A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.
5. května 2026 aktualizováno: 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.
Přehled studie
Postavení
Zatím nenabíráme
Detailní popis
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 studie
Intervenční
Zápis (Odhadovaný)
72
Fáze
- Raná fáze 1
Kontakty a umístění
Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.
Studijní místa
-
-
Victoria
-
Melbourne, Victoria, Austrálie, 3004
- Nucleus Network
-
-
Kritéria účasti
Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
Přijímá zdravé dobrovolníky
Ano
Popis
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
Studijní plán
Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Sekvenční přiřazení
- Maskování: Čtyřnásobek
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Experimentální: Single Ascending Dose (CTX001)
Up to 5 sequential, single ascending dose cohorts.
|
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.
|
|
Komparátor placeba: Single Ascending Dose (Placebo)
Up to 5 sequential, single ascending dose cohorts
|
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.
|
|
Experimentální: Food Effect (CTX001)
Up to 3 sequential, single ascending dose food effect cohorts
|
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.
|
|
Komparátor placeba: Food Effect (Placebo)
Up to 3 sequential, single ascending dose food effect cohorts
|
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.
|
|
Experimentální: Multiple Ascending Dose (CTX001)
Up to 3 multiple ascending dose cohorts.
|
CTX001 (tablet) is administered daily for 7 consecutive days.
|
|
Komparátor placeba: Multiple Ascending Dose (Placebo)
Up to 3 multiple ascending dose cohorts
|
Placebo (tablet) is administered daily for 7 consecutive days.
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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.
|
Sekundární výstupní opatření
Měření výsledku |
Časové okno |
|---|---|
|
Maximum Observed Plasma Concentration (Cmax) of CTX001
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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).
|
Spolupracovníci a vyšetřovatelé
Zde najdete lidi a organizace zapojené do této studie.
Sponzor
Termíny studijních záznamů
Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.
Hlavní termíny studia
Začátek studia (Odhadovaný)
1. června 2026
Primární dokončení (Odhadovaný)
1. října 2026
Dokončení studie (Odhadovaný)
30. prosince 2026
Termíny zápisu do studia
První předloženo
5. května 2026
První předloženo, které splnilo kritéria kontroly kvality
5. května 2026
První zveřejněno (Aktuální)
11. května 2026
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
11. května 2026
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
5. května 2026
Naposledy ověřeno
1. května 2026
Více informací
Termíny související s touto studií
Klíčová slova
Další relevantní podmínky MeSH
- Urogenitální onemocnění
- Patologické procesy
- Mužská urogenitální onemocnění
- Onemocnění ledvin
- Urologická onemocnění
- Ženské urogenitální onemocnění
- Ženské urogenitální onemocnění a těhotenské komplikace
- Chronické onemocnění
- Atributy nemoci
- Metabolické choroby
- Hematologická onemocnění
- Renální insuficience
- Poruchy metabolismu železa
- Patologické stavy, příznaky a symptomy
- Nutriční a metabolické nemoci
- Hemická a lymfatická onemocnění
- Nedostatky železa
- Renální insuficience, chronická
- Anémie
- Anorganické chemikálie
- Prvky
- Kovy
- Kovy, těžké
- Přechodové prvky
- Železo
- exagamglogene autotemcel
Další identifikační čísla studie
- CTX001-101
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
NE
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Ne
Studuje produkt zařízení regulovaný americkým úřadem FDA
Ne
produkt vyrobený a vyvážený z USA
Ne
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .