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A Phase 1b Study to Assess the Safety Profile, Pharmacokinetics, and Anti-VEGF Activity of PTC299 in Patients With Advanced Cancer

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Qué se está evaluando

PTC299

+ Docetaxel

Medicamento
Quiénes están siendo reclutados

Neoplasias

A partir de 18 años
+32 Criterios de eligibilidad
Ver todos los criterios de elegibilidad
Cómo está diseñado el estudio

Estudio de Tratamiento

Fase 1
Intervencional
Inicio del estudio: julio de 2008
Ver detalles del protocolo

Resumen

Patrocinador PrincipalPTC Therapeutics
Última actualización: 27 de enero de 2026
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Fecha de inicio: 3 de julio de 2008

Fecha en la que se inscribió al primer participante.

The study will be conducted in 3 stages. In Stage 1 of the study, successive groups of 3 to 6 participants will receive progressively higher PTC299 dose levels; in this stage, treatment will be given in repeated 6-week cycles consisting of 4 weeks of oral PTC299 twice per day followed by a 2-week, no-drug period. In Stage 2, additional groups of 3 to 6 participants will be enrolled at tolerable dose levels to receive treatment in repeated 6-week cycles consisting of oral PTC299 administered 2 or 3 times per day continuously (that is, without the 2-week no-drug period as in Stage 1). In Stage 3, additional groups of 3 to 6 participants will be enrolled at tolerable dose levels to receive treatment in repeated 3-week cycles consisting of oral PTC299 administered 2 or 3 times per day continuously in combination with docetaxel (75 milligrams/meter squared \[mg/m\^2\] intravenously \[IV\] every 3 weeks). All planned dose levels in all stages are expected to achieve circulating blood levels of PTC299 known to be active in animal models of human cancer. Treatment for each participant can continue as long as the therapy appears to be safely offering tumor control to that participant. Up to 76 evaluable participants will be accrued across the 3 stages.

Título OficialA Phase 1b Study to Assess the Safety Profile, Pharmacokinetics, and Anti-VEGF Activity of PTC299 in Patients With Advanced Cancer
NCT00704821
Patrocinador PrincipalPTC Therapeutics
Última actualización: 27 de enero de 2026
Extraido de una base de datos validada por el gobierno.Reclamar como socio

Protocolo

Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.
Detalles del Diseño

Se reclutarán 76 pacientes

Número total de participantes que el ensayo clínico espera reclutar.

Estudio de Tratamiento

Estos estudios prueban nuevas formas de tratar una enfermedad, condición o problema de salud. El objetivo es determinar si un nuevo medicamento, terapia o enfoque funciona mejor o tiene menos efectos secundarios que las opciones existentes.



Elegibilidad

Los investigadores buscan pacientes que cumplan ciertos criterios, conocidos como criterios de elegibilidad: estado general de salud o tratamientos previos.
Condiciones
Criterios

Cualquier sexo

Sexo biológico de los participantes elegibles para inscribirse.

A partir de 18 años

Rango de edades de los participantes que pueden unirse al estudio.

Voluntarios sanos no permitidos

Indica si personas sanas, sin la condición que se estudia, pueden participar.

Condiciones

Patología

Neoplasias

Criterios

16 criterios de inclusión requeridos para participar
Age ≥18 years.

Body weight 40-100 kg.

Capable of swallowing oral medication.

The Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

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16 criterios de exclusión impiden participar
History of severe hypersensitivity reactions to docetaxel or polysorbate 80. Note: This criterion applies to Stage 3 study candidates only.

Presence of malignancy that is refractory to docetaxel (that is, best response with prior docetaxel was progressive disease at first assessment). Note: This criterion applies to Stage 3 study candidates only.

Unstable brain or leptomeningeal disease based on history and physical examination. Note: Enrollment of participants with central nervous system metastases is permitted if such disease is considered stable in the judgment of the investigator. A baseline magnetic resonance imaging (MRI) scan of the brain is required if there is clinical suspicion of central nervous system metastases, hemorrhage, thromboembolism, or increased intracranial pressure.

Any of the following in the past 3 months: myocardial infarction, unstable angina, coronary/peripheral artery bypass graft, congestive heart failure (New York Heart Association Class III or IV), cerebrovascular accident, transient ischemic attack, any other arterial thromboembolic event, or pulmonary embolism.

