A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors

NCT ID: NCT04657068

Last Updated: 2026-01-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

502 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-12-13

Study Completion Date

2026-12-31

Brief Summary

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This clinical trial is evaluating a drug called ART0380 in participants with advanced or metastatic solid tumors. The main goals of this study are to:

* Find the recommended dose of ART0380 that can be given safely to participants alone and in combination with gemcitabine or irinotecan
* Learn more about the side effects of ART0380 alone and in combination with gemcitabine or irinotecan
* Learn more about the effectiveness of ART0380 alone and in combination with gemcitabine or irinotecan

Detailed Description

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ART0380 is a new investigational medicinal product that is a potent and selective inhibitor of Ataxia telangiectasia and Rad3-related (ATR). ART0380 is being developed as an oral anti-cancer agent for the treatment of participants with cancers that harbor defects in deoxyribonucleic acid (DNA) repair and in combination with agents including those that cause DNA damage.

This study is an open-label Phase I/IIa study designed to evaluate the safety, tolerability, PK and preliminary efficacy of ART0380 as monotherapy or in combination with gemcitabine or irinotecan in participants with advanced or metastatic solid tumors, advanced or solid tumors that fail to express Ataxia-Telangiectasia Mutated protein kinase (ATM) by immunohistochemistry, and high grade serous ovarian, primary peritoneal or fallopian tube carcinoma.

Conditions

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Advanced Cancer Metastatic Cancer Ovarian Cancer Primary Peritoneal Cancer Fallopian Tube Cancer Endometrial Cancer Metastatic Colorectal Cancer Pancreatic Ductal Adenocarcinoma Acinar Cell Carcinoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part A1

Part A1 evaluated intermittent and continuous dosing of ART0380 monotherapy. Treatment was given in 21-day cycles.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Part A2

Part A2 evaluated intermittent dosing of ART0380 in combination with gemcitabine in 21-day cycles.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Gemcitabine

Intervention Type DRUG

Gemcitabine was administered on Days 1 and 8 of a 21-day cycle.

Part A3

Part A3 evaluated intermittent dosing of ART0380 in combination with irinotecan in 21-day cycles.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Part B1

In Part B1, up to 7 cohorts enrolled participants with solid cancers with alterations in the ATM (ataxia-telangiectasia mutated) gene likely to predict for loss of ATM protein will be treated with either

* ART0380 monotherapy Or
* ART0380 in combination with irinotecan

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Part B2

In Part B2, participants with high grade serous ovarian, primary peritoneal, or fallopian tube carcinoma were randomized (open label) 1:1 to either ART0380 in combination with gemcitabine or gemcitabine alone.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Gemcitabine

Intervention Type DRUG

Gemcitabine was administered on Days 1 and 8 of a 21-day cycle.

Part B3

in Part B3, participants with persistent or recurrent endometrial cancer (EC) received ART0380 monotherapy on either a continuous daily dose or on an intermittent schedule for a 21-day cycle.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Part B4

In Part B4, participants with advanced or metastatic solid tumors received ART0380 monotherapy on either a continuous daily dose or on an intermittent schedule for a 21-day cycle.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Part B5

In Part B5, participants with colorectal cancer (CRC) will receive ART0380 in combination with irinotecan on a 21-day cycle.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Part B6

In Part B6, participants with pancreatic ductal adenocarcinoma (PDAC) or acinar cell carcinoma will receive ART0380 in combination with irinotecan on a 21-day cycle.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Part A3 Fed/Fast

Part A3 Fed/Fast will evaluate intermittent dosing of ART0380 in combination with irinotecan in 21-day cycles in a fasting or fed state.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Part B7

In Part B7, participants with colorectal cancer (CRC) will receive ART0380 in combination with irinotecan on a 21-day cycle.

Group Type EXPERIMENTAL

ART0380

Intervention Type DRUG

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Irinotecan

Intervention Type DRUG

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Interventions

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ART0380

Participants will receive ART0380 by mouth either intermittently (either once daily 3 days on, 4 days off; days 2-4 and 9-11;or days 1-3 and 8-10) or continuously (once daily each day) in 21 day cycles.

Intervention Type DRUG

Gemcitabine

Gemcitabine was administered on Days 1 and 8 of a 21-day cycle.

Intervention Type DRUG

Irinotecan

Irinotecan will be administered as a 90-minute infusion on Days 1 and 8 of a 21 day cycle.

