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Advanced Solid Tumors That Are MTAP DeficientPhase 1/2PRIMROSENCT06130553

A Study of AZD3470, a PRMT5 Inhibitor, Given as Monotherapy and in Combination in Patients With MTAP Deficient Advanced/Metastatic Solid Tumors

A Phase 1/2 study testing AZD3470 and Datopotamab deruxtecan in people with Advanced Solid Tumors That Are MTAP Deficient, run by AstraZeneca at 21 sites in 8 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This is a first time in human (FTiH) Phase I/IIa, open-label, multi-centre study of AZD3470 in participants with advanced or metastatic solid tumors with MTAP deficiency. The study consists of several study modules, evaluating the safety, tolerability, pharmacokinetic (PK), pharmacodynamics, and preliminary efficacy… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
AZD3470 and Datopotamab deruxtecan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
334 participants
Where
21 sitesin 8 countries
Study dates
18 Jan 2024to 4 Dec 2028
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Inclusion Criteria (All Modules) Participants are ≥ 18 years (or the legal age of consent in the jurisdiction) at the time of signing the informed consent form.

Participants are able to provide written informed consent and are willing and able to comply with study procedures.

Participants are willing to provide archival and/or newly obtained (baseline) tumor tissue for central testing, including required biomarker assessment(s) (and any module-specific biomarker requirements).

Participants have tumors meeting the protocol-defined MTAP-deficiency requirement, based on acceptable prior testing and/or central testing per protocol.

Participants have received prior systemic therapy appropriate for the tumor type and disease stage and have disease progression on or after prior therapy; participants must have had ≥ 1 prior line of systemic treatment in the recurrent/metastatic (advanced) setting.

Participants have ECOG performance status 0-1. Participants have life expectancy ≥ 12 weeks, in the opinion of the Investigator.

Participants have measurable disease per RECIST v1.1. Participants have adequate organ and bone marrow function per protocol-defined laboratory/assessment criteria.

Participants have a treatment-free interval ≥ 3 weeks from prior anticancer therapy before starting study drug (with any additional protocol-defined washout requirements for certain therapies/procedures).

Contraception use by men and women is consistent with local regulations and protocol-defined requirements.

Additional Inclusion Criteria (Module 2: Non-squamous NSCLC) Participants have histologically or cytologically confirmed non-squamous NSCLC, Stage IIIB/IIIC not amenable to curative therapy or Stage IV.

Participants have documented radiographic extracranial disease progression while on or after the most recent treatment regimen for advanced/metastatic NSCLC (CNS-only progression is not eligible).

NSCLC of mixed histology is allowed if not predominantly squamous; no small cell or large cell neuroendocrine components.

Participants meet one of the following:

Tumor has a documented EGFR alteration eligible for EGFR-directed therapy (per protocol-defined criteria) and the participant has received prior systemic therapy appropriate for EGFR-altered advanced/metastatic NSCLC (per protocol), OR Tumor is negative for EGFR alterations eligible for EGFR-directed therapy, has no other known actionable genomic alterations for which locally approved/available targeted therapies exist (per protocol-defined criteria), meets any additional protocol-required biomarker criteria for this cohort (as applicable), and the participant has received prior systemic therapy appropriate for non-actionable-alteration advanced/metastatic NSCLC (per protocol).

Exclusion Criteria (All Modules) Participants have spinal cord compression, or symptomatic and unstable brain metastases, leptomeningeal disease, or primary CNS malignancy. Participants with asymptomatic, radiographically stable brain metastases who do not require steroids (or who have completed definitive therapy and are neurologically stable off steroids, per protocol) may be eligible.

Participants have a history of allogeneic organ transplantation. Participants have any clinically significant abnormal laboratory finding or severe and uncontrolled medical condition that, in the Investigator's opinion, makes participation unsafe, including active infection requiring systemic treatment.

Participants have clinically significant cardiovascular disease or risk factors (including reduced LVEF, cardiomyopathy, clinically active cardiovascular disease, recent major ischemic events or revascularization procedures, uncontrolled angina, severe valvular disease, uncontrolled hypertension, clinically significant heart failure, or recent stroke/TA clinically significant ECG abnormalities, prolonged QTc, or conditions/medications that increase risk of QTc prolongation or arrhythmic events)..

Participants require therapeutic anticoagulation for treatment of acute thromboembolic events, per protocol.

Participants have active hepatitis B or hepatitis C infection (including detectable viral load, per protocol-defined testing).

Participants have known HIV infection. Participants have current ILD/pneumonitis, or a history of (non-infectious) ILD/pneumonitis requiring systemic steroids or supplemental oxygen, or suspected ILD/pneumonitis that cannot be ruled out by screening imaging.

