays-Bas, Norvège, Pologne, Portugal, Roumanie, Espagne, Suède, Turquie, Royaume-Uni
Promoteur
Blueprint Medicines Corporation(Industrie)
N° de protocole
BLU-263-1201
Pathologies
Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis
Interventions
Médicament

Traitements évalués

  • Elenestinib
  • Placebo

Lieux · 88

  • Basel, SuisseUniversity Hospital BaselRecrute dans ce centre
  • Lucerne, SuisseLuzerner KantonsspitalRecrute dans ce centre
  • Amiens, FranceCHU Amiens-PicardieRecrute dans ce centre
  • Caen, FranceCHU de CaenRecrute dans ce centre
  • Grenoble, FranceCHU GrenobleRecrute dans ce centre
  • Limoges, FranceCHU de LimogesRecrute dans ce centre
  • Nantes, FranceCHU de NantesRecrute dans ce centre
  • Paris, FranceHôpital de la Pitié SalpétrièreRecrute dans ce centre
  • Paris, FranceHôpital Necker - Départementd 'HématologieA dultesRecrute dans ce centre
  • Poitiers, FranceCHU de PoitiersRecrute dans ce centre
  • Toulouse, FranceCHU Toulouse - Hopital LarreyRecrute dans ce centre
  • Aachen, AllemagneUniversitätsklinikum RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und StammzelltransplantationRecrute dans ce centre
  • Berlin, AllemagneCharité - Universitätsmedizin Berlin Institute of AllergologyRecrute dans ce centre
  • Erlangen, AllemagneUniversity Clinic ErlangenRecrute dans ce centre
  • Hamburg, AllemagneUniversity Clinic Hamburg EppendorfRecrute dans ce centre
  • Lübeck, AllemagneUniversitatsklinikum Schleswig-Holstein, Campus LubeckRecrute dans ce centre
  • Mannheim, AllemagneUniversitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät MannheimRecrute dans ce centre
  • Munich, AllemagneLMU KlinikumRecrute dans ce centre
  • München, AllemagneKlinikum rechts der Isar der Technischen Universitaet MuenchenRecrute dans ce centre
  • Milan, ItalieUOC EmatologiaRecrute dans ce centre
  • Florence, ItalieSOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria CareggiRecrute dans ce centre
  • Bologna, ItalieUnita Operativa di Ematologia AOU Policlinico S. Orsola-MalpighiRecrute dans ce centre
  • Catania, ItalieAOU Policlinico G.Rodolico - San MarcoRecrute dans ce centre
  • Milan, ItalieFondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoRecrute dans ce centre
  • Pavia, ItalieS.C. Ematologia Fondazione I.R.C.C.S. Policlinico San MatteoRecrute dans ce centre
  • Salerno, ItalieS.S.D. Immunologia Clinica e Allergologia Azienda Ospedaliera Universitaria San Giovanni di Dio e Ruggi d'AragonaRecrute dans ce centre
  • Verona, ItalieUnità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di VeronaRecrute dans ce centre
  • Vicenza, ItalieAzienda Unita Locale Socia Sanitaria N8 BericaRecrute dans ce centre
  • Linz, AutricheKepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and OncologyRecrute dans ce centre
  • Birmingham, États-UnisUniversity of Alabama at BirminghamRecrute dans ce centre

Au total, dans le monde, les 88 centres listés au registre recrutent.

Statut par centre selon le registre ClinicalTrials.gov (dernière mise à jour publiée : 7 octobre 2026).

Ce statut peut avoir changé et ne dit rien de votre éligibilité : avant toute démarche, vérifiez avec votre oncologue ou le centre.

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