Efficacy and Safety Study of Paltusotine for Adults with Carcinoid Syndrome due to Well-Differentiated Neuroendocrine Tumors

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What is this study about?

This study investigates the effects of paltusotine compared to placebo in adults with carcinoid syndrome, a condition that occurs in some people with well-differentiated neuroendocrine tumors (NETs). Carcinoid syndrome is characterized by symptoms like flushing (sudden reddening of the skin, particularly the face) and frequent bowel movements, which occur when these tumors release certain substances into the bloodstream.

The purpose of this study is to evaluate whether paltusotine, an oral medication taken daily, can reduce the number of flushing episodes experienced by patients with carcinoid syndrome. The study will also assess whether the medication affects the frequency of bowel movements and examine its overall safety.

Participants in this study will either receive paltusotine or a placebo for 12 weeks. During this time, they will need to keep a daily record of their symptoms. Some participants who are currently taking other medications called somatostatin receptor ligands (SRLs) will need to stop those treatments before starting the study medication. Blood tests and imaging studies like CT scans or MRI may be performed to monitor the participants’ condition throughout the study.

1 Screening Period

You will enter a screening period to determine if you qualify for the study. During this time, you’ll need to complete a Carcinoid Syndrome Symptom Diary for 2 weeks, recording your daily flushing episodes and bowel movements.

If you’re currently taking somatostatin receptor ligands (SRLs), you’ll need to stop this medication and go through a washout period. During the washout, you’ll need to show an increase in flushing episodes with an average of more than 1 flushing episode per day over 14 days.

A CT or MRI scan will be performed to assess your disease status.

2 Randomization

After qualifying, you’ll be randomly assigned to one of two groups: either the paltusotine group or the placebo group.

Neither you nor your doctor will know which group you’re in during the study.

3 Treatment Period

The treatment period lasts for 12 weeks.

If you’re in the paltusotine group, you’ll take paltusotine 40 mg tablets daily.

If you’re in the placebo group, you’ll take placebo tablets that look identical to the paltusotine tablets.

You’ll need to continue completing your Carcinoid Syndrome Symptom Diary throughout the 12-week treatment period, recording your flushing episodes and bowel movements daily.

4 Study Visits

You’ll attend regular study visits where healthcare providers will monitor your health and assess how you’re responding to treatment.

These visits will include physical examinations, vital sign measurements, and possibly blood tests to evaluate your condition.

The healthcare team will review your symptom diary and ask about any side effects you may be experiencing.

5 Final Assessment

At the end of the 12-week treatment period, a final assessment will be conducted.

The main focus will be comparing your average number of flushing episodes per day at the end of the study versus when you started.

A secondary focus will be comparing your average number of bowel movements per day at the end of the study versus when you started.

6 Follow-up Period

After completing the treatment period, you’ll enter a follow-up period for at least 2 weeks after your last dose.

During this time, you’ll need to continue using contraception if applicable (as detailed in the study requirements).

Male participants should continue to use condoms when sexually active with female partners of childbearing potential for at least 2 weeks after the last dose.

Who Can Join the Study?

You must be willing and able to provide written informed consent before any study procedures.
You must be willing to comply with the study procedures, including completing at least 70% of the Carcinoid Syndrome Symptom Diary for specific periods.
You must be 18 years of age or older at screening.
You must have documented carcinoid syndrome (a condition causing symptoms like flushing and frequent bowel movements) requiring medical therapy with either:
- If not currently on somatostatin receptor ligands (SRLs): an average of more than 1 flushing episode per day over 14 days and elevated blood markers (5-HIAA or serotonin)
- If stopping SRL treatment: an increase in flushing episodes to more than 1 per day over 14 days during the washout period
You must have documented advanced or metastatic (spread) well-differentiated neuroendocrine tumors (NETs) that are Grade 1 or Grade 2 (low to intermediate growing tumors). Grade 3 tumors are not eligible.
You must not have had significant disease progression in the last 6 months before randomization, as assessed by your doctor.
You must have documentation showing positive somatostatin receptor tumor status by PET scan or somatostatin receptor scintigraphy (special imaging tests).
If you’re currently treated with octreotide/lanreotide (medications for NETs) and will stop for the study, you must have previously shown elevated levels of certain markers (5-HIAA or serotonin).
For females who have heterosexual intercourse: You must either be unable to have children (surgically sterile or postmenopausal), or use highly effective contraception from screening until at least 2 weeks after the last dose.
For males: You must agree to use a condom when sexually active with female partners who could become pregnant, or be surgically sterile, or remain abstinent, from screening until at least 2 weeks after the last dose. You also cannot donate sperm during this time.

Who Cannot Join the Study?

You have a history of unusual heart valve damage (heart valve fibrosis).
You currently have uncontrolled high blood pressure (hypertension) or a significant heart problem.
You have liver problems that affect how well your liver functions.
You have severe kidney disease or are on dialysis.
You are currently taking certain medications called glucocorticoids (a type of steroid medication).
You have had a major surgery within 2 months before starting the study.
You are pregnant or breastfeeding, or planning to become pregnant during the study.
You have been diagnosed with cancer within the last 5 years (except for certain skin cancers).
You have participated in another clinical trial within 30 days before this study.
You have a known allergy to the study medication or similar drugs.
You are taking other medications that might interfere with the study drug.

Where you can join this trial?

