AzTrials
AzTrials

Breast CancerPhase 2BYLieveNCT03056755

Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments

A Phase 2 study testing Alpelisib, Fulvestrant and Letrozole in people with Breast Cancer, run by Novartis Pharmaceuticals at 94 sites in 19 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This was a Phase II, multicenter, open-label, three-cohort, non-comparative study of alpelisib plus endocrine therapy (either fulvestrant or letrozole) in subjects (pre- and post-menopausal women and men) with HR-positive, HER2-negative aBC harboring PIK3CA mutation(s) in the tumor, whose disease had progressed on or… Read more about this study

Who is it for?
Ages 18 to 99 years
Conditions the study is for
Breast Cancer
What is being tested?
Alpelisib, Fulvestrant and Letrozole
Could I receive a placebo?
No, everyone receives an active treatment
How many people
383 participants
Where
94 sitesin 19 countries
Study dates
29 Aug 2017to 12 Nov 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 to 99 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

None listed.

Who cannot join

  • Subjects with a known hypersensitivity to alpelisib, fulvestrant, letrozole, goserelin, or leuprolide or to any of their excipients.
  • Subjects with prior treatment with any PI3K inhibitor (PI3Ki).
  • Subjects with central nervous system (CNS) involvement unless they met all of the following criteria:
  • At least 4 weeks from prior therapy completion (including radiation and/or surgery) to starting the study treatment, ii. Clinically stable CNS tumor at the time of screening untreated or without evidence of progressions for at least 4 weeks after treatment as determined by clinical examination and brain imaging (magnetic resonance imaging (MRI) or computed tomography (CT) during screening period and stable low dose of steroids for 2 weeks prior to initiating study treatment.
  • Subjects with an established diagnosis of diabetes mellitus type I or uncontrolled type II.
  • Any concurrent severe and/or uncontrolled medical conditions that would, in the investigator's judgment, contraindicate subject participation in the study (e.g., chronic active hepatitis, severe hepatic impairment, etc.).
Show all 16Show fewer
  • Subjects with a history of noncompliance to medical regimens.
  • Subjects with impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of the study drugs based on investigator discretion.
  • Subjects with documented pneumonitis/interstitial lung disease which was active and requiring treatment.
  • Subjects concurrently using other anticancer therapy. All anticancer therapy had to be discontinued prior to Day 1 of study treatment.
  • Subjects who had major surgery within 14 days prior to starting treatment with alpelisib, or did not recover from major side effects.
  • Subjects with significant cardiac abnormalities.
  • History of acute pancreatitis within 1 year of screening or past medical history of chronic pancreatitis.
  • Subjects who did not use highly effective contraception while on alpelisib and through the duration after the final dose of alpelisib (not applicable to postmenopausal women).
  • Subjects with unresolved osteonecrosis of the jaw.
  • Other inclusion/exclusion criteria may apply

Trial locations and contacts

94 sites in 19 countries.

