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

10 czerwca 2026 zaktualizowane przez: Cajal Therapeutics Inc.

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.

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.

Przegląd badań

Status

Rekrutacyjny

Warunki

Interwencja / Leczenie

Szczegółowy opis

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.

Typ studiów

Interwencyjne

Zapisy (Szacowany)

72

Faza

  • Wczesna faza 1

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

Lokalizacje studiów

  • Australia
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Rekrutacyjny
        • Nucleus Network

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dorosły

Akceptuje zdrowych ochotników

Tak

Opis

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Randomizowane
  • Model interwencyjny: Zadanie sekwencyjne
  • Maskowanie: Poczwórny

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: Single Ascending Dose (CTX001)
Up to 5 sequential, single ascending dose cohorts.
Lek: 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.
Komparator placebo: Single Ascending Dose (Placebo)
Up to 5 sequential, single ascending dose cohorts
Lek: 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.
Eksperymentalny: Food Effect (CTX001)
Up to 3 sequential, single ascending dose food effect cohorts
Lek: 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.
Komparator placebo: Food Effect (Placebo)
Up to 3 sequential, single ascending dose food effect cohorts
Lek: 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.
Eksperymentalny: Multiple Ascending Dose (CTX001)
Up to 3 multiple ascending dose cohorts.
Lek: CTX001 Multiple Dose
CTX001 (tablet) is administered daily for 7 consecutive days.
Komparator placebo: Multiple Ascending Dose (Placebo)
Up to 3 multiple ascending dose cohorts
Lek: Placebo Multiple Dose
Placebo (tablet) is administered daily for 7 consecutive days.

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Ramy czasowe: From first dose of study drug through the last study visit, approximately 15 days.
An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to the study intervention. An AE was considered a treatment-emergent AE (TEAE) if the AE started after initial study drug administration and before the last study visit.
From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Serious TEAEs
Ramy czasowe: From first dose of study drug through the last study visit, approximately 15 days.
A serious AE (SAE) is defined as any AE that, at any dose, meets one or more of the following criteria: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital anomaly or birth defect; or any important medical event that may jeopardize the participant or may require medical intervention to prevent one of the outcomes listed above.
From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Physical Examination
Ramy czasowe: From the first dose of study drug through the last study visit; approximately 15 days.
Abnormalities in physical examinations were based on investigator's discretion.
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
Ramy czasowe: From first dose of study drug through last study visit, approximately 15 days.
Safety laboratory evaluations included blood counts, serum chemistries, coagulation studies, and urinalyses. Determination of clinical significance was based on investigator discretion.
From first dose of study drug through last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Vital Signs
Ramy czasowe: From the first dose of study drug through the last study visit, approximately 15 days.
Vital signs included heart rate, respiratory rate, blood pressure, and temperature. Determination of clinical significance was based on investigator discretion.
From the first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Electrocardiograms
Ramy czasowe: From the first dose of study drug through the last study visit, approximately 15 days.
Electrocardiograms are tests that measure the electrical activity of the heart. Determination of clinical significance was based on investigator discretion.
From the first dose of study drug through the last study visit, approximately 15 days.

Miary wyników drugorzędnych

Miara wyniku
Ramy czasowe
Maximum Observed Plasma Concentration (Cmax) of CTX001
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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
Ramy czasowe: 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).
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).

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Cajal Therapeutics Inc.

Śledczy

  • Główny śledczy: Aarthy Joseph, Dr, Nucleus Network

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Szacowany)

1 czerwca 2026

Zakończenie podstawowe (Szacowany)

1 października 2026

Ukończenie studiów (Szacowany)

30 grudnia 2026

Daty rejestracji na studia

Pierwszy przesłany

5 maja 2026

Pierwszy przesłany, który spełnia kryteria kontroli jakości

5 maja 2026

Pierwszy wysłany (Rzeczywisty)

11 maja 2026

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

12 czerwca 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

10 czerwca 2026

Ostatnia weryfikacja

1 maja 2026

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • CTX001-101

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIE

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Nie

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

produkt wyprodukowany i wyeksportowany z USA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

Badania kliniczne na Niedokrwistość

Badania kliniczne na CTX001 With and Without Iron

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