Participants have active gastrointestinal disease, malabsorption, or other GI condition/surgery that would significantly interfere with oral drug absorption or tolerability.

Participants have a history of another primary malignancy. Participants have unresolved clinically significant toxicity from prior anticancer therapy (typically Grade ≥ 2).

Participants have had prior treatment with a PRMT5 inhibitor Participants are pregnant, breastfeeding, or intend to become pregnant during study participation.

Additional Exclusion Criteria (Module 2 Only) Participants have inaccessible veins and/or inability to place required venous access (e.g., port), per Investigator judgment.

Participants have contraindication to required CNS imaging (brain MRI preferred or CT with contrast).

Participants have clinically significant corneal disease. Participants have known active tuberculosis infection, per clinical evaluation and local practice.

Participants have significant third-space fluid (e.g., pleural effusion/ascites) not amenable to required repeated drainage, per Investigator judgment.

Participants have severe pulmonary function compromise due to intercurrent pulmonary illness (e.g., severe COPD/asthma/restrictive lung disease, recent pulmonary embolism), per protocol.

Participants have recent radiotherapy that does not meet protocol-defined washout requirements and/or ongoing radiation-related toxicities requiring corticosteroids.

Participants have had prior treatment with protocol-prohibited anticancer therapies.

Trial locations and contacts

21 sites in 8 countries. Contact the study team or a site near you to ask about taking part.

AstraZeneca Clinical Study Information Center contact

1-877-240-9479

information.center@astrazeneca.com

21 sites in 8 countries

United States8 sites, 7 recruiting
  • Research Site

    Baltimore, Maryland, 21231

    Recruiting
  • Research Site

    Fairfax, Virginia, 22031

    Recruiting
  • Research Site

    New Haven, Connecticut, 06510

    Recruiting
  • Research Site

    Pittsburgh, Pennsylvania, 15232

    Recruiting
  • Research Site

    Portland, Oregon, 97239

    Recruiting
  • Research Site

    Providence, Rhode Island, 02903

    Recruiting
  • Research Site

    San Francisco, California, 94143

    Recruiting
  • Research Site

    West Hollywood, California, 90048

    Withdrawn

Other trials in United States

China4 sites, 4 recruiting
  • Research Site

    Beijing, 100142

    Recruiting
  • Research Site

    Chengdu, 610041

    Recruiting
  • Research Site

    Shanghai, 200433

    Recruiting
  • Research Site

    Wuhan, 430030

    Not yet recruiting

Other trials in China

Japan2 sites, 2 recruiting
  • Research Site

    Chūōku, 104-0045

    Recruiting
  • Research Site

    Kashiwa, 227-8577

    Recruiting

Other trials in Japan

South Korea2 sites, 2 recruiting
  • Research Site

    Seoul, 03080

    Recruiting
  • Research Site

    Seoul, 06351

    Recruiting

Other trials in South Korea

Spain2 sites, 2 recruiting
  • Research Site

    Barcelona, 8035

    Recruiting
  • Research Site

    Madrid, 28027

    Recruiting

Other trials in Spain

Australia1 site, 1 recruiting
  • Research Site

    Melbourne, 3000

    Recruiting

Other trials in Australia

France1 site, 1 recruiting
  • Research Site

    Villejuif, 94805

    Not yet recruiting

Other trials in France

Netherlands1 site, 1 recruiting
  • Research Site

    Amsterdam, 1066CX

    Recruiting

Other trials in Netherlands

About this study

Official title: PRIMROSE: A Modular Phase I/IIa, Multi-centre, Dose Escalation, and Expansion Study of AZD3470, a MTA Cooperative PRMT5 Inhibitor, as Monotherapy and in Combination With Anticancer Agents in Participants With Advanced/Metastatic Solid Tumors That Are MTAP Deficient

This is a first time in human (FTiH) Phase I/IIa, open-label, multi-centre study of AZD3470 in participants with advanced or metastatic solid tumors with MTAP deficiency. The study consists of several study modules, evaluating the safety, tolerability, pharmacokinetic (PK), pharmacodynamics, and preliminary efficacy of AZD3470 as monotherapy or in combination with other anti-cancer agents.

Read the detailed description

This first time in human, open-label, multi-centre study of AZD3470 in participants with advanced or metastatic solid tumors with MTAP deficiency follows a modular design. Module 1 Part A will include the dose escalation cohorts. Part B will include the dose optimization and expansion cohorts. The purpose of the Phase 2 Module 2 is to evaluate the efficacy and safety of AZD3470 in combination with Dato-DXd versus Dato-DXd alone - dose optimization and expansion. New modules for combination treatments may be added in the future based on emerging data.