94 sites in 19 countries

United States25 sites

  • New Mexico Cancer Care Alliance

    Albuquerque, New Mexico, 87106

  • Kaiser Permanente Medical Group

    Anaheim, California, 92807

  • Mercy Medical Center

    Baltimore, Maryland, 21202

  • Greater Baltimore Med Center Cancer Center

    Baltimore, Maryland, 21204

  • Beverly Hills Cancer Center

    Beverly Hills, California, 90211

  • St Vincent Frontier Cancer Center

    Billings, Montana, 59102

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Lahey Clinic

    Burlington, Massachusetts, 01805

  • Uni Hosp of Cleveland Cancer Center

    Cleveland, Ohio, 44106

  • Texas Oncology

    Dallas, Texas, 75246

  • Josephine Ford Cancer Center

    Detroit, Michigan, 48202

  • Banner MD Anderson Cancer Center

    Gilbert, Arizona, 85234

  • University of Calif Irvine Med Cntr

    Irvine, California, 92660

  • University of Kansas Cancer Center

    Kansas City, Kansas, 66205

  • University of Louisville James Graham Brown Cancer Center

    Louisville, Kentucky, 40202

  • Yale University Yale Cancer Center

    New Haven, Connecticut, 06511

  • Memorial Sloane Ketterin Cancer Ctr

    New York, New York, 10065

  • Virginia Oncology Associates

    Norfolk, Virginia, 23502

  • Advent Health Cancer Institute

    Orlando, Florida, 32804

  • Mayo Clinic Arizona

    Phoenix, Arizona, 85054

  • Cancer Care Centers of South Texas HOAST

    San Antonio, Texas, 78229

  • UT Health San Antonio

    San Antonio, Texas, 78229

  • Kaiser Permanent Southern Californi

    San Diego, California, 92120

  • UCSF

    San Francisco, California, 94115

  • Northwest Medical Specialists

    Tacoma, Washington, 98405

Other trials in United States

France10 sites

  • Novartis Investigative Site

    Bordeaux, 33076

  • Novartis Investigative Site

    Caen, 14021

  • Novartis Investigative Site

    Lille, 59020

  • Novartis Investigative Site

    Lyon, 69373

  • Novartis Investigative Site

    Montpellier, 34298

  • Novartis Investigative Site

    Nice, Alpes Maritimes, 06189

  • Novartis Investigative Site

    Saint-Cloud, Hauts De Seine, 92210

  • Novartis Investigative Site

    Saint-Herblain, 44805

  • Novartis Investigative Site

    Strasbourg, 67000

  • Novartis Investigative Site

    Toulouse, 31059

Other trials in France

Italy8 sites

  • Novartis Investigative Site

    Ancona, AN, 60126

  • Novartis Investigative Site

    Bergamo, BG, 24127

  • Novartis Investigative Site

    Bologna, BO, 40138

  • Novartis Investigative Site

    Genova, GE, 16132

  • Novartis Investigative Site

    Milan, MI, 20133

  • Novartis Investigative Site

    Milan, MI, 20141

  • Novartis Investigative Site

    Naples, 80131

  • Novartis Investigative Site

    Roma, RM, 00168

Other trials in Italy

Germany7 sites

  • Novartis Investigative Site

    Augsburg, 86150

  • Novartis Investigative Site

    Berlin, 14169

  • Novartis Investigative Site

    Dresden, Saxony, 01307

  • Novartis Investigative Site

    Erlangen, 91054

  • Novartis Investigative Site

    Essen, 45136

  • Novartis Investigative Site

    Heidelberg, 69120

  • Novartis Investigative Site

    Ulm, 89081

Other trials in Germany

Spain7 sites

  • Novartis Investigative Site

    Barcelona, Catalonia, 08035

  • Novartis Investigative Site

    Barcelona, 08036

  • Novartis Investigative Site

    Castellon, 12002

  • Novartis Investigative Site

    L'Hospitalet de Llobregat, Barcelona, 08907

  • Novartis Investigative Site

    Madrid, 28041

  • Novartis Investigative Site

    Salamanca, Castille and León, 37007

  • Novartis Investigative Site

    Seville, 41013

Other trials in Spain

Argentina6 sites

  • Novartis Investigative Site

    CABA, Buenos Aires, C1118AAT

  • Novartis Investigative Site

    CABA, Buenos Aires, C1125ABD

  • Novartis Investigative Site

    CABA, Buenos Aires, C1426ANZ

  • Novartis Investigative Site

    La Rioja, 5300

  • Novartis Investigative Site

    Rosario, Santa Fe Province, S2000DSV

  • Novartis Investigative Site

    Rosario, Sante Fe, S200KZE

Other trials in Argentina

United Kingdom5 sites

  • Novartis Investigative Site

    Edinburgh, Scotland, EH4 2XU

  • Novartis Investigative Site

    Leicester, LE2 7LG

  • Novartis Investigative Site

    London, SW3 6JJ

  • Novartis Investigative Site

    Nottingham, NG5 1PB

  • Novartis Investigative Site

    Sutton, Surrey, SM2 5PT

Other trials in United Kingdom

Canada4 sites

  • Novartis Investigative Site

    Halifax, Nova Scotia, B3H 2Y9

  • Novartis Investigative Site

    Kitchener, Ontario, N2G 1G3

  • Novartis Investigative Site

    Toronto, Ontario, M5B 1N9

  • Novartis Investigative Site

    Vancouver, British Columbia, V5Z 4E6

Other trials in Canada

Israel4 sites

  • Novartis Investigative Site

    Petah Tikva, 4941492

  • Novartis Investigative Site

    Ramat Gan, 5265601

  • Novartis Investigative Site

    Rehovot, 7661041

  • Novartis Investigative Site

    Tel Aviv, 6423906

Other trials in Israel

Chile3 sites

  • Novartis Investigative Site

    Santiago, 7500921

  • Novartis Investigative Site

    Santiago, 8420383

  • Novartis Investigative Site

    Temuco, Araucania, 4810469

Other trials in Chile

Taiwan3 sites

  • Novartis Investigative Site

    Tainan, 70403

  • Novartis Investigative Site

    Taipei, 10002

  • Novartis Investigative Site

    Taipei, 103616

Other trials in Taiwan

Belgium2 sites

  • Novartis Investigative Site

    Leuven, 3000

  • Novartis Investigative Site

    Liège, 4000

Other trials in Belgium

Denmark2 sites

  • Novartis Investigative Site

    Odense C, 5000

  • Novartis Investigative Site

    Vejle, DK-7100

Other trials in Denmark

Singapore2 sites

  • Novartis Investigative Site

    Singapore, 168583

  • Novartis Investigative Site

    Singapore, 217562

Other trials in Singapore

South Korea2 sites

  • Novartis Investigative Site

    Seoul, Korea, 03080

  • Novartis Investigative Site

    Seoul, 06351

Other trials in South Korea

India1 site

  • Novartis Investigative Site

    Delhi, 110085

Other trials in India

Japan1 site

  • Novartis Investigative Site

    Osaka, Osaka, 540-0006

Other trials in Japan

Mexico1 site

  • Novartis Investigative Site

    Jalisco, 45640

Other trials in Mexico

Netherlands1 site

  • Novartis Investigative Site

    Maastricht, Limburg, 6229 HX

Other trials in Netherlands

About this study

Official title: BYLieve: A Phase II, Multicenter, Open-label, Three-cohort, Non- Comparative Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole in Patients With PIK3CA Mutant, Hormone Receptor (HR) Positive, HER2-negative Advanced Breast Cancer (aBC), Who Have Progressed on or After Prior Treatments

This was a Phase II, multicenter, open-label, three-cohort, non-comparative study of alpelisib plus endocrine therapy (either fulvestrant or letrozole) in subjects (pre- and post-menopausal women and men) with HR-positive, HER2-negative aBC harboring PIK3CA mutation(s) in the tumor, whose disease had progressed on or after prior treatments.

Read the detailed description

The study comprised two phases:

  1. Core Phase: included a treatment phase for all subjects from first subject first treatment (FSFT) until disease progression, unacceptable toxicity, or death until 18 months post last subject first treatment (LSFT) + 1 month safety follow-up (total 19 months post LSFT), discontinuation from the study treatment for any other reason, or sponsor terminates the study. At the start of the Core Phase, subjects were assigned to Cohort A, Cohort B, or Cohort C based on previous therapy as follows:
  • Cohort A: alpelisib (300 mg oral QD) + fulvestrant (500 mg intramuscular (IM)) to subjects whose last prior treatment was a CDK4/6i plus any AI;
  • Cohort B: alpelisib (300 mg oral QD) + letrozole (2.5 mg oral QD) to subjects whose last prior treatment was a CDK4/6i plus fulvestrant;
  • Cohort C: alpelisib (300 mg oral QD)+ fulvestrant (500 mg IM) to subjects who failed prior AI based therapy and whose last prior treatment was systemic chemotherapy or endocrine therapy (as monotherapy or in combination with targeted treatment except CDK 4/6i + AI). Endocrine therapy included letrozole, fulvestrant and CDK 4/6 inhibitor plus fulvestrant.
  1. Extension Phase: included a treatment phase starting at the end of the treatment Core Phase until last subject last visit(LSLV) (up to 36 months). Following the end of the Core Phase, subjects continuing to derive benefit from study treatment who were not eligible for Post-Study Drug Supply (PSDS) in their country based on local regulations were re-consented and transitioned to the Extension Phase. Subjects continued on their existing study treatment assigned in the Core Phase until disease progression/lack of clinical benefit or any other reason for which the subject may have been discontinued from study treatment. If PSDS became available for a subject, the subject was to be discontinued from the study and to access treatment via PSDS. When subjects discontinued treatment for any reason, the end of treatment visit was performed after discontinuation of the medication , followed by safety follow-up 30 days after last dose.

A total of 340 subjects were planned to be enrolled, with approximately 112 subjects in each cohort. In the Core Phase, 379 subjects were analyzed, with 127 subjects in Cohort A, 126 subjects in Cohort B, and 126 subjects in Cohort C. In the Extension Phase, 11 subjects were analyzed, with 1 subject in Cohort A and 10 subjects in Cohort C.