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Plan de Estudio

Conoce todos los tratamientos administrados en este estudio, su descripción detallada y en qué consisten.
Grupos de Tratamiento
Objetivos del Estudio

4 grupos de intervención están designados en este estudio

0% de probabilidad de ser asignado al grupo placebo

Grupos de Tratamiento

Grupo I

Experimental
In Stage 1, PTC299 will be given orally twice a day (BID) each day at about the same time each day for the first 28 days of each cycle. Each cycle will constitute a 4-week (28 days) period followed by a ≥2-week (14-day) washout period. Participants will be assigned to 0.3 milligrams (mg)/kilograms (kg)/dose BID, 0.6 mg/kg/dose BID, or 1.2 mg/kg/dose BID sequentially based on safety evaluations by the Sponsor's medical monitor in Cycle 1. Treatment cycles can continue if therapy appears to be safely offering tumor control to participant.

Grupo II

Experimental
In Stage 2, PTC299 will be given BID or 3 times a day (TID) orally each day at about the same time each day; therefore, 84 (for BID) or 126 (for TID) doses of PTC299 will be delivered during the 6-week (42-day) period in each cycle. Each participant will be assigned to 100 mg/dose BID, 100 mg/dose TID, 120 mg/dose TID, 160 mg/dose TID, or 200 mg/dose TID sequentially based on safety evaluations by the Sponsor's medical monitor in Cycle 1. Treatment cycles can continue if therapy appears to be safely offering tumor control to participant.

Grupo III

Experimental
In Stage 3, PTC299 will be given BID or TID (for total of 42 \[for BID\] or 63 \[for TID\] doses) orally about same time per day starting on Day 1 in each 3-week (21-day) cycle. Starting dose will be 1 dose level below maximum tolerated dose (MTD) from Stage 2 or 80 mg/dose BID if 100 mg/dose TID in Stage 2 exceeds MTD. Dose escalation will be based on safety evaluations by Sponsor's medical monitor in Cycle 1. Treatment cycles can continue if therapy appears to be safely offering tumor control to participant. Participants will also receive docetaxel as a 1-hour IV infusion on Day 1 per cycle at starting dose of 75 mg/m\^2 with body surface area calculation based on body weight. Dose will be sequentially reduced to 60 and 50 mg/m\^2 in participants with a docetaxel-related dose-limiting toxicity (DLT). Participants will be medicated with oral corticosteroids with docetaxel administration. Recommended dose is 8 mg BID dexamethasone for 3 days starting 1 day prior to docetaxel administration.

Grupo IV

Experimental
In Stage 4, PTC299 will be given orally each day continuously starting on first day of each cycle at about same time per day; so, 42 doses of PTC299 will be delivered for 3 weeks (21-day) in Cycle 1 (BID dosing), and 84 doses will be delivered for 3-weeks (21-day) in Cycle 2 and beyond (TID dosing). For Cycle 1, participants will be assigned to 600, 800, or 1000 mg/dose BID sequentially based on safety evaluations by Sponsor's medical monitor at enrollment. For Cycle 2 and beyond, participants will receive PTC299 160 mg/dose TID. During Cycle 1, Cohort 1 participants will eat a meal containing of ≤15 grams (g) of dietary fat and Cohort 2 participants will eat a meal containing of ≤25 g of dietary fat prior to each dose of PTC299. If ≤2 of 6 participants had experienced DLT during Cycle 1 in either diet plan, then the dose will be escalated to 800 mg BID in Cohort 3 at the optimal diet. Treatment cycles can continue if therapy appears to be safely offering tumor control to participant.

Objetivos del Estudio

Objetivos Primarios

Objetivos Secundarios

Centros del Estudio

Estos son los hospitales, clínicas o centros de investigación donde se lleva a cabo el estudio. Puedes encontrar la ubicación más cercana a ti y su estado de reclutamiento.

Este estudio tiene una ubicación

Suspendido

Memorial Sloan-Kettering Cancer Center

New York, United StatesAbrir Memorial Sloan-Kettering Cancer Center en Google Maps
Suspendido1 Centros de Estudio