A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.

Официальное название

A Phase 1, 3-Part, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Oral Doses of CTX001 in Healthy Adult Participants.

Обзор

This study is testing CTX001 for certain conditions where the body does not have enough available iron or has difficulty storing or moving iron properly. The purpose of this study is to investigate any side effects that may happen with CTX001, how CTX001 is absorbed by and processed in the body, and how CTX001 affects iron levels in the blood when administered with or without iron and/or food.

Подробное описание

The study drug CTX001 is an investigational treatment for certain diseases where the body does not have enough iron in the bone marrow (the hollow inner parts of bones). Iron is an important part of red blood cells, which are made in the bone marrow. When there is not enough iron in the bone marrow, it can lead to anaemia. Conditions like chronic kidney disease, bone marrow diseases, intestinal diseases, and bleeding disorders all can cause anaemia due to low iron levels, or iron that gets trapped in other body parts like the spleen and cannot get to the bone marrow. Although anemia can sometimes be treated with iron pills or iron infusions, some patients cannot tolerate these treatments and others do not respond to them because either their intestines don't absorb the iron or because the body has improperly stored the iron where it can't be used to make red blood cells. CTX001 is a pill that is swallowed by mouth. Inside the body, CTX001 is expected to bind to iron, help the iron be absorbed by the intestine and help the iron move out of body parts where it might be stuck. If effective, CTX001 could be used to treat anemia in patients with chronic health conditions.

Вмешательства

• Препарат CTX001 With and Without Iron
  There are two treatment periods. In the first, CTX001 is administered as a single dose (tablet) by itself. In the second, CTX001 is administered as a single dose (tablet) with iron.
• Препарат CTX001 With Iron
  There are two treatment periods. In one, CTX001 is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, CTX001 is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
• Препарат CTX001 Multiple Dose
  CTX001 (tablet) is administered daily for 7 consecutive days.
• Препарат Placebo With and Without Iron
  There are two treatment periods. In the first, placebo is administered as a single dose (tablet) by itself. In the second, placebo is administered as a single dose (tablet) with iron.
• Препарат Placebo With Iron
  There are two treatment periods. In one, placebo is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, placebo is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
• Препарат Placebo Multiple Dose
  Placebo (tablet) is administered daily for 7 consecutive days.

Первичные конечные точки

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [Срок оценки: From first dose of study drug through the last study visit, approximately 15 days.]
• Number of Participants with Serious TEAEs [Срок оценки: From first dose of study drug through the last study visit, approximately 15 days.]
• Number of Participants with Clinically Significant Changes in Physical Examination [Срок оценки: From the first dose of study drug through the last study visit; approximately 15 days.]
• Number of Participants with Clinically Significant Changes in Safety Laboratory Values [Срок оценки: From first dose of study drug through last study visit, approximately 15 days.]
• Number of Participants with Clinically Significant Changes in Vital Signs [Срок оценки: From the first dose of study drug through the last study visit, approximately 15 days.]
• Number of Participants with Clinically Significant Changes in Electrocardiograms [Срок оценки: From the first dose of study drug through the last study visit, approximately 15 days.]

Вторичные конечные точки (10)

• Maximum Observed Plasma Concentration (Cmax) of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Time to Maximum Observed Plasma Concentration (Tmax) of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Area Under the Curve from Time Zero Extrapolated to Infinity (AUC0-inf) of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Area Under the Curve From Time Zero to Time of Last Quantifiable Concentration (AUC0-t) of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Apparent Clearance (CL/F) of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Plasma Half-Life of CTX001 [Срок оценки: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Maximum Change in Serum Iron From Baseline [Срок оценки: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Maximum Change in Serum Transferrin From Baseline [Срок оценки: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Maximum Change in Serum Transferrin Saturation From Baseline [Срок оценки: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]
• Maximum Change in Serum Ferritin From Baseline [Срок оценки: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).]

Критерии участия

Критерии включения

• Capable of giving informed consent
• Agrees to use effective contraception
• Body Mass Index (BMI) between 18.0 and 32.0 kg/m2
• Healthy by medical evaluation and medical history
• Hematological parameters, serum iron, transferrin and ferritin are within normal range and transferrin saturation is within normal range and greater than or equal to 20%
• Can swallow tablets and has suitable venous access for blood sampling

Критерии исключения

• Has dietary requirements that may be difficult to accommodate
• Is a regular user of cannabis or has a history of illicit drug abuse within 1 year
• Has a history of alcohol abuse or binge drinking within 6 months
• Is a regular user of tobacco or nicotine-containing products
• Unwilling or unable to comply with the lifestyle guidelines described in the protocol
• Has clinically significant history or evidence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurological, immunological, or psychiatric disorder(s) as determined by the Investigator
• Has any concurrent disease or condition or physical, psychological, mental, and/or social reason that, in the opinion of the Investigator, would make the participant unsuitable for participation in the clinical study
• Has received a blood transfusion within 1 year
• Has donated whole blood within 6 months or plasma within 30 days
• Requires prescription medication or regular use of non-prescription medication
• Is an employee of the Sponsor, the CRO, or of any organization or site(s) associated with this study, or any immediate family member who is in a dependent relationship with a study site employee who is involved in the conduct of the study

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Да

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Австралия · 1 центр

• Nucleus Network — Melbourne

Идентификаторы

NCT: NCT07577817 · CTX001-101

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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