Conditions
Breast Cancer
Being tested
Alpelisib, Fulvestrant, Letrozole, Goserelin, Leuprolide
Keywords
advanced breast cancer, PIK3CA, CDK 4/6 inhibitor, fulvestrant, letrozole, HR+, HER2-negative, post menopausal, pre-menopausal, aromatase inhibitor, endocrine treatment, AI, ET
Medical subject headings
Breast Neoplasms, Hereditary Sensory and Autonomic Neuropathies

What participants receive

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

  • AlpelisibDrug

    Also called BYL719

    300 mg of alpelisib film-coated tablets administered orally once daily

  • FulvestrantDrug

    500 mg of fulvestrant via intramuscular injection administered on Days 1, 15 on Cycle 1 and Day 1 at each cycle thereafter. Cycle=28 days

  • LetrozoleDrug

    2.5 mg of letrozole film-coated tablets administered orally once daily

  • GoserelinDrug

    3.6 mg of goserelin via injectable subcutaneous implant administered every 28 days. Only for men in Cohort B and premenopausal women.

  • LeuprolideDrug

    7.5 mg of leuprolide via injectable intramuscular depot administered every 28 days. Only for men in cohort B and premenopausal women.

Study groups in NCT03056755
Study groupRoleWhat they receive
Cohort A: Pre-treated with CDK 4/6i + AIExperimentalParticipants who received any Cyclin-Dependent Kinases 4 and 6 inhibitor (CDK 4/6i) plus aromatase inhibitor (AI) as immediate prior treatment will receive alpelisib + fulvestrant
Receives: Drug: Alpelisib; Drug: Fulvestrant; Drug: Goserelin; Drug: Leuprolide
Cohort B: Pre-treated with CDK 4/6i + fulvestrantExperimentalPatients who received any CDK 4/6i plus fulvestrant as immediate prior treatment will receive alpelisib + letrozole
Receives: Drug: Alpelisib; Drug: Letrozole; Drug: Goserelin; Drug: Leuprolide
Cohort C: Pre-treated with systemic chemotherapy or ETExperimentalParticipants who received systemic chemotherapy or endocrine therapy (ET) (as monotherapy or in combination with targeted treatment except CDK 4/6i + AI) as immediate prior treatment will receive alpelisib + fulvestrant.
Receives: Drug: Alpelisib; Drug: Fulvestrant; Drug: Goserelin; Drug: Leuprolide

What is being measured

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

  1. Core Phase: Percentage of Participants Who Were Alive Without Disease Progression at 6 Months

    Percentage of participants who were alive without disease progression at 6-month follow-up based on local investigator assessment per RECIST v1.1 in Cohort A, Cohort B and Cohort C. Participants who progressed, died, or discontinued study before 6 months were counted as a failure.

    Measured: At 6 months

Secondary outcomes (7)

  1. Core Phase: Progression Free Survival (PFS)

    PFS is defined as the time from the date of first dose of study medication to the date of the first documented progression or death due to any cause occurring in the study. PFS was assessed based on local investigator's assessment according to RECIST v1.1. PFS was censored if no PFS event was observed before the cut-off date. The censoring date was the date of last adequate tumor assessment before the cut-off date. If a PFS event was observed after two or more missing or non-adequate tumor assessments, then PFS was censored at the last adequate tumor assessment. PFS was estimated using the Kaplan-Meier method.

    Measured: From date of first dose to date of first documented progression or death, up to 46 months

  2. Core Phase: Progression Free Survival on Next Line Treatment (PFS2)

    PFS2 is defined as time from the date of first dose of study medication to the date of first documented progression on next-line therapy or death from any cause. The first documented progression on next-line treatment is based on investigator assessment of progressive disease. PFS2 was estimated using the Kaplan-Meier method.

    Measured: From date of first dose to date of first documented progression on next-line therapy or death, up to approximately 55 months

  3. Core Phase: Overall Response Rate (ORR)

    ORR is defined as the percentage of participants with best overall response (BOR) of complete response (CR) or partial response (PR) based on local investigator's assessment according to RECIST v1.1 in each cohort. CR: Disappearance of all non-nodal target lesions and all non-target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm and all lymph nodes assigned as non-target lesions must be non-pathological in size (<10 mm short axis) PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters.

    Measured: Up to 46 months

  4. Core Phase: Clinical Benefit Rate (CBR)

    CBR is defined as the percentage of participants with a BOR of CR or PR or an overall lesion response of stable disease (SD) or Non-CR/ Non-PD lasting ≥ 24 weeks based on local investigator's assessment according to RECIST v1.1. CR: Disappearance of all non-nodal target lesions and all non-target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm and all lymph nodes assigned as non-target lesions must be non-pathological in size (<10 mm short axis) PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. SD: Neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for progressive disease.

    Measured: Up to 46 months

  5. Core Phase: Duration of Response (DOR)

    DOR is the time from the date of first documented response (confirmed CR or PR based on local investigator's assessment according to RECIST v1.1) to the date of first documented progression or death due to underlying cancer. Subjects continuing without progression or death due to underlying cancer were censored at the date of their last adequate tumor assessment. DOR was estimated using the Kaplan-Meier method.

