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El ensayo clínico NCT07491172 para Cardiovascular, Enfermedad Metabólica, Dislipidemias, Trastorno de lípidos, Hipertrigliceridemia, Hipercolesterolemia Familiar Heterocigota (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hipercolesterolemia está reclutando. Consulte la vista de tarjeta del Radar de Ensayos Clínicos y las herramientas de descubrimiento de IA para conocer todos los detalles. O haga cualquier pregunta aquí.
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A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias Fase I 90 Etiqueta abierta

Reclutando
Los detalles del ensayo clínico están disponibles principalmente en inglés. ¡Sin embargo, IA Trial Radar puede ayudar! Simplemente haga clic en 'Explicar el estudio' para ver y discutir la información del estudio en el idioma que haya seleccionado.
El ensayo clínico NCT07491172 está diseñado para estudiar el tratamiento de Cardiovascular, Enfermedad Metabólica, Dislipidemias, Trastorno de lípidos, Hipertrigliceridemia, Hipercolesterolemia Familiar Heterocigota (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hipercolesterolemia. Es un estudio intervencionista de Fase I. Su estado actual es: reclutando. El estudio se inició el 21 de junio de 2024, con el objetivo de reclutar a 90 participantes. Dirigido por CRISPR Therapeutics, se espera que finalice el 1 de junio de 2028. Los datos se actualizaron por última vez en ClinicalTrials.gov el 24 de marzo de 2026.
Resumen
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
Descripción detallada
This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.
Título oficial

A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias

Condiciones médicas
CardiovascularEnfermedad MetabólicaDislipidemiasTrastorno de lípidosHipertrigliceridemiaHipercolesterolemia Familiar Heterocigota (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Severe Hypertriglyceridemia (sHTG)Mixed HyperlipemiaHipercolesterolemia
Otros ID del estudio
  • CRSP-CVD-400
Número del NCT
NCT07491172
Inicio del estudio (real)
2024-06-21
Última actualización
2026-03-24
Fecha de finalización (estimada)
2028-06
Inscripción (prevista)
90
Tipo de estudio
Intervencionista
FASE
Fase I
Estado general
Reclutando
Palabras clave
Refractory Dyslipidemias
Objetivo principal
Tratamiento
Método de asignación
N/A
Modelo de intervención
Grupo único
Enmascaramiento
Ninguno (Abierto)
Brazos / Intervenciones
Grupo de participantesIntervención/Tratamiento
ExperimentalCTX310
Subjects will receive an intravenous (IV) infusion.
CTX310
CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).
Resultado primario
Medida de resultadoDescripción de la medidaPeriodo de tiempo
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
Incidence of dose-limiting toxicities and frequency of adverse events
From CTX310 infusion up to 12 months
Resultado secundario
Medida de resultadoDescripción de la medidaPeriodo de tiempo
To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline in lipid values (LDL-C, non-HDL-C, Trigs, apoB and HDL-C)
Over 12 months, compared to baseline
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Frequency and severity of adverse events (AE), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs.
From CTX310 infusion up to 12 months
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Levels of CTX310 in blood over time
From CTX310 infusion up to 12 months
To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline of ANGPTL3
Over 12 months, compared to baseline
Asistente de participación
Criterios de elegibilidad

Criterios de edad
Adulto, Adulto mayor
Edad mínima
18 Years
Criterios de sexo
Todos
  1. Age of ≥18 and ≤75 years at the time of signing the informed consent.
  2. Able to provide written informed consent.
  3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL.
  4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening
  5. Female participants must be postmenopausal or surgically sterile.
  6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study.

  1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.

  2. Evidence of liver disease, defined as but not limited to:

    LFTS >2 × upper limit of normal (ULN), or total bilirubin >2 × ULN, or INR >1.5 × ULN, or liver stiffness measured by liver elastography

  3. Abnormal or compromised function of kidney, heart, blood or liver.

  4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.

  5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).

  6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.

  7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast).

  8. Women of childbearing potential.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.

CRISPR Therapeutics AG logoCRISPR Therapeutics6 estudios activos para explorar
Contactos centrales del estudio
Contacto: Clinical Trials, 877-214-4634, [email protected]
18 Centros del estudio en 4 países

Florida

Research Site 10, Jacksonville, Florida, 32216, United States
Aún no recluta
Research Site 17, Orlando, Florida, 32789, United States
Aún no recluta
Research Site 11, Port Orange, Florida, 32127, United States
Aún no recluta

North Carolina

Research Site 14, Durham, North Carolina, 27710, United States
Aún no recluta
Research Site 13, High Point, North Carolina, 27260, United States
Aún no recluta

Ohio

Research Site 9, Cleveland, Ohio, 44195, United States
Aún no recluta

Oregon

Research Site 12, Portland, Oregon, 97239, United States
Aún no recluta
Research Site 1, Adelaide, 5000, Australia
Reclutando
Research Site 15, Brisbane, 4064, Australia
Aún no recluta
Research Site 16, Brisbane, Australia
Aún no recluta
Research Site 7, Camperdown, 2050, Australia
Aún no recluta
Research Site 3, Heidelberg, 3084, Australia
Reclutando
Research Site 2, Melbourne, 3168, Australia
Reclutando
Research Site 5, Auckland, 2025, New Zealand
Reclutando
Research Site 4, Christchurch, 8011, New Zealand
Reclutando
Research Site 18, Cambridge, United Kingdom
Aún no recluta
Research Site 8, London, E1 1BB, United Kingdom
Aún no recluta
Research Site 6, London, SE1 1YR, United Kingdom
Reclutando