A Study to Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
Myelodysplastic Syndromes · Anemia
De qué trata este estudio
El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.
- Tipo de estudio
- De intervención
- Fase
- Fase 2
- Rango de edad
- 18 años - No especificado
- Sexo admitido
- Todos los sexos
- Voluntarios sanos
- No aceptados
- Participantes
- 70 Estimado
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Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.
Acerca de la investigación
The primary objective of this study is to two part. First, dose selection for dose optimization based on safety of ofirnoflast. Second, to evaluate the efficacy of ofirnoflast administered in the study population.
The secondary objectives of this study are to evaluate the extended hematologic response to ofirnoflast, to assess the safety and tolerability of ofirnoflast during dose optimization, and to evaluate hematologic improvement with ofirnoflast treatment.
¿Quién podría participar?
La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.
Inclusion Criteria:
- At least 18 years of age at the time of signing informed consent.
- Capable of giving signed informed consent
- Documented diagnosis of very low-, low-, or intermediate-risk MDS
- Documented diagnosis of anemia
- Documented low serum erythropoietin level
- Relapsed or refractory disease after 1 to 3 prior lines of therapy for lower-risk MDS
- Willing to provide a bone marrow aspirate at Screening.
- Eastern Cooperative Group Performance Status (ECOG PS) of 0, 1, or 2
- Life expectancy of more than 6 months at screening.
- Participants of childbearing potential must have a negative pregnancy test at screening (serum) and Day 1 (urine).
- Participants and partners must use contraception consistent with local regulations and protocol-defined criteria during the intervention period and for at least 30 days after the last dose; periodic abstinence and withdrawal are not acceptable methods.
Exclusion Criteria:
- Anemia due to other causes (e.g., iron deficiency).
- Low platelet count
- Low neutrophil count
- History of hemoglobinopathies, intrinsic RBC membrane/enzyme defects, or hemolytic anemia.
- Prior history of AML, secondary MDS, or other malignancy (except non-melanoma skin cancer or in situ cervical/breast carcinoma) unless disease-free for \>1 year.
- Diagnosis of MPN, CMML, or overlap MDS/MPN per WHO classification.
- Any condition or concomitant treatment that may impair absorption of orally administered study intervention.
- Uncontrolled infection or severe organ dysfunction.
- Concomitant intercurrent illness or condition that, per investigator judgment, would compromise safe participation (e.g., uncontrolled hypertension, uncontrolled seizure, unstable angina, new-onset/exacerbated cardiac arrhythmia).
- Prior treatment with disease-modifying agents (e.g., hypomethylating agents) or immunosuppressive therapy, except prior lenalidomide (permitted).
- Treatment with cytotoxic chemotherapy or experimental agents within 4 weeks prior to first dose.
- History of stem cell, bone marrow, or solid organ transplant.
- Known hypersensitivity to ofirnoflast or its excipients.
- Severe renal or hepatic impairment
- Inability to swallow tablets.
- Participation in another interventional clinical study within 90 days prior to first dose.
- QTcF \>480 ms.
- Prior treatment with ofirnoflast.
- Medications or food products that are strong inhibitors, substrates, and inducers of CYP3A4/5, CYP2C9, P-glycoprotein (P-gp) and/or Breast Cancer Resistance Protein (BCRP) may cause drug-drug interactions and should be avoided.
Tratamiento, grupos y participación
El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.
- Asignación
- Asignación al azar
- Enmascaramiento
- Sin enmascaramiento
Part 1: Low dose ofirnoflast
Participants receive low dose ofirnoflast, up to 6 participants total. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Part 1: Medium-low dose ofirnoflast
Participants receive medium-low dose ofirnoflast, up to 6 participants total. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Part 1: Medium-high dose ofirnoflast
Participants receive medium-high dose ofirnoflast, up to 6 participants total. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Part 1: High dose ofirnoflast Optional
Participants receive high dose ofirnoflast, up to 6 participants total. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Part 2: First Dose ofirnoflast Selected
Twenty participants receive the first selected dose of ofirnoflast, dose to be determined during Part 1 of study. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Part 2: Second Dose ofirnoflast Selected
Twenty participants receive the second dose of ofirnoflast selected, dose to be determined during Part 1 of study. Treatment continues through the 24-Week Initial Treatment Period and, for participants demonstrating clinical benefit, the Extension Period (up to an additional 24 weeks).
Drug: Ofirnoflast
Ofirnoflast DRUG
tablet
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Dose selection for dose optimization
Plazo: Days 1-28 of each Part 1 Cohort
Incidence of dose-limiting toxicities during the 28-day DLT observation window at each dose level to determine the dose levels acceptable for advancement to Part 2.
Evaluate the efficacy of ofirnoflast in to low- to intermediate-risk MDS who are transfusion dependent
Plazo: Weeks 1-24
The efficacy will be evaluated by proportion of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks
Medidas secundarias
Assess safety and tolerability of ofirnoflast across dose levels evaluated
Plazo: Weeks 1-24
The safety and tolerability of ofirnoflast will be evaluated based on the incidence and severity of TEAEs
Evaluate the overall hematologic response to ofirnoflast
Plazo: Weeks 1-24
The hematologic response to ofirnoflast will be evaluated by proportion of participants achieving overall response
Evaluate the duration of response in participants achieving transfusion independence with ofirnoflast
Plazo: Weeks 1-24
The duration of hematologic response to ofirnoflast will be evaluated by the duration of transfusion independence greater than or equal to 16 consecutive weeks
Evaluate the hematologic improvement in participants treated with ofirnoflast
Plazo: Weeks 1-24
The hematologic improvement will be evaluated by assessment of red blood cells
Evaluate the hematologic improvement in participants treated with ofirnoflast
Plazo: Weeks 1-24
The hematologic improvement will be evaluated by assessment of platelets
Evaluate the hematologic improvement in participants treated with ofirnoflast
Plazo: Weeks 1-24
The hematologic improvement will be evaluated by assessment of neutrophils
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (1)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
1 centros
Cleveland ClinicCleveland, Ohio, United StatesEstado desconocido
Equipo y fechas
- Patrocinador principal
- Halia Therapeutics, Inc.
- Inicio del estudio
- 2026-11 Estimado
- Finalización primaria
- 2028-10 Estimado
- Finalización del estudio
- 2028-12 Estimado
- Primera publicación
- 31 jul 2026 Real
- Última actualización
- 10 nov 2026 Real
Contactos oficiales del estudio
Título oficial del estudio
A Phase 2b, Multicenter, Open-Label, Dose-Escalation, and Randomized Dose-Optimization Study of Ofirnoflast (HT-6184) for the Treatment of Myelodysplastic Syndromes in Adults With Very Low- to Intermediate-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
Consulta a Wellness Clinical Research
Tu mensaje va a WCR, incluso si otra organización realiza este estudio. WCR puede orientarte, pero podría no realizar este estudio, tener acceso a su equipo ni poder inscribirte.
Fuente: API v2 de ClinicalTrials.gov. Patrocinadores e investigadores envían la información. La NLM no aprueba la seguridad ni la ciencia de todos los estudios registrados. Aviso del registro.
Consultado: 12 nov 2026 · Última actualización del registro: 10 nov 2026