Intervention Type DRUG

Other Intervention Names

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Gemzar Camptosar Camptothecin-11

Eligibility Criteria

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Inclusion Criteria

* Signed informed consent
* Discontinued all previous treatments for cancer for at least 21 days or 5 half-lives, whichever is shorter, and recovered from the acute effects of therapy to CTCAE Grade ≤1. Palliative radiotherapy must have completed 1 week prior to start of study treatment.
* If patients have a known germline BRCA mutation or a cancer with a somatic BRCA mutations or which is HRD positive and for which there is an approved PARP inhibitor, participants should have received such treatment before participating in the study unless contra-indicated
* At least 1 radiologically evaluable lesion (measurable and/or non-measurable) that can be assessed at baseline and is suitable for repeated radiological evaluation by RECIST v1.1 or Prostate Cancer Working Group-3 Guidelines (PCWG-3)
* Acceptable hematologic, renal, hepatic, and coagulation functions independent of transfusions and granulocyte colony-stimulating factor
* Non-irradiated tumor tissue sample (archival or newly obtained core biopsy of a tumor lesion) available for submission for analysis.
* Female patients of childbearing potential and male patients with female partners of childbearing potential are required to use highly effective contraception plus one barrier method during their participation in the study and for 7 months and 5 months respectively following the last dose. For male and female patients given gemcitabine or irinotecan, highly effective contraception plus one barrier method must be used from study entry until 6 months after the last dose of study treatment. Male patients are required to refrain from donating sperm and female patients are required to refrain from donating eggs, during their participation in the study and for 6 months following last dose.
* Estimated life expectancy of ≥12 weeks
* Reliable and willing to make themselves available for the duration of the study and are willing to follow study procedures
* Performance status of 0-1 on the ECOG Scale


* Advanced or metastatic cancer which is refractory to standard therapies, or for which no standard therapies exist, or for which the investigator feels no other active therapy is required for the duration of the study
* Performance status of 0-1 on the Eastern Cooperative Oncology Group (ECOG) scale


•Advanced or metastatic cancer for which gemcitabine is an appropriate treatment. Prior treatment with gemcitabine is permitted.


* Advanced or metastatic cancer for which irinotecan is an appropriate treatment. Prior treatment with irinotecan is permitted.
* For food effect cohort only: Patients must be able to eat a high-fat meal within a 30 minute period, as provided by the study site.


* Patients with advanced or metastatic solid tumors with alterations to the ATM gene likely to predict for loss of ATM protein
* Have at least 1 measurable lesion assessable using standard techniques by RECIST v1.1
* For France only ART0380 Monotherapy; Patient that is not eligible for curative treatment, for whom all standard of care therapies have failed and no therapies known to provide clinical benefit are available.
* Combination arms; Patients for which irinotecan is an appropriate treatment. Prior treatment with irinotecan is permitted.
* For Spain only ART0380 Combination therapy, Patient that is not eligible for curative treatment, for whom standard of care therapies have failed.


* Patients with a known germline BRCA mutation, or a cancer with a known somatic BRCA mutation, or which is known to be HRD positive, and for which there is an approved PARP inhibitor should have received such treatment before participating in the study, unless contra-indicated.
* Females with histologically-confirmed diagnosis of high grade serous carcinoma of the ovary, fallopian tube or primary peritoneum that is not amenable to curative therapy
* Platinum-resistant disease, defined as disease progression within 6 months of last receipt of platinum-based chemotherapy. Patients must not have had primary platinum-refractory disease (disease that progressed during first-line platinum-based therapy).
* No more than one prior regimen in the platinum-resistant setting. Hormonal therapies and antiangiogenic therapies (as single agents) and PARP inhibitors used as maintenance therapy are not considered as separate lines of therapy. Patients should have previously received bevacizumab and chemotherapy unless contra-indicated.
* Have not received prior treatment with gemcitabine unless administered in combination with a platinum with no disease progression within 12 months after completion of that regimen
* Have at least 1 measurable lesion assessable using standard techniques by RECIST v1.1


* Persistent or recurrent endometrial cancer with biological selection.:
* Patients should have received taxane/platinum chemotherapy, unless contraindicated.
* Measurable disease.


* Advanced or metastatic solid cancers of any histology with biological selection
* If a PD-1/PDL-1 inhibitor (eg, pembrolizumab) is approved and available for the patient's cancer, the patient should have received such treatment before participating in this study.
* Radiologically evaluable disease
* Performance status of 0-1 on the ECOG scale


* Metastatic CRC with alterations to the ATM gene
* Participants should have previously received appropriate prior lines of therapy in this setting.
* Have at least 1 measurable lesion assessable using standard techniques by RECIST v1.1.
* Part B5 (3L) only:
* Patients must have received a maximum of exactly 2 prior chemotherapy regimens for the treatment of CRC which must have included a fluoropyrimidine, oxaliplatin and irinotecan unless contraindicated or unavailable
* Part B7 (2L) only:
* Patients must have received exactly 1 prior chemotherapy regimen for the treatment of CRC which must have included a fluoropyrimidine unless contraindicated or unavailable


* Metastatic or locally advanced PDAC or acinar cell carcinoma with alterations to the ATM gene
* Participants should have previously received prior lines of therapy in this setting
* Have at least 1 measurable lesion assessable using standard techniques by RECIST v1.1
* Serum albumin ≥3g/dL within 7 days prior to first dose.