Being tested
AZD3470, Datopotamab deruxtecan
Keywords
solid tumor, MTAP deficient

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AZD3470Drug

    AZD3470 is a novel, potent and selective, second-generation, MTAP-selective, inhibitor of PRMT5.

  • Datopotamab deruxtecanDrug

    Also called Dato-DXd

    AZD3470 in combination with Dato-DXd + Dato-Dxd monotherapy

Study groups in NCT06130553
Study groupRoleWhat they receive
Module 1: AZD3470 MonotherapyExperimentalPart A dose escalation and back-fill cohorts and Part B dose optimization and expansion cohorts of varying doses of AZD3470
Receives: Drug: AZD3470
Module 2: AZD3470 in combination with Dato-DXdExperimentalVarying doses of AZD3470 in combination with Dato-Dxd
Receives: Drug: AZD3470; Drug: Datopotamab deruxtecan
Module 2: Dato-DXd aloneExperimentalControl arm
Receives: Drug: Datopotamab deruxtecan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. All Modules: Incidence of adverse events (AEs) and serious adverse events (SAEs). To determine the RP2D of AZD3470 as monotherapy and in combination with anticancer agents

    Number of participants with AEs and SAEs.

    Measured: From time of informed consent to 28 days post last dose of study treatment

  2. Module 1: Incidence of dose-limiting toxicities (DLT)

    Incidence of dose-limiting toxicities (DLT) as determined by number of patients with at least 1 dose-limiting toxicity (DLT)

    Measured: From first dose of study treatment until the end of Cycle 1 (each cycle is 21 days)

  3. Module 2: Progression Free Survival assessed by the Investigator according to RECIST v1.1

    PFS - defined as time from date of randomization until progression per RECIST v1.1 as assessed by the Investigator at local site, or death due to any cause.

    Measured: From date of randomization up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

Secondary outcomes (19)
  1. All modules: Radiological response assessed by the Investigator evaluated according to RECIST v1.1 - ORR (Objective Response Rate)

    Proportion of participants who have a complete or partial radiological response as determined by the Investigator according to RECIST v1.1

    Measured: From date of first dose of AZD3470 up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

  2. All modules: Radiological response assessed by the Investigator evaluated according to RECIST v1.1 - DoR (Duration of Response)

    DoR - the time from date of first documented objective response until date of documented disease progression per Tumor RECIST v1.1 as assessed by the Investigator at local site or death due to any cause.

    Measured: From date of first dose of AZD3470 up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

  3. All modules: Radiological response assessed by the Investigator evaluated according to RECIST v1.1 - Best percentage change in tumor size

    Best percentage change from baseline in TL (target lesion) tumor size is based on the RECIST 1.1. TL measurements as assessed by the Investigator.

    Measured: From date of first dose of AZD3470 up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

  4. Module 1: Progression Free Survival assessed by the Investigator evaluated according to RECIST v1.1

    PFS - defined as time from date of first dose (nonrandomized study parts) or date of randomization (randomized study parts) until progression per RECIST v1.1 as assessed by the Investigator at local site, or death due to any cause.

    Measured: From date of first dose/randomization up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

  5. All modules: Radiological response assessed by the Investigator evaluated according to RECIST v1.1 - DCR (Disease Control Rate) at 12 weeks

    DCR at 12 weeks defined as the percentage of participants who have a CR (complete response) or PR (partial response) or who have SD (stable disease) per RECIST 1.1 as assessed by the Investigator at local site and derived from the raw tumor data for at least 11 weeks after date of first dose to allow for an early assessment within the assessment window.

    Measured: From date of first dose of AZD3470 up until progression, or the last evaluable assessment in the absence of progression (for each patient this is expected to be measured at 12 weeks).

  6. All modules: Overall Survival (OS)

    Overall Survival (OS) - defined as time from date of first dose (nonrandomized study parts) or date of randomization (randomized study parts) until the date of death due to any cause.

    Measured: From date of first dose of AZD3470 up until the date of death due to any cause (approximately 2 years).

  7. Module 1 Endpoints Part A (Dose escalation) Measurement of PK parameters: AUC

    Part A (Dose escalation) Measurement of PK parameters: Area under the concentration time curve (AUC).

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  8. Module 1 Endpoints Part A (Dose escalation) Measurement of PK parameters: C-max

    Part A (Dose escalation) Measurement of PK parameters: Maximum observed plasma concentration of the study drug (C-max).

