Carcinoid SyndromeCarcinoidCarcinoid TumorCarcinoid Tumor of IleumCarcinoid Tumor of CecumCarcinoid Tumor of LiverCarcinoid Tumor of PancreasCarcinoid Syndrome DiarrheaCarcinoid Intestine Tumor
Investigational drug(s) / intervention(s)
PaltusotinePlacebo
Paltusotine: Experimental Drug: Randomized
Placebo: Matching Placebo Drug: Randomized
Study summary
A Phase 3, randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of paltusotine treatment vs placebo as well as the long-term safety of paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The purpose of this study is to continue the evaluation of the safety, efficacy, and pharmacokinetics (PK) of paltusotine in participants with carcinoid syndrome.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* 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 be symptomatically controlled and 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.
Exclusion Criteria:
* 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 has been completely locally excised and carcinoma in situ of the cervix has also been resected, 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
* Alcohol or drug abuse is not permitted at any time during the study
* Diagnosed with symptomatic cholelithiasis
Primary outcome measure(s)
Participants will record the number of flushing per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing flushing episodes. — Measured at Week 12 Treatment group difference of change from baseline to Week 12 in flushing episodes/day averaged over the 14 days prior to Week 12.
Trial sites (73)
Facility
City
Region
Status
Banner MD Anderson Cancer Center
Gilbert
Arizona
Recruiting
Hoag Memorial Hospital Presbyterian
Newport Beach
California
Recruiting
Yale University - New Haven Hospital - Yale Cancer Center
New Haven
Connecticut
Recruiting
University of Miami
Miami
Florida
Recruiting
Moffitt Cancer Center
Tampa
Florida
Recruiting
Winshop Cancer Institute - Emory University
Atlanta
Georgia
Recruiting
University of Iowa Health Care
Iowa City
Iowa
Recruiting
University of Kentucky Medical Center
Lexington
Kentucky
Recruiting
Louisiana State University Health Sciences
Metairie
Louisiana
Recruiting
Henry Ford Cancer - Detroit
Detroit
Michigan
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Icahn School of Medicine at Mount Sinai
New York
New York
Recruiting
University Hospitals Cleveland Medical Center
Cleveland
Ohio
Recruiting
Hospital of the University of Pennsylvania
Philadelphia
Pennsylvania
Recruiting
Huntsman Cancer Institute, University of Utah
Salt Lake City
Utah
Recruiting
University of Virginia Comprehensive Cancer Center
Charlottesville
Virginia
Recruiting
Medical College of Wisconcin
Milwaukee
Wisconsin
Recruiting
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
Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo
CABA
Argentina
Recruiting
Instituto Médico de la Fundación Estudios Clínicos
Santa Fe
Argentina
Recruiting
Universitair Ziekenhuis (UZ) Leuven
Leuven
Belgium
Recruiting
Fundacao PIO XII - Hospital de Amor Barretos
Barretos
Brazil
Recruiting
Sociedade Literaria e Caritativa Santos Agostinho - Hospital Sao José
Criciúma
Brazil
Recruiting
Nucleo de Pesquisa e Desenvolvimento de Medicamentos (MPDM)
Fortaleza
Brazil
Recruiting
Associacao Hospitalar Moinhos de Vento
Porto Alegre
Brazil
Recruiting
Instituto Nacional de Cancer (INCA)
Rio de Janeiro
Brazil
Recruiting
AC Camargo Cancer Center
São Paulo
Brazil
Recruiting
Clinical Universidad Catolica del Maule, Ltda
Maule
Chile
Recruiting
Centro de Oncologia de Precision
Santiago
Chile
Recruiting
CECIM-Centro de Estudios Cliicos e Investigaciones Medicas
Santiago
Chile
Recruiting
Hospital Universitario San Ignacio
Bogotá
DC
Recruiting
Instituto Nacional de Cancerologia
Bogotá
DC
Recruiting
CHRU Tours - Hopital Trousseau
Chambray-lès-Tours
France
Recruiting
Hopital Beaujun - APHP
Clichy
France
Recruiting
APHM- Hopital de la Timone
Marseille
France
Recruiting
Centre Hospitalier Universitaire Nantes
Nantes
France
Recruiting
Centre Antoine Lacassagne
Nice
France
Recruiting
+ 33 more sites — see the full list on the official registry below.
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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