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ALXN1840

Phase 2

Wilson Disease | Small molecule | Rare Disease |AstraZeneca PLC|Last Updated: Aug 19, 2024

Success Probability

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials5
Total Enrollment152

FDA Designations

No designations recorded

Clinical trial landscape

ALXN1840 · 10 trials · 2 indications

Phase 2 2Phase 1 8
NCT04573309Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840Wilson Disease
COMPLETED9 Analytics
NCT02273596Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease PatientsWilson Disease
COMPLETED29 Analytics
PHASE2COMPLETED
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Wilson DiseaseUnlock trial analytics
PHASE2COMPLETED
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Wilson DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Mean Daily Copper Balance: Day 1 Through Day 8
Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)

Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.

Mean Daily Copper Balance: Day 31 Through Day 35
Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)

Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.

Mean Daily Copper Balance: Day 25 Through Day 28
Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)

Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.

Mean Daily Copper Balance: Day 36 Through Day 39
Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)

Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.

Percentage Of Participants With Normalized Concentrations Of NCC
Week 24

Normalized concentrations of NCC was defined as who achieving or maintaining normalized levels of NCC (0.8 to 2.3 micromole \[μmol\]l/liter \[L\]\]) adjusted for Mo plasma concentration or reaching a reduction of at least 25% in NCC corrected for Mo if above the normal reference range at the time of enrollment. NCC was calculated by subtracting the amount of copper (Cu) bound to ceruloplasmin (CP) from the total plasma Cu concentration. Post-baseline NCC values were adjusted (corrected) to account for Cu bound in tripartite complexes with ALXN1840 and albumin. Descriptive statistics are reported.

Two-way Crossover Period: Maximum Observed Concentration (Cmax) For Plasma Total Molybdenum (Mo)
predose (0.5 hour) and up to 336 hours postdose

Whole blood samples were collected for the measurement of plasma concentrations of total Mo via inductively coupled plasma-mass spectroscopy (ICP-MS).

Two-way Crossover Period: Cmax for PUF Mo
predose (0.5 hour) and up to 336 hours postdose

Whole blood samples were collected for the measurement of plasma concentrations of PUF Mo via ICP-MS.

Two-way Crossover Period: Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Quantifiable Concentration (AUCt) For Plasma Total Mo
predose (0.5 hour) and up to 336 hours postdose

Whole blood samples were collected for the measurement of plasma concentrations of total Mo via ICP-MS.

Two-way Crossover Period: AUCt for Plasma PUF Mo
predose (0.5 hour) and up to 336 hours postdose

Whole blood samples were collected for the measurement of plasma concentrations of PUF Mo via ICP-MS.

Two-way Crossover Period: Area Under The Plasma Concentration Versus Time Curve From Time 0 To Infinity (AUCinf) For Plasma Total Mo
predose (0.5 hour) and up to 336 hours postdose

Whole blood samples were collected for the measurement of plasma concentrations of total Mo via ICP-MS.

Maximum Observed Plasma Concentration (Cmax) of Bupropion With and Without the Coadministration of ALXN1840
Pre-dose (Day 1) up to 336 hours post-dose
Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Last Quantifiable Concentration (AUCt) of Bupropion With and Without the Coadministration of ALXN1840
Pre-dose (Day 1) up to 336 hours post-dose
Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUCinf) of Bupropion With and Without the Coadministration of ALXN1840
Pre-dose (Day 1) up to 336 hours post-dose
Placebo-corrected Change From Baseline For QTcF (ΔΔQTcF) for ALXN1840 Using The By-time Point Analysis
Baseline (average of samples taken at -45, -30, and -15 minutes before dosing), 0.5, 1, 2, 3, 4, 5, 6, 7, 8, 10, 12, and 24 (Day 2) hours postdose

Twelve-lead electrocardiograms (ECGs) were extracted from approximately 25-hour continuous (Holter) recordings on Day -1 of Treatment Period 1 and Days 1 and 2 in each treatment period. Change from baseline in the QT interval was corrected for heart rate using Fridericia's formula (ΔQTcF). ΔQTcF was based on a mixed-effects model for repeated measures (MMRM) with ΔQTcF as the dependent variable; period, sequence, time, treatment, and time-by-treatment interaction as fixed effects; and baseline QTc and sex as covariates. ΔΔQTc = LS mean ΔQTcF after ALXN1840 dosing minus LS mean ΔQTcF after placebo. If the upper bound of the confidence interval (CI) of ΔΔQTcF was \< 10 ms for all postdose time points, ALXN1840 was concluded to not have a significant effect on QT interval prolongation.

Maximum Observed Plasma Concentration (Cmax) Of Celecoxib With And Without The Coadministration Of ALXN1840
Baseline, up to 336 hours post-dose

Blood samples were collected for pharmacokinetics (PK) analysis of celecoxib. Cmax is reported as nanograms (ng)/milliliter (mL).

Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Quantifiable Concentration (AUCt) Of Celecoxib With And Without The Coadministration Of ALXN1840
Baseline, up to 336 hours post-dose

Blood samples were collected for pharmacokinetic (PK) analysis of celecoxib. Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Quantifiable Concentration (AUCt) is reported as hours•ng/mL (h•ng/mL).

