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Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen
EU CTIS ID: 2024-519875-24-00
What this study is testing
To assess the efficacy of paltusotine vs placebo in reducing flushing episodes
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Willing and able to provide written informed consent prior to any study-related procedures.
- 10. Male participants must agree to use a condom when sexually active with a female partner of childbearing potential from Screening until at least 2 weeks after the last dose of study drug (or be surgically sterile [ie, vasectomy with a confirmed absence of sperm in ejaculate] or agree to remain abstinent on a long-term and persistent basis). Male participants should also agree to not donate sperm for the duration of the study and until at least 2 weeks after the last dose of study drug.
- 2. Willing and able to comply with the study procedures as specified in the protocol, including at least 70% compliance with the Carcinoid Syndrome Symptom Diary for the 2-week period prior to the S2 Visit and prior to the Day 1 Visit.
- 3. Male or female ≥18 years of age, at the time of Screening.
- 4. 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 SRLs, they must exhibit an average of >1 flushing episode/day over a period of 14 days and have a screening plasma 5-HIAA or serotonin result ≥2×ULN. 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.
- 5. Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated NET(s). Tumors must be Grade 1 or Grade 2 with Ki-67 index ≤10% per the World Health Organization neuroendocrine neoplasm classification. Participants with Ki-67 index >10% are not eligible.
You likely can't join if
- 1. Diarrhea attributed to any condition(s) other than carcinoid syndrome.
- 10. Unable to administer SA octreotide (octreotide acetate injection), or prior nonresponse documented with somatostatin agonists.
- 11. Known allergy or hypersensitivity to any of the test materials or related compounds.
- 12. Any clinically significant and active infection such as those requiring systemic treatment within 14 days before randomization.
- 13. 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 <30 mL/min/1.73 m2, cirrhosis, baseline AST and/or ALT >2×ULN, and/or TB >1.5×ULN. (Participants with previously diagnosed Gilbert’s syndrome not accompanied by other hepatobiliary disorders and associated with TB <3.5 mg/dL [<51.3 µmol/L] will be permitted.)
- 14. Current alcohol or drug abuse, or within the last year, is not permitted.
See the full eligibility criteria
Who can join
- 1. Willing and able to provide written informed consent prior to any study-related procedures.
- 10. Male participants must agree to use a condom when sexually active with a female partner of childbearing potential from Screening until at least 2 weeks after the last dose of study drug (or be surgically sterile [ie, vasectomy with a confirmed absence of sperm in ejaculate] or agree to remain abstinent on a long-term and persistent basis). Male participants should also agree to not donate sperm for the duration of the study and until at least 2 weeks after the last dose of study drug.
- 2. Willing and able to comply with the study procedures as specified in the protocol, including at least 70% compliance with the Carcinoid Syndrome Symptom Diary for the 2-week period prior to the S2 Visit and prior to the Day 1 Visit.
- 3. Male or female ≥18 years of age, at the time of Screening.
- 4. 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 SRLs, they must exhibit an average of >1 flushing episode/day over a period of 14 days and have a screening plasma 5-HIAA or serotonin result ≥2×ULN. 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.
- 5. Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated NET(s). Tumors must be Grade 1 or Grade 2 with Ki-67 index ≤10% per the World Health Organization neuroendocrine neoplasm classification. Participants with Ki-67 index >10% are not eligible.
- 6. No significant disease progression as assessed by the Investigator within the last 6 months before randomization into the RCP. Note: A CT or MRI scan will be performed during the Screening Period and should be compared with previous pretrial imaging to assess disease stability for established participants on SRL maintenance therapy, while clinical symptoms and/or biomarker assessments should be used to assess disease stability for newly diagnosed participants naïve to SRLs.
- 7. Historical documentation of positive somatostatin receptor tumor status by PET or somatostatin receptor scintigraphy. Note: If participant does not have historical documentation, this can be done during the Screening Period.
- 8. Participants who are currently treated with octreotide/lanreotide and who agree to wash out of treatment must have historical instance of an elevated 5-HIAA or serotonin level, using either a urine or blood sample. Note: For participants who do not have a historical instance of an elevated 5-HIAA or serotonin level, the result from the samples taken during screening can be considered historical as it is prior to randomization.
- 9. Female participants who engage in heterosexual intercourse must: Be of nonchildbearing potential, defined as either surgically sterile (ie, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), OR Be postmenopausal with at least 1 year of amenorrhea. In participants with less than 1 year of amenorrhea, confirmation is required with 2 FSH measurements. A documented, historical test result measured prior to Screening may be used as 1 of the 2 measurements. The FSH value should be ≥30 IU/L to confirm menopausal status, OR Agree to use a highly effective method of contraception from the beginning of the Screening Period until at least 2 weeks after the last dose of study drug. Contraceptive use by men and women also should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Periodic abstinence (ie, calendar, ovulation, symptothermal, and postovulation methods) and withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method (LAM) are not acceptable methods of contraception. Female condom and male condom should not be used together.
What rules you out
- 1. Diarrhea attributed to any condition(s) other than carcinoid syndrome.
- 10. Unable to administer SA octreotide (octreotide acetate injection), or prior nonresponse documented with somatostatin agonists.
- 11. Known allergy or hypersensitivity to any of the test materials or related compounds.
- 12. Any clinically significant and active infection such as those requiring systemic treatment within 14 days before randomization.
- 13. 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 <30 mL/min/1.73 m2, cirrhosis, baseline AST and/or ALT >2×ULN, and/or TB >1.5×ULN. (Participants with previously diagnosed Gilbert’s syndrome not accompanied by other hepatobiliary disorders and associated with TB <3.5 mg/dL [<51.3 µmol/L] will be permitted.)
- 14. Current alcohol or drug abuse, or within the last year, is not permitted.
- 2. Uncontrolled/severe diarrhea associated with significant volume contraction, dehydration, or hypotension.
- 3. Requires second line treatments (eg, telotristat) for control of carcinoid syndrome symptoms in the opinion of the Investigator.
- 4. Treatment with specific NET therapy <4 weeks before Screening (such as everolimus or sunitinib) or hepatic embolization, radiotherapy, PRRT, and/or tumor debulking <12 weeks before Screening. In addition, the following conditions must be followed: Completion or discontinuation of this prior treatment should have occurred according to SOC practice. Per the Investigator’s assessment, SSAs are appropriate for management of the participant’s carcinoid syndrome symptoms, and there is no anticipated need for the participant to initiate another line of antitumor treatment within the next 4 months (ie, for the duration of the RCP). Withholding or discontinuing antitumor SOC therapy (excluding approved SSAs) solely to enable study participation is not permitted.
- 5. Major surgery within 8 weeks before Screening.
- 6. History of another primary malignancy <3 years prior to the date of randomization in the RCP unless at least 1 of the following criteria are met: 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.
- 7. Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry.
- 8. Poorly controlled diabetes mellitus defined as having a HbA1c ≥8.5% (ie, ≥69.5 mmol/mol) or estimated HbA1c based on fructosamine if HbA1c is not evaluable (eg, due to hemoglobinopathy).
- 9. History of unstable angina or acute myocardial infarction within the 12 weeks preceding the Screening Period or other clinically significant cardiac disease (including clinically significant carcinoid heart disease) at the time of Screening as judged by the Investigator.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Mexico
- Colombia
- Chile
- Brazil
- United Kingdom
- Argentina
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Mexico; Colombia; Chile; Brazil; United Kingdom and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.