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  9. Module 1 Endpoints Part A (Dose escalation) Measurement of PK parameters: half life

    Part A (Dose escalation) Measurement of PK parameters: Terminal elimination half-life (t 1/2)

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  10. Module 1 Endpoints Part A (Dose escalation) Measurement of PK parameters: Ae (excreted in urine)

    Part A (Dose escalation) Measurement of PK parameters: amount of AZD3470 excreted in urine (Ae).

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  11. Module 1 Endpoints Part A (Dose escalation) Measurement of PK parameters: Clr (renal clearance)

    Part A (Dose escalation) Measurement of PK parameters: renal clearance (Clr).

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  12. Module 1 Endpoints Part A drug-drug interaction (DDI) - Measurement of PK parameters of Midazolam: Cmax

    Part A (DDI)- Plasma geometric mean ratio (Maximum observed plasma concentration of the study drug (C-max)) of Midazolam evaluated with and without AZD3470

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  13. Module 1 Endpoints Part A (DDI) - Measurement of PK parameters of Midazolam: AUC

    Part A (DDI) - Plasma geometric mean ratio (Area under the concentration time curve (AUC)) of Midazolam evaluated with and without AZD3470

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  14. Module 1 Endpoints Part A (DDI) - Measurement of PK parameters of Dextromethorphan: Cmax

    Part A (DDI)- Plasma geometric mean ratio (Maximum observed plasma concentration of the study drug (C-max)) of Dextromethorphan evaluated with and without AZD3470

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  15. Module 1 Endpoints Part A (DDI) - Measurement of PK parameters of Dextromethorphan: AUC

    Part A (DDI) - Plasma geometric mean ratio (Area under the concentration time curve (AUC)) of Dextromethorphan evaluated with and without AZD3470

    Measured: At predefined intervals throughout the treatment period (for each patient this is expected to be measured up to approximately 4 weeks)

  16. Module 1 Endpoints Part A pharmacodynamic backfill cohorts - Measurement of SDMA in tumor.

    Part A pharmacodynamic backfill- Percentage change from baseline tumor SDMA as measured by immunohistochemistry.

    Measured: From screening baseline timepoint to up to four weeks on treatment timepoint.

  17. Module 2: Time to response (TTR)

    The time from date of randomization until the date of first documented objective response, per RECIST 1.1. as assessed by the investigator

    Measured: From randomization until the date of first documented objective response, per RECIST v1.1 as assessed by the investigator

  18. Module 2: PK parameters of AZD3470 and Dato-DXd

    Measurement of PK parameters:

    • Maximum total plasma concentration Cmax
    • Time to maximum concentration Tmax
    • Area under total plasma concentration curve AUC

    Measured: From C1D1 until EOT/28 Day Follow Up

  19. Module 2: Presence of ADAs (anti-drug antibody) for Dato-DXd

    To evaluate the immunogenicity of Dato-DXd Presence of ADAs (anti-drug antibody) for Dato-DXd

    Measured: From C1D1 until EOT/28 Day Follow Up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Nov 2023
  2. Study start18 Jan 2024
  3. Primary completion4 Dec 2028 (estimated)
  4. Study completion4 Dec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
This is a FTiH (first time in human), modular Phase I/IIa, open-label, multi-centre study of AZD3470 as Monotherapy and in Combination with Anti-cancer agents, in participants with MTAP deficient advanced/metastatic solid tumors. The study consists of individual modules, each evaluating safety and tolerability of AZD3470 dosed as a monotherapy or in combination with specific treatments. Module 1 describes AZD3470 monotherapy, and will have at least two parts. Part A consisting of dose escalation cohorts and Part B consisting of optimization and expansion cohorts. Module 2 describes AZD3470 in combination with Dato-DXd and will consist of cohorts of AZD3470 in combination with Dato-DXd and a cohort of Dato-DXd as monotherapy
Masking
None (open label)
Enrolment
334 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06130553
Sponsor's protocol ID
D9970C00001
Other IDs
165618
Acronym
PRIMROSE
Status last verified
Aug 2026
First submitted
19 Oct 2023
Last updated
27 Aug 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request 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.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AZD3470 and Datopotamab deruxtecan in people with Advanced Solid Tumors That Are MTAP Deficient, run by AstraZeneca at 21 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in United States, China, Japan, South Korea, Spain and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

All Modules: Incidence of adverse events (AEs) and serious adverse events (SAEs). To determine the RP2D of AZD3470 as monotherapy and in combination with anticancer agents (time frame: From time of informed consent to 28 days post last dose of study treatment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026.

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