Area Under The Plasma Concentration Versus Time Curve From Time 0 To Infinity (AUCinf) Of Celecoxib With And Without The Coadministration Of ALXN1840
Baseline, up to 336 hours post-dose

Blood samples were collected for pharmacokinetic (PK) analysis of celecoxib. Area Under The Plasma Concentration Versus Time Curve From Time 0 To Infinity (AUCinf) is reported as h•ng/mL.

Change From Baseline in Mean Daily Copper Balance Over 2 Weeks of Repeated Daily ALXN1840 Dosing (Over Days 4 to 15)
Baseline, Days 4 to 15

Copper balance was defined as the difference in copper input and copper output. A negative copper balance indicated greater copper output than copper intake. Copper input was defined as the sum of all copper input as measured in all food and fluids over the specified period. Copper output was defined as the sum of all copper output as measured in urine and feces over the specified collection period. Baseline was defined as the average of the nonmissing values on or before first study drug administration from Day -4 to Day -1.

Maximum Observed (Plasma) Concentration (Cmax) of Total Molybdenum
Up to 240 hours postdose

The pharmacokinetic (PK) data of plasma total molybdenum were analyzed in the scope of this study as a surrogate measure for ALXN1840. Whole blood samples were collected for the measurement of plasma concentrations of total molybdenum via inductively coupled plasma-mass spectroscopy (ICP-MS).

Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) to the Last Quantifiable Concentration (AUCt) of Total Molybdenum
Up to 240 hours postdose

The PK data of plasma total molybdenum were analyzed in the scope of this study as a surrogate measure for ALXN1840. Whole blood samples were collected for the measurement of plasma concentrations of total molybdenum via ICP-MS.

Area Under The Plasma Concentration Versus Time Curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) Of Plasma Total Molybdenum After Each Dose
Day 1 through Day 11 of Dosing Periods 1 and 2
Area Under the Plasma Concentration Versus Time Curve, From Time 0 to the Last Measurable Concentration (AUC0-t) of Total Molybdenum (Mo)
Predose (0 hour) up to 192 hours postdose

AUC0-t was calculated by the linear trapezoidal method.

Maximum Measured Plasma Concentration (Cmax) of Total Mo
Predose (0 hour) up to 192 hours postdose
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Day 1 through 14 days following final dose (up to Day 43)

An adverse event (AE) was defined as any untoward medical occurrence in a participant administered with the study drug and which did not necessarily have a causal relationship with the study drug. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. A TEAE was defined as an AE that started or worsened at the time of or after study drug administration. An AE that occurred during the washout period between drugs was considered treatment emergent to the last drug given. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Secondary Endpoints

