A Phase 1 interventional study of AZD4063 in Dilated Cardiomyopathy, sponsored by AstraZeneca. Recruiting at 4 sites in Netherlands. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-09-04.
Sponsored by AstraZeneca · Phase 1, Interventional, and Treatment
Among 15 studies in Cardiomyopathy, Dilated with a site in Netherlands — 3 open to participants now, 4 under way, 2 of them phase 3. Cardiomyopathy, Dilated · Netherlands.
Among the 15 studies in Cardiomyopathy, Dilated with a site in Netherlands, this one is the 10th largest by planned enrollment of the 11 interventional ones, is the only one that started in 2025, is the only one in phase 1, and was the 14th registered.
255 studies on the registry are indexed under Cardiomyopathy, Dilated; 66 are open to participants now.
This study's planned enrollment of 23 is below the median of 43 across 145 interventional studies indexed under Cardiomyopathy, Dilated.
Browse Cardiomyopathy, Dilated studies →3 of the 15 studies in Cardiomyopathy, Dilated with a site in Netherlands are open now, in 11 countries. Each count lists the studies.
Of the 53 Cardiomyopathy, Dilated studies recruiting now, 30 (57%) set no upper age limit and 15 (28%) stop at 75 or younger; 13 take children under 18; 9 take healthy volunteers.
This study: 18 Years to 80 Years · all sexes · no healthy volunteers.
The full table on the hub →15 studies; the count open now beside each. This study's row is marked.
First in 2010; this study’s year is marked. 1 started in 2025.
Median enrollment 77 across the 11 interventional studies, 97 across the 4 observational. Of the 7 that completed or were terminated, 2 (29%) have results posted.
10 of 15 have an industry lead sponsor. The 10 most frequent:
AstraZeneca is the lead sponsor of 3,499 studies on the registry; 270 are open to participants now.
Of its 368 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
AstraZeneca has 3 studies filed under Cardiomyopathy, Dilated — fewer than ten, so there is no comparison to draw.
3 of the studies in Cardiomyopathy, Dilated with a site in Netherlands that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07210723Phase 3Kardigan, Inc.A Study of the Efficacy and Safety of Danicamtiv in Participants With Symptomatic Genetic and Familial Dilated Cardiomyopathy | 2027-09 |
| NCT05981092Alexion Pharmaceuticals, Inc.A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM) | 2027-08-12 |
| NCT07241104AstraZenecaA Study of AZD4063 in PLN R14del Dilated Cardiomyopathy | 2027-11-22 |
Among the 15 studies in Cardiomyopathy, Dilated with a site in Netherlands, from their registry updates:
Nothing recorded among them since 2026-09-25.
The purpose of the study is to assess the safety, tolerability and the pharmacokinetics (PK) of AZD4063 after single dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.
This is a Phase 1, first in human, unblinded, ascending dose study which will consist of a SAD (single ascending dose) part and an Optional part.
The SAD part of the study will assess the single doses of AZD4063 across 4 cohorts. It will consist of:
Optional cohorts may be added based on emerging safety, PK and PD data of the preceding cohorts.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Any laboratory values with the following deviations:
Any vital sign values with the following deviations at Screening
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive Dose 1 of AZD4063 via SC injection in Cohort 1 of SAD.
Drug: AZD4063
Participants will receive Dose 2 of AZD4063 via SC injection in Cohort 2 of SAD.
Drug: AZD4063
Participants will receive Dose 3 of AZD4063 via SC injection in Cohort 3 of SAD.
Drug: AZD4063
Participants will receive Dose 4 of AZD4063 via SC injection in Cohort 4 of SAD.
Drug: AZD4063
Participants will receive Dose 5 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Drug: AZD4063
Participants will receive Dose 6 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Drug: AZD4063
Participants will receive Dose 7 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Drug: AZD4063
Participants will receive Dose 8 of AZD4063 via SC injection in the optional cohort of the study. This additional cohort will be added depending on the findings.
Drug: AZD4063
AZD4063 will be administered as solution for injection via subcutaneous route of administration.
Number of participants with adverse events (AEs)
The safety and tolerability of AZD4063 following the SC administration of single doses in participants with PLN R14del dilated cardiomyopathy will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210
Area under plasma concentration-time curve from 0 to infinity (AUCinf)
The AUCinf of single ascending doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210
Area under the plasma concentration-curve from 0 to the last quantifiable concentration (AUClast)
The AUClast of single doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210
Maximum plasma drug concentration (Cmax)
The Cmax of single doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 80; Cohorts 3 and 4: Day 1 to 210
Renal clearance (CLR)
The CLR of the single doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)
Cumulative amount of analyte excreted (Ae)
The Ae of single doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)
Fraction of dose excreted unchanged in urine (Fe)
The Fe of single doses of AZD4063 following SC administration will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)
Change in endomyocardial biopsy PLN messenger ribonucleic acid (mRNA levels) from baseline to estimated peak knockdown and estimated return-to-baseline
The effects of single ascending doses of AZD4063 on knockdown of PLN mRNA will be evaluated.
Time frame: Cohorts 1 and 2: Day 1 to 94; Cohorts 3 and 4: Day 1 to Follow up (Day 224)
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Time frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Data sharing details ↗ · vivli.org
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for Nov 2027, 23 months after the start.
How these figures are worked out, and which studies are left out of them.
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