Exclusion Criteria

* Women who are pregnant, breast feeding, or who plan to become pregnant while in the study or within 7 months after the last administration of study treatment
* Men who plan to father a child while in the study or within5 months after the last administration of study treatment
* Serious concomitant systemic disorder that would compromise the participants ability to adhere to the protocol including: one or more opportunistic HIV/AIDs-related infections within the past 12 months, a known hepatitis B virus, or known hepatitis C virus; documented active or chronic tuberculosis infection; malignancy prior to the one currently being treated that is not in remission
* Have ongoing interstitial lung disease or pneumonitis (whether symptomatic or asymptomatic).
* Moderate or severe cardiovascular disease
* Valvulopathy that is severe, moderate, or deemed clinically significant
* Documented major electrocardiogram (ECG) abnormalities which are clinically significant
* Symptomatic or uncontrolled brain metastases, spinal cord compression, or leptomeningeal disease requiring concurrent treatment
* Received a live vaccine within 30 days before the first dose of study treatment
* History or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate
* Recent major surgery within 4 weeks prior to entry into the study or minor surgery within 1 week of entry into the study
* Significant bleeding disorder or vasculitis or had a Grade ≥3 bleeding episode within 12 weeks prior to enrollment
* Currently enrolled in a clinical trial involving an investigational product or any other type of medical research judged not to be scientifically or medically compatible with this study


* Patients who have symptoms or signs of clinically unacceptable deterioration of the primary disease at the time of screening.
* Patients receiving inhibitors of UGT1A1 within 2 weeks before the first dose of study treatment
* Part A3 Fed-fasted cohort only: Patients receiving acid reducing agents within 1 week before the first dose of study treatment will be excluded
* Part B6 only: Neuroendocrine (carcinoid, islet cell) or adenosquamous carcinoma pancreatic cancer
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Artios Pharma Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Melissa Johnson, MD

Role: STUDY_CHAIR

Tennessee Oncology

Antonio Gonzalez, MD, PHD

Role: STUDY_CHAIR

Clinica Universidad de Navarra, Madrid

Susanna Ulahannan, MD

Role: PRINCIPAL_INVESTIGATOR

Oklahoma University

Kim Reiss Binder, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pennsylvania / Abramson Cancer Center

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status RECRUITING

Mayo Clinic (Arizona)

Scottsdale, Arizona, United States

Site Status RECRUITING

University of Arkansas - Winthrop P. Rockefeller Cancer Institute

Little Rock, Arkansas, United States

Site Status RECRUITING

USC Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status RECRUITING

Sansum Clinic

Santa Barbara, California, United States

Site Status RECRUITING

Providence Medical Foundation

Santa Rosa, California, United States

Site Status RECRUITING

Rocky Mountain Cancer Center

Denver, Colorado, United States

Site Status RECRUITING

Sarah Cannon Research Institute at HealthONE

Denver, Colorado, United States

Site Status RECRUITING

Florida Cancer Specialists

Fort Myers, Florida, United States

Site Status RECRUITING

Mayo Clinic (Florida)

Jacksonville, Florida, United States

Site Status RECRUITING

Cancer Specialists of North Florida

Jacksonville, Florida, United States

Site Status RECRUITING

Florida Cancer Specialists

Orlando, Florida, United States

Site Status COMPLETED

Florida Cancer Specialists

Sarasota, Florida, United States

Site Status RECRUITING

Florida Cancer Specialists

West Palm Beach, Florida, United States

Site Status RECRUITING

Hope and Healing Cancer Services

Hinsdale, Illinois, United States

Site Status RECRUITING

Community Health Network

Indianapolis, Indiana, United States

Site Status RECRUITING

Our Lady of the Lake

Baton Rouge, Louisiana, United States

Site Status RECRUITING

Maryland Oncology Hematology - Primary

Columbia, Maryland, United States

Site Status RECRUITING

Minnesota Oncology Hematology

Maple Grove, Minnesota, United States

Site Status RECRUITING

Mayo Clinic (Minnesota)

Rochester, Minnesota, United States

Site Status RECRUITING

Washington University

St Louis, Missouri, United States

Site Status RECRUITING

Hematology Oncology Associates of Central New York

East Syracuse, New York, United States

Site Status RECRUITING

Northwell Health Cancer Institute

Lake Success, New York, United States

Site Status RECRUITING

Oncology Hematology Care Primary

Cincinnati, Ohio, United States

Site Status RECRUITING

Taylor Cancer Research Center

Maumee, Ohio, United States

Site Status RECRUITING

Stephenson Cancer Center

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Oregon Health & Science University

