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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Ascending Dose (CTX001) | Experimental | Up to 5 sequential, single ascending dose cohorts. |
|
| Single Ascending Dose (Placebo) | Placebo Comparator | Up to 5 sequential, single ascending dose cohorts |
|
| Food Effect (CTX001) | Experimental | Up to 3 sequential, single ascending dose food effect cohorts |
|
| Food Effect (Placebo) | Placebo Comparator | Up to 3 sequential, single ascending dose food effect cohorts |
|
| Multiple Ascending Dose (CTX001) | Experimental | Up to 3 multiple ascending dose cohorts. |
|
| Multiple Ascending Dose (Placebo) | Placebo Comparator | Up to 3 multiple ascending dose cohorts |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX001 With and Without Iron | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Treatment-Emergent Adverse Events (TEAEs) | 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 | 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 | 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 | 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 | Vital signs included heart rate, respiratory rate, blood pressure, and temperature. Determination of clinical significance was based on investigator discretion. |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Plasma Concentration (Cmax) of CTX001 | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Anthony Johnson | Contact | +61 07 3707 2787 | a.johnson@nucleusnetwork.com.au |
| Name | Affiliation | Role |
|---|---|---|
| Aarthy Joseph, Dr | Nucleus Network | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nucleus Network | Recruiting | Melbourne | Victoria | 3004 | Australia |
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|
| CTX001 With Iron | Drug | 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. |
|
| CTX001 Multiple Dose | Drug | CTX001 (tablet) is administered daily for 7 consecutive days. |
|
| Placebo With and Without Iron | Drug | 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. |
|
| Placebo With Iron | Drug | 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. |
|
| Placebo Multiple Dose | Drug | Placebo (tablet) is administered daily for 7 consecutive days. |
|
| From the first dose of study drug through the last study visit, approximately 15 days. |
| Number of Participants with Clinically Significant Changes in Electrocardiograms | 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. |
| 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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). |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D000090463 | Iron Deficiencies |
| D051436 | Renal Insufficiency, Chronic |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D051437 | Renal Insufficiency |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000729927 | exagamglogene autotemcel |
| D007501 | Iron |
| ID | Term |
|---|---|
| D019216 | Metals, Heavy |
| D004602 | Elements |
| D007287 | Inorganic Chemicals |
| D028561 | Transition Elements |
| D008670 | Metals |
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