Clinical trials appendix


As of 12 November 2024

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Asset Trial Population Patients Design Endpoints Status Development stage Indication
Asset
Wainua
(eplontersen, ligand-conjugated antisense)
Trial
CVRM
Phase III
CARDIO-TTRansform
NCT04136171
Partnered (Ionis Pharmaceuticals, Inc.)
Population
Hereditary or wild-type transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)
Patients
1438
Design
  • Arm 1: Wainua s.c.
  • Arm 2: placebo
Endpoints
  • Primary endpoints: composite outcome of CV mortality and recurrent CV clinical events at Week 140
  • Secondary endpoints: 6MWT, KCCQ, CV events and CV mortality
Status
  • FPCD: Q1 2020
  • Data anticipated: 2026
Development stage
Approved medicines
Indication
Amyloidosis
Asset
Wainua
(eplontersen, ligand-conjugated antisense)
Trial
CVRM
Phase III
NEURO-TTRansform
NCT04136184
Partnered (Ionis Pharmaceuticals, Inc.)
Population
Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
Patients
168
Design
  • Arm 1: Wainua s.c.
  • Arm 2: inotersen s.c.
Endpoints
  • Primary endpoints (at Week 35): change from baseline in mNIS+7 and percent change from baseline in TTR concentration
  • Secondary endpoint (Week 35): changes from baseline in Norfolk QOL
  • Primary endpoints (at Week 66): change from baseline in mNIS+7, change from baseline in the Norfolk QoL-DN Questionnaire and percent change from baseline in TTR concentration
Status
  • FPCD: Q1 2020
  • LPCD: Q3 2023
  • Data readout: Q2 2022
  • Co-primary endpoints met at Week 35 and Week 66
Development stage
Approved medicines
Indication
Amyloidosis
Asset
Wainua
(eplontersen, ligand-conjugated antisense)
Trial
CVRM
Phase III
EPIC-ATTR
NCT06194825
Population
ATTR-CM
Patients
64
Design
  • Arm 1: Wainua s.c. Q4W
  • Arm 2: placebo
  • China only
Endpoints
  • Primary endpoint (at week 24): percent change from baseline in serum TTR concentration
  • Secondary endpoints: PK, immunogenicity, disease biomarkers (NT pro-BNP, hsTnT)
Status
  • FPCD: Q4 2023
  • Data anticipated: H1 2025
Development stage
Approved medicines
Indication
Amyloidosis
Asset
AZD0233
(oral CX3CR1)
Trial
CVRM
Phase I
NCT06381466
Population
Healthy volunteers
Patients
96
Design
  • Randomised, SAD/MAD dose escalating trial
Endpoints
  • Primary endpoints: safety and tolerability
  • Secondary endpoints: PK parameters
Status
  • FPCD: Q2 2024
  • Data anticipated: H2 2025
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase II
PURSUIT
NCT06173570
Population
Dyslipidaemia
Patients
428
Design
  • Randomised trial with equal distribution across five parallel treatment arms to either placebo or one of four AZD0780 doses
Endpoints
  • Primary endpoint: percent change in LDL-C level from baseline to Week 12
  • Secondary endpoints: percent change from baseline of LDL-C at Week 12, plasma concentrations summarised by sampling timepoint, percent change from baseline at Week 12 in other lipid parameters and inflammatory markers and safety and tolerability
Status
  • FPCD: Q1 2024
  • LPCD: Q2 2024
  • Data anticipated: H1 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase II
NCT06692764
Population
Participants with ASCVD or risk equivalents and LDL-C ≥70 mg/dL on stable medication
Patients
172
Design
  • Multi-centre, randomised, double-blind, placebo-controlled, crossover trial
Endpoints
  • Primary endpoint: ambulatory 24-hour average systolic blood pressure at Week 4
  • Secondary endpoint: ambulatory 24-hour average diastolic blood pressure at Week 4
Status
  • FPCD: Q4 2024
  • Data anticipated: H1 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT05384262
Population
Healthy volunteers
Patients
183
Design
  • Randomised, placebo-controlled SAD/MAD trial
Endpoints
  • Primary endpoints: safety and tolerability
Status
  • FPCD: Q2 2022
  • LPCD: Q2 2024
  • Data readout: Q4 2024
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT05787002
Population
Healthy volunteers
Patients
16
Design
  • Open-label, two-period, two-sequence crossover trial to assess the effect of AZD0780 on the PK of Crestor
Endpoints
  • Primary endpoints: PK parameters, safety and tolerability
Status
  • FPCD: Q1 2023
  • LPCD: Q2 2023
  • Data readout: Q4 2023
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT05817461
Population
Healthy volunteers
Patients
8
Design
  • Open-label, two-part sequential human ADME trial
Endpoints
  • Primary endpoints: mass balance recovery, absorption, metabolism, excretion of [14C]AZD0780 and absolute bioavailability of AZD0780
  • Secondary endpoints: safety and tolerability
Status
  • FPCD: Q2 2023
  • LPCD: Q2 2023
  • Data readout: Q4 2023
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT06576765
Population
Hepatic impairment and matched healthy controls
Patients
32
Design
  • Multi-centre, single-dose, non-randomised, open-label, parallel-group trial
Endpoints
