Resumen

Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad del tratamiento con paltusotine frente a placebo, así como la seguridad a largo plazo de paltusotine, en adultos con síndrome carcinoide debido a tumores neuroendocrinos bien diferenciados. El propósito de este estudio es continuar la evaluación de la seguridad, eficacia y farmacocinética (FK) de paltusotine en participantes con síndrome carcinoide.

Descripción detallada

Este es un estudio global, aleatorizado, de grupos paralelos, controlado con placebo para evaluar la eficacia y seguridad de paltusotine en adultos con síndrome carcinoide. El estudio incluye un período de selección de hasta 11 semanas, un período de control aleatorizado doble ciego de 16 semanas, un período de extensión abierta de 104 semanas y un período de seguimiento de 4 semanas.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

Male or female ≥18 years of age, at the time of Screening.
Willing and able to comply with the study procedures as specified in the protocol, including at least 70% compliance with the study diary for the 2-week period.
Documented carcinoid syndrome requiring medical therapy. Participants must exhibit symptoms of flushing with or without frequent BMs as follows:
For participants who are naïve/not currently treated with somatostatin receptors ligands (SRL), they must exhibit an average of \>1 flushing episode/day over a period of 14 days
For participants who will wash out from SRL treatment, they must exhibit an increase in daily average flushing episodes and an average of \>1 flushing episode/day over a period of 14 days during the Washout Period.
Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated neuroendocrine tumor(s) \[NETs\].
No significant disease progression as assessed by the Investigator within the last 6 months before randomization.

Criterios de exclusión

Diarrhea attributed to any condition(s) other than carcinoid syndrome.
Uncontrolled/severe diarrhea associated with significant volume contraction, dehydration, or hypotension.
Requires second line treatments (eg, telotristat) for control of carcinoid syndrome symptoms in the opinion of the Investigator.
Treatment with specific NET therapy \<4 weeks before Screening (such as everolimus or sunitinib) or hepatic embolization, radiotherapy, peptide receptor radionuclide therapy (PRRT), and/or tumor debulking \<12 weeks before Screening.
Major surgery within 8 weeks before Screening.
History of another primary malignancy \<3 years prior to the date of randomization, except for adequately treated basal or squamous cell carcinoma of the skin, cancer of the breast or cervix in situ, previously treated malignancy, if all treatment for that malignancy was completed at least 3 years prior to first dose of study treatment, and no current evidence of disease, concurrent malignancy determined to be clinically stable and not requiring treatment.
Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry.
Poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) ≥8.5%
Unable to administer short-acting (SA) octreotide (octreotide acetate injection), or prior nonresponse documented with somatostatin agonists.
Clinically significant concomitant disease or indicator of disease that is not a result of the primary disease under study, including but not limited to cardiovascular disease, estimated glomerular filtration rate 2×upper limit of normal \[ULN\], and/or total bilirubin (TB) \>1.5×ULN. (Participants with previously diagnosed Gilbert's syndrome not accompanied by other hepatobiliary disorders and associated with TB

Ubicaciones

Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

Buenos Aires, Argentina

RECRUITING

Sanatorio Guemes

Buenos Aires, Argentina

WITHDRAWN

Centro de Endocrinologia y Diabetes Dr. A. Gutman ICM - Investigaciones

Buenos Aires, Argentina

RECRUITING

Instituto Médico Especializado Alexander Fleming

Buenos Aires, Argentina

RECRUITING

Instituto Médico de la Fundación Estudios Clínicos

Santa Fe, Argentina

RECRUITING

Estudio aleatorizado, de grupos paralelos, controlado con placebo para evaluar la eficacia y seguridad de paltusotine en adultos con síndrome carcinoide debido a tumores neuroendocrinos bien diferenciados

Resumen

Descripción detallada

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Paltusotine

Placebo

Ubicaciones

Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

Sanatorio Guemes

Centro de Endocrinologia y Diabetes Dr. A. Gutman ICM - Investigaciones

Instituto Médico Especializado Alexander Fleming

Instituto Médico de la Fundación Estudios Clínicos

Patrocinadores

Contactos

Crinetics Clinical Trials