Portland, Oregon, United States

Site Status RECRUITING

University of Pennsylvania / Abramson Cancer Center

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Thomas Jefferson University, Sidney Kimmel Cancer Center, Clinical Research Organization

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Tennessee Oncology, PLLC

Chattanooga, Tennessee, United States

Site Status RECRUITING

Baptist Cancer Center

Memphis, Tennessee, United States

Site Status COMPLETED

SCRI Oncology Partners

Nashville, Tennessee, United States

Site Status RECRUITING

Texas Oncology - Central/South Texas

Austin, Texas, United States

Site Status RECRUITING

Mary Crowley Cancer Research

Dallas, Texas, United States

Site Status RECRUITING

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, United States

Site Status RECRUITING

Texas Oncology - Northeast Texas

Flower Mound, Texas, United States

Site Status RECRUITING

Oncology Consultants

Houston, Texas, United States

Site Status COMPLETED

Texas Oncology - San Antonio

San Antonio, Texas, United States

Site Status RECRUITING

Utah Cancer Specialists

Salt Lake City, Utah, United States

Site Status RECRUITING

Virginia Cancer Specialists

Fairfax, Virginia, United States

Site Status RECRUITING

Institut Gustave Roussy

Villejuif, Cedex, France

Site Status RECRUITING

Institut Bergonie

Bordeau, , France

Site Status RECRUITING

Hospital General Universitario de Elche

Elche, Alicante, Spain

Site Status RECRUITING

H. Parc Tauli

Sabadell, Barcelona, Spain

Site Status RECRUITING

Next Oncology Barcelona, IOB

Barcelona, Catalonia, Spain

Site Status RECRUITING

Hospital Arnau de Vilanova

Lleida, Catalonia, Spain

Site Status RECRUITING

Hospital Universitario La Paz

Madrid, Madrid, Spain

Site Status RECRUITING

Next - Hospital Quironsalud Madrid

Pozuelo de Alarcón, Madrid, Spain

Site Status RECRUITING

Hospital Clínico Universitario Virgen de la Arrixaca

El Palmar, Murcia, Spain

Site Status RECRUITING

Clínica Universidad de Navarra

Madrid, Planta -2, Spain

Site Status RECRUITING

START Rioja

Rioja, Spain, Spain

Site Status RECRUITING

Instituto Valenciano de Oncología (IVO)

Valencia, Spain, Spain

Site Status RECRUITING

Hospital Teresa Herrera (CHUAC)

A Coruña, , Spain

Site Status RECRUITING

Institut Català d'Oncologia Badalona - Hospital Germans Trias i Pujol

Badalona, , Spain

Site Status RECRUITING

Vall d'Hebron Institute of Oncology (VIHO)

Barcelona, , Spain

Site Status RECRUITING

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status RECRUITING

ICO Hospitalet

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitario Reina Sofia de Córdoba

Córdoba, , Spain

Site Status RECRUITING

Hospital Universitari Doctor Josep Trueta- ICO de Girona

Girona, , Spain

Site Status RECRUITING

Hospital General Universitario Gregorio Marañón

Madrid, , Spain

Site Status RECRUITING

MD Anderson Cancer Center (Madrid

Madrid, , Spain

Site Status RECRUITING

Hospital Clinico San Carlos

Madrid, , Spain

Site Status RECRUITING

START Madrid Fundacion Jimenez Diaz

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status RECRUITING

START Madrid (Hospital San Chinarro)

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario Virgen de la Victoria

Málaga, , Spain

Site Status RECRUITING

Hospital Universitario De Navarra

Pamplona, , Spain

Site Status RECRUITING

Hospital Virgen Macarena

Seville, , Spain

Site Status RECRUITING

Hospital Virgen del Rocío

Seville, , Spain

Site Status RECRUITING

Incliva Biomedical Research Institute, University of Valencia

Valencia, , Spain

Site Status RECRUITING

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Site Status RECRUITING

Guy's and St Thomas' NHS Foundation Trust

London, London, United Kingdom

Site Status RECRUITING

The Christie NHS Foundation Trust - The Christie Clinic

Manchester, United Kingdom, United Kingdom

Site Status RECRUITING

Beatson West of Scotland Cancer Centre

Glasgow, , United Kingdom

Site Status RECRUITING

Sarah Cannon Research Institute UK

London, , United Kingdom

Site Status RECRUITING

Countries

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United States France Spain United Kingdom

Central Contacts

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Sarah Cannon Development Innovations

Role: CONTACT

844-710-6157

Other Identifiers

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ART0380C001

Identifier Type: -

Identifier Source: org_study_id

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