  • Primary endpoint: PK parameters
  • Secondary endpoints: safety and tolerability
Status
  • FPCD: Q3 2024
  • LPCD: Q4 2024
  • Data anticipated: H1 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT06592482
Population
Renal impairment and matched healthy controls
Patients
42
Design
  • Multi-centre, single-dose, non-randomised, open-label, parallel-group trial
Endpoints
  • Primary endpoint: PK parameters
  • Secondary endpoints: safety and tolerability
Status
  • FPCD: Q3 2024
  • LPCD: Q4 2024
  • Data anticipated: H1 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT06671405
Population
Healthy volunteers
Patients
78
Design
  • Open-label, fixed sequence trial to assess the PK of AZD0780 when administered in combination with itraconazole, carbamazepine, and the PK of midazolam and EE/LNG when administered with AZD0780
Endpoints
  • Primary endpoint: PK parameters
  • Secondary endpoints: safety and PK parameters
Status
  • FPCD: Q4 2024
  • Data anticipated: H2 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD0780
(PCSK9 inhibitor)
Trial
CVRM
Phase I
NCT06742853
Population
Healthy volunteers with elevated LDL-C
Patients
120
Design
  • Randomized, single-blind, placebo-controlled
Endpoints
  • Primary endpoints: percent change in LDL-C at Week-4 and safety and tolerability
Status
  • FPCD: Q4 2024
  • Data anticipated: H2 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD1705
(Angptl3 inhibitor)
Trial
CVRM
Phase I
NCT06238466
Population
Dyslipidaemia
Patients
112
Design
  • Part A: single dose of AZD1705 with an in-clinic period of 3 days followed by an outpatient follow-up period of approximately 16 weeks
  • Part B: 2 doses of AZD1705 given 28 days apart with an in-clinic period followed by an outpatient follow-up period of approximately 20 weeks
Endpoints
  • Primary endpoints: AEs and SAEs
  • Secondary endpoints: AUCinf, AUClast, Cmax, Ae, fe, CLR, LDL-C, ApoB, triglycerides and target plasma protein
Status
  • FPCD: Q1 2024
  • Data anticipated: H2 2025
Development stage
Early development
Indication
Dyslipidaemia
Asset
AZD2373
(APOL1)
Trial
CVRM
Phase I
NCT04269031
Population
Healthy volunteers
Patients
30
Design
  • SAD dose escalation in 6 cohorts with 6 volunteers receiving AZD2373 and 2 volunteers receiving placebo in each cohort 
  • Arm 1: AZD2373 s.c.
  • Arm 2: placebo s.c.
  • US only
Endpoints
  • Primary endpoints: safety and tolerability 
  • Secondary endpoint: PK parameters
Status
  • FPCD: Q1 2020
  • LPCD: Q3 2021
  • Data readout: Q3 2022
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD2373
(APOL1)
Trial
CVRM
Phase I
NCT05351047
Population
Healthy volunteers
Patients
24
Design
  • MAD dose escalation in 3 cohorts with 6 volunteers per cohort receiving AZD2373 and 2 volunteers per cohort receiving placebo
  • Arm 1: AZD2373 s.c.
  • Arm 2: placebo s.c.
  • US only
Endpoints
  • Primary endpoints: safety and tolerability
  • Secondary endpoints: PK parameters, effect of s.c. MAD administrations of AZD2373 on plasma concentrations of APOL1 protein and APOL1 G0, G1, G2 allele genotype status in trial participants
Status
  • FPCD: Q2 2022
  • LPCD: Q1 2023
  • Data readout: Q4 2023
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD2389
(anti-fibrotic mechanism)
Trial
CVRM
Phase II
BORANA
NCT06750276
Population
Participants with liver fibrosis and compensated cirrhosis
Patients
36
Design
  • Randomised, single-blind, placebo-controlled trial
Endpoints
  • Primary endpoints: safety and tolerability
Status
  • FPCD: Q4 2024
  • Data anticipated: 2026
Development stage
Early development
Indication
Liver disease
Asset
AZD2389
(anti-fibrotic mechanism)
Trial
CVRM
Phase I
NCT06138795
Population
Healthy volunteers
Patients
104
Design
  • Randomised, placebo-controlled SAD/MAD trial
Endpoints
  • Primary endpoints: safety and tolerability
Status
  • FPCD: Q4 2023
  • Data anticipated: H1 2025
Development stage
Early development
Indication
Healthy volunteer
Asset
AZD2693
(PNPLA3 ASO)
Trial
CVRM
Phase IIb
FORTUNA
NCT05809934
Population
NASH with fibrosis
Patients
180
Design
  • Randomised, double-blind, placebo-controlled, multi-centre trial
  • Arm 1: AZD2693 s.c. dose 1
  • Arm 2: AZD2693 s.c. dose 2
  • Arm 3: placebo s.c.
  • Global trial
Endpoints
  • Primary endpoints: efficacy, safety and tolerability
Status
  • FPCD: Q2 2023
  • LPCD: Q3 2024
  • Data anticipated: 2026
Development stage
Early development
Indication
Liver disease
Asset
AZD2693
(PNPLA3 ASO)
Trial
CVRM
Phase I
NCT04483947
Population
NASH/NAFLD F0-F3
Patients
74
Design
  • MAD with 4 cohorts receiving AZD2693 and placebo in each cohort 
  • Arm 1: AZD2693 s.c.
  • Arm 2: placebo s.c.
  • US only
Endpoints
  • Primary endpoints: safety and tolerability 
  • Secondary endpoint: PK parameters
Status
  • FPCD: Q2 2021
  • LPCD: Q3 2023
  • Data readout: Q2 2024
Development stage
Early development
Indication
Liver disease