    Measured: From date of first documented response to first documented progression or death, up to 33.3 months

  6. Core Phase: Overall Survival (OS)

    OS is defined as the time of start of treatment to date of death or lost to follow-up. If a subject was not known to have died, then the OS data was censored at the date of the last known alive status for the patient.

    Measured: From date of first dose and up to approximately 55 months

  7. Extension Phase: Percentage of Participants With Clinical Benefit as Assessed by the Investigator During the Extension Phase

    Clinical Benefit Rate, as assessed by the Investigator during the Extension Phase, was defined as the proportion of patients with a best overall response of Complete Response (CR) or Partial Response (PR), or an overall lesion response of Stable Disease (SD) or non-complete response/non-progressive disease that lasted at least 24 weeks, based on the local investigator's assessment according to Response Evaluation Criteria in Solid Tumors version 1.1.

    Measured: Day 1 of each extension cycle up to Cycle 29 (each cycle lasting 28 days); median duration of exposure to study treatment (alpelisib or fulvestrant) = 41 months

Timeline

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

  1. First posted17 Feb 2017
  2. Study start29 Aug 2017
  3. Primary completion14 Jun 2021
  4. Study completion12 Nov 2024

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
383 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03056755
Sponsor's protocol ID
CBYL719X2402
Other IDs
2023-509167-24-00, 2023
Acronym
BYLieve
Status last verified
Dec 2025
First submitted
15 Feb 2017
Last updated
13 Jan 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

  • Rugo HS, Lerebours F, Ciruelos E, Drullinsky P, Ruiz-Borrego M, Neven P, Park YH, Prat A, Bachelot T, Juric D, Turner N, Sophos N, Zarate JP, Arce C, Shen YM, Turner S, Kanakamedala H, Hsu WC, Chia S. Alpelisib plus fulvestrant in PIK3CA-mutated, hormone receptor-positive advanced breast cancer after a CDK4/6 inhibitor (BYLieve): one cohort of a phase 2, multicentre, open-label, non-comparative study. Lancet Oncol. 2024 Dec;25(12):e629-e638. doi: 10.1016/S1470-2045(24)00673-9.

    PubMed 39637900

  • Borrego MR, Lu YS, Reyes-Cosmelli F, Park YH, Yamashita T, Chiu J, Airoldi M, Turner N, Fein L, Ghaznawi F, Singh J, Pantoja K, Schnell C, Akdere M, Chia S. SGLT2 inhibition improves PI3Kalpha inhibitor-induced hyperglycemia: findings from preclinical animal models and from patients in the BYLieve and SOLAR-1 trials. Breast Cancer Res Treat. 2024 Nov;208(1):111-121. doi: 10.1007/s10549-024-07405-8. Epub 2024 Aug 23.

    PubMed 39177931

  • Rodon J, Demanse D, Rugo HS, Burris HA, Simo R, Farooki A, Wellons MF, Andre F, Hu H, Vuina D, Quadt C, Juric D. A risk analysis of alpelisib-induced hyperglycemia in patients with advanced solid tumors and breast cancer. Breast Cancer Res. 2024 Mar 4;26(1):36. doi: 10.1186/s13058-024-01773-1.

    PubMed 38439079

  • Rugo HS, Lerebours F, Ciruelos E, Drullinsky P, Ruiz-Borrego M, Neven P, Park YH, Prat A, Bachelot T, Juric D, Turner N, Sophos N, Zarate JP, Arce C, Shen YM, Turner S, Kanakamedala H, Hsu WC, Chia S. Alpelisib plus fulvestrant in PIK3CA-mutated, hormone receptor-positive advanced breast cancer after a CDK4/6 inhibitor (BYLieve): one cohort of a phase 2, multicentre, open-label, non-comparative study. Lancet Oncol. 2021 Apr;22(4):489-498. doi: 10.1016/S1470-2045(21)00034-6.

    PubMed 33794206

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Alpelisib, Fulvestrant and Letrozole in people with Breast Cancer, run by Novartis Pharmaceuticals at 94 sites in 19 countries.

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

Who may be eligible to take part?

The sponsor lists 0 inclusion and 16 exclusion criteria. 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: finished, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 94 sites in United States, France, Italy, Germany, Spain and 14 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 phase mean?

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?

Yes. Results are posted in the registry (first posted 27 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Core Phase: Percentage of Participants Who Were Alive Without Disease Progression at 6 Months (time frame: At 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03056755, which also lists 4 linked publications.

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 13 Jan 2026.

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