Verified and Recommended Sites

Site Name	City	Country	Status
Wojskowy Instytut Medyczny Państwowy Instytut Badawczy	Warsaw	Poland

Verified Sites

Site Name	City	Country
IRCCS Humanitas Research Hospital	Rozzano	Italy
Oncopole Claudius Regaud	Toulouse	France
Technische Universitaet Dresden	Dresden	Germany
Katholieke Universiteit te Leuven	Leuven	Belgium
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Futuremeds Sp. z o.o.	Wroclaw	Poland

Other Sites

Site Name	City	Country
Centre Antoine Lacassagne	Nice	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Istituto Europeo Di Oncologia S.r.l.	Milan	Italy
Azienda Ospedaliero Universitaria Di Modena	Modena	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hopital Beaujon	Clichy	France
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
Uniwersyteckie Centrum Stomatologii I Medycyny Specjalistycznej Sp. z o.o.	Poznan	Poland
Azienda Universitaria Ospedaliera Consorziale Policlinico Bari	Bari	Italy
Hniwefdb Vhgc djvhmukh	Barcelona	Spain
Vnswhy dmp Rkqqw Umpsmwqzdk Hgtdqjni	Sevilla	Spain
Avvmdobdbc Pdxqcrmc Hjqxshpp Du Mtjrbvkau	Marseille	France
Psvxofamlhbtrafafbgdv Mjwcgvw	Marburg	Germany
Cqwfvo Hxfmpilcave Rwoebidn Ufakadspzqhdh Ds Tjnft	Tours	France
Hpweiazt Uosohdnwpuwtd Fimxbclth Jwukhsj Dvbn	Madrid	Spain
Avdyskd Oxywaesfxqz Uwegtuuejjxmw Cxoyhlezjeki Dldtu Sfvmck E Dhjce Sfmpnqp Dg Tmbsbi	Turin	Italy
Uwvuupfrasyvcitxuvmxa Hrefadplak Aqa	Heidelberg	Germany
Ushpjcwckyzsnkqypwhhc Wjdzllehf Alt	Wuerzburg	Germany
Abkmfiz Uglrb Saoxjroan Lkdref Do Boqdwvi	Bologna	Italy
Si Voxyydjyurzwmxc Uwhyfuhvzv Hrmaysar	Dublin	Ireland
Uilcnpmkrq Or Aivcyfr	Edegem	Belgium
Ubhocfidms Mfyyhqy Cermab Hhqkzljglslcelxtg	Hamburg	Germany
Ukxizfcthsdjtdffyjxcs Eiczj Ady	Essen	Germany
Ueyuxqdrstcb Znymupdrrq Gtkv	Gent	Belgium
Cfrkxz Hyxjuhcwxzq Lksm Svm	Pierre Benite	France
Hocwniaj Usnbayvqtewpv 1d Dt Obzckhf	Madrid	Spain
Hmiafuhp Ueeaxftdfozla Vnumdr Dy Ld Vxsmswad	Malaga	Spain
Uhygvceggalovi Cxpiujm Kavmdqblx Idh Peviu Kp Ghuafmvzsap Suvdilruj Uevkqferienk Mitgemgmck W Kaclspcuvj	Katowice	Poland
Cqregt Dg Lnpxr Czauuq Ls Chjbhq Exhdvk Mxsnrzy	Rennes	France
Agyjxzj Oulbtpuhxvvhehnaukglognxs Snh Lzpub Gqyochs	Orbassano	Italy
Gmjtpu Hjujlgasxtn Drtqytssqoi Cyext Sdpoy Swlig	Paris	France
Arexblf Oykhoogdgivdezeixltgclcyg Scsr Ajezf	Rome	Italy
Cmkbcz Rkmiqeoe Ctvmzkyn Dg Vllxmz Sdfqni	Verona	Italy
Zsbxrmkzruvxr Bix Bvfsn Gcmk	Bad Berka	Germany
Avjeghxty Kdljlw Sr Gweijh	Hamburg	Germany
Infrjogduc Obpmovcjw Pxach Dbg Affkpcnyd Tbdpmcvlzeyd Bsvwqktxc	Bucharest	Romania
Cdbnqrg Dyuatxfnoltbwrwwkdqfevo	Warsaw	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	01.08.2025
Denmark	Not yet recruiting	01.08.2025
France	Recruiting	01.08.2025
Germany	Recruiting	01.08.2025
Ireland	Not yet recruiting	01.08.2025
Italy	Recruiting	01.08.2025
Poland	Recruiting	01.08.2025
Romania	Recruiting	01.08.2025
Spain	Recruiting	01.08.2025
Sweden	Not yet recruiting	01.08.2025

Trial locations

Investigated Drugs:

Paltusotine

Paltusotine is a medication being studied for the treatment of carcinoid syndrome in adults who have a specific type of tumor called well-differentiated neuroendocrine tumors. It aims to reduce symptoms of carcinoid syndrome, particularly flushing episodes (sudden reddening of the skin, typically on the face and upper body).

Placebo is an inactive substance that looks like the real medicine but contains no active ingredient. It’s used as a comparison to determine if the active medication (paltusotine) is effective.

Carcinoid syndrome – A collection of symptoms that occurs when a carcinoid tumor releases specific chemicals into the bloodstream. These symptoms typically include flushing of the face and upper chest, diarrhea, and frequent bowel movements. Patients may also experience abdominal cramping, wheezing, and heart valve problems. The syndrome occurs in about 10% of people with carcinoid tumors, particularly when the tumor has spread to the liver. The severity and frequency of symptoms can vary widely between individuals, with some experiencing mild occasional flushing while others have more severe manifestations.

Trial ID:

2024-519875-24-00

Protocol code:

CRN00808-12

Trial Phase:

Therapeutic confirmatory (Phase III)

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Efficacy and Safety Study of Paltusotine for Adults with Carcinoid Syndrome due to Well-Differentiated Neuroendocrine Tumors

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?