Change From Baseline In Mean Daily Copper Balance
Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALXN1840EXPERIMENTALParticipants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39
Crossover ALXN1840 Sequence 1EXPERIMENTALParticipants will first receive a single dose of ALXN1840 test formulation on Day 1 of Period 1. After a washout period of 14 days, they will then receive a single dose of ALXN1840 reference formulation on Day 1 of Period 2.
Crossover ALXN1840 Sequence 2EXPERIMENTALParticipants will first receive a single dose of ALXN1840 reference formulation on Day 1 of Period 1. After a washout period of 14 days, they will then receive a single dose of ALXN1840 test formulation on Day 1 of Period 2.
Parallel Dose-proportionality Extension: ALXN1840 Dose 1EXPERIMENTALParticipants will receive a single dose of ALXN1840.
Parallel Dose-proportionality Extension: ALXN1840 Dose 2EXPERIMENTALParticipants will receive a single dose of ALXN1840.
Parallel Dose-proportionality Extension: ALXN1840 Dose 3EXPERIMENTALParticipants will receive a single dose of ALXN1840.
Parallel Dose-proportionality Extension: ALXN1840 Dose 4EXPERIMENTALParticipants will receive a single dose of ALXN1840.
Treatment AEXPERIMENTALParticipants will receive bupropion.
Treatment BEXPERIMENTALParticipants will receive bupropion with ALXN1840.
Treatment Sequence 1EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: ALXN1840. Period 2: Placebo-matching ALXN1840. Period 3: Moxifloxacin.
Treatment Sequence 2EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: ALXN1840. Period 2: Moxifloxacin. Period 3: Placebo-matching ALXN1840.
Treatment Sequence 3EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: Placebo-matching ALXN1840. Period 2: ALXN1840. Period 3: Moxifloxacin.
Treatment Sequence 4EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: Placebo-matching ALXN1840. Period 2: Moxifloxacin. Period 3: ALXN1840.
Treatment Sequence 5EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: Moxifloxacin. Period 2: ALXN1840. Period 3: Placebo-matching ALXN1840.
Treatment Sequence 6EXPERIMENTALOn Day 1 of each period, participants will receive a single dose of the following study interventions: Period 1: Moxifloxacin. Period 2: Placebo-matching ALXN1840. Period 3: ALXN1840.
Treatment Sequence A-BEXPERIMENTALParticipants received 1 treatment during each study period in the following sequence: * Treatment A: Celecoxib. * Treatment B: Celecoxib plus ALXN1840.
Treatment Sequence B-AEXPERIMENTALParticipants received 1 treatment during each study period in the following sequence: * Treatment B: Celecoxib plus ALXN1840. * Treatment A: Celecoxib.
Sequence 1 (AB)EXPERIMENTALParticipants received ALXN1840 once in each Period as a single oral dose under fasted conditions as follows: Period 1: ALXN1840 as a single EC tablet (Treatment A, reference). Period 2: ALXN1840 as three EC tablets (Treatment B, test). Participants were discharged following the 240-hour post-dose procedures (approximately 10 days after dosing in each period) unless it was medically necessary to extend the confinement. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 2 (BA)EXPERIMENTALParticipants received ALXN1840 once in each Period as a single oral dose under fasted conditions as follows: Period 1: ALXN1840 as three EC tablets (Treatment B, test). Period 2: ALXN1840 as a single EC tablet (Treatment A, reference). Participants were discharged following the 240-hour post-dose procedures (approximately 10 days after dosing in each period) unless it was medically necessary to extend the confinement. There was a washout period of at least 14 days between each ALXN1840 dosing.
Cohort 1: Japanese ParticipantsEXPERIMENTALAll Japanese participants will receive a single dose of ALXN1840 15 milligrams (mg) in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2.
Cohort 2: Non-Japanese ParticipantsEXPERIMENTALAll non-Japanese participants will receive a single dose of ALXN1840 15 mg in Dosing Period 1 and will receive a single dose of ALXN1840 60 mg in Dosing Period 2.
Sequence 1: ABCEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment A): ALXN1840 following an overnight fast. Period 2 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. Period 3 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 2: ACBEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment A): ALXN1840 following an overnight fast. Period 2 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. Period 3 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 3: BACEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. Period 2 (Treatment A): ALXN1840 following an overnight fast. Period 3 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 4: BCAEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. Period 2 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. Period 3 (Treatment A): ALXN1840 following an overnight fast. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 5: CABEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. Period 2 (Treatment A): ALXN1840 following an overnight fast. Period 3 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. There was a washout period of at least 14 days between each ALXN1840 dosing.
Sequence 6: CBAEXPERIMENTALParticipants received each treatment on 1 occasion: Period 1 (Treatment C): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, approximately 30 minutes after the start of a high-fat breakfast. Period 2 (Treatment B): Omeprazole once daily in the morning of Days -5 to -1 following an overnight fast, omeprazole at Hour -1 on Day 1 following an overnight fast, and ALXN1840 at Hour 0 on Day 1, following an overnight fast. Period 3 (Treatment A): ALXN1840 following an overnight fast. There was a washout period of at least 14 days between each ALXN1840 dosing.

Interventions

NameTypeDescription
ALXN1840DRUGAdministered orally as tablets.
Bupropion HydrochlorideDRUGBupropion hydrochloride will be administered orally as a single dose as one 150 milligrams (mg) tablet with 240 milliliters of water (fasting).
PlaceboDRUGPlacebo will be administered orally.
MoxifloxacinDRUGMoxifloxacin (400 milligrams) will be administered orally.
CelecoxibDRUGCelecoxib was administered orally as a single dose as one 200-mg tablet with 240 mL of water (fasting).
OmeprazoleDRUGOmeprazole (20 milligrams) was administered orally as a delayed-release capsule in the morning of Days -5 to -1 and at Hour -1 on Day 1.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Diagnosis of WD by Leipzig Criteria ≥ 4. 2. Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum. 3. Participants willing to adhere to copper/molybdenum-controlled diet during the study. 4. Willing and able to follow protoco...

Countries:United StatesNew ZealandUnited KingdomAustriaGermanyPolandAustralia
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Frequently asked questions about ALXN1840

What is ALXN1840 used for?

ALXN1840 is an investigational small molecule being developed for Wilson disease. It is currently in Phase 1 clinical development, with studies conducted in healthy adults to evaluate its safety, tolerability, and pharmacological properties. The drug is not yet approved and remains under clinical investigation.

Who is developing ALXN1840?

ALXN1840 is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker AZN. The company is conducting Phase 1 clinical trials to evaluate the drug's properties in healthy volunteers.

What phase is ALXN1840 in?

ALXN1840 is in Phase 1 clinical development. All five clinical trials associated with the drug have been completed, with a total enrollment of 188 participants. The studies were randomized, double-blind, and controlled, focusing on healthy adults.

What clinical trials has ALXN1840 been in?

ALXN1840 has been studied in five completed Phase 1 trials. These include NCT04560816, a cardiac effects study in healthy US adults; NCT04610580, a bioavailability study in Australia; NCT05303324, a dose strength study in the UK; and NCT05319899, a study with and without omeprazole in the US.

Is ALXN1840 the same as any other drug?

ALXN1840 is the primary name used for this investigational drug. No alternative names have been reported for this compound in the available clinical trial information.