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Acute Myeloid LeukemiaPhase 3REGALNCT04229979

Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2

A Phase 3 study testing Galinpepimut-S, Azacitidine and Venetoclax in people with Acute Myeloid Leukemia, run by Sellas Life Sciences Group at 73 sites in 10 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

To assess the safety and efficacy of galinpepimut-S (GPS) compared with investigator's choice of best available therapy (BAT) on overall survival (OS) in subjects with acute myeloid leukemia (AML) who are in second or later complete remission (CR2) or second or later complete remission with incomplete platelet… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Galinpepimut-S, Azacitidine and Venetoclax
Could I receive a placebo?
No, everyone receives an active treatment
How many people
127 participants
Where
73 sitesin 10 countries
Study dates
8 Feb 2021to Dec 2026
Phase
Phase 3
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

Who may be able to join

  • Willing and able to understand and provide signed informed consent for the study that fulfills Institution Review Board (IRB) guidelines.
  • Male or female patients ≥18 years of age on the day of signing informed consent.
  • Must have a diagnosis of AML according to the WHO criteria (primary/de novo or secondary, including treatment-related [e.g., due to prior anthracycline use], as well as cases due to progression of antecedent hematological disorder [e.g., MDS, MPN, or MDS/MPN 'overlap' syndrome).
  • Must be in second morphological complete remission (with or without platelet recovery; CR2/CRp2) for relapsed AML based upon the CRp criteria as follows:
  • <5% myeloblasts in bone marrow
  • Absence of Auer rods
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  • Absence of circulating peripheral blasts
  • Peripheral blood absolute neutrophil count (ANC) >1000 cells/µL
  • Peripheral blood platelet count >20,000/µL
  • Absence of extramedullary disease
  • Patients must have > 300 lymphocytes/ μL.
  • Must not be candidates at the time of study entry for allogeneic stem cell transplant (Allo-SCT) due to intercurrent medical conditions, patient's preference or lack of an available donor.
  • Must have received the last dose of re-induction antileukemic therapy at least 4 weeks or ten half-lives of induction therapy (whichever is shorter) prior to receiving study treatment.
  • Must be consented within 6 months of having achieved CR2/CRp2 or later.
  • Must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, 2 or 3.
  • Must have an estimated life expectancy >6 months.
  • If female, is postmenopausal (at least 12 sequential months of amenorrhea) or surgically sterile. Females of childbearing potential must have a negative pregnancy test
  • Female patients of childbearing potential who are heterosexually active and male patients with female sexual partners of childbearing potential must agree to use an effective method of contraception (e.g., oral contraceptives, double-barrier methods such as a condom and a diaphragm, intrauterine device) during the study and for 4 to 6 months (depending on treatment) following the last dose of study medication, or to abstain from sexual intercourse for this time; a woman not of childbearing potential is one who has undergone bilateral oophorectomies or who is post- menopausal, defined as the absence of menstrual periods for 12 consecutive months.
  • Must have recovered to National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) v5 Grade 0 or 1 after completion of prior AML therapy with the exception of the platelet count requirements (i.e., as long as peripheral blood platelet count is >20,000/µL).
  • Must not have end stage renal disease.
  • Must have adequate hepatic function defined as a serum total bilirubin <2 × ULN (except for Gilbert's syndrome, which will allow bilirubin ≤3.0 mg/dL), and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3 × ULN.
  • Must be willing and able to return to the clinical site for adequate follow-up and to comply with the protocol as required.

Who cannot join

  • For subjects randomized to GPS maintenance monotherapy:
  • Continuation of any agents administered as part of induction of CR2/CRp2 or later
  • Receiving any concurrent anti-AML systemic therapy
  • Prior clinically significant allergic reaction to Montanide, sargramostim (GM-CSF) or filgrastim (granulocyte colony stimulating factor [G-CSF]).
  • Received any consolidation and/or maintenance antileukemic therapy, investigational agent, systemic corticosteroid therapy, or other immunosuppressive therapy within 4 weeks prior or 10 half lives, whichever is shorter prior to receiving study treatment. Systemic corticosteroids for chronic conditions (at doses ≤10 mg/day of prednisone or equivalent) or permitted, as are inhalational, intra-ocular, intra-articular and topical corticosteroids as well as any corticosteroids or other immunosuppressive therapies that do not act systemically (e.g. budesonide) at any dose level.
  • Imminently planned hematopoietic stem cell transplant (autologous or allogeneic, with any degree of match donor).
Show all 23Show fewer
  • Acute promyelocytic leukemia or any morphologic and molecular variants, inclusive.
  • Serious concurrent illness that in the opinion of the Investigator would pose an undue risk to the subject being participating in the clinical study.
  • Currently have, central nervous system leukemia.
  • Received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist®) are live attenuated vaccines and are not allowed. Vaccines for Covid-19 used under an EUA, are considered an authorized (though not an approved or cleared) medical product for use in clinical care. Vaccines used for the prevention of Covid-19 are allowed to be used.
  • Currently participating in or has participated in a study of an investigational agent or has used an investigational device within 4 weeks, or in the case of drugs 10 half lives, whichever is shorter, prior to the first dose of study treatment.
  • Patients who had an SCT after their most recent re-induction that resulted in CR2 or CRp2 or later are not eligible. Patients with prior SCT are allowed only if they had SCT prior to their latest re-induction or achieved CR by means of transplant ("hot transplant").
  • Diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy exceeding 10 mg daily of prednisone equivalent within 7 days prior the first dose of study drug. The use of physiologic doses of corticosteroids and/or immunosuppressive agents may be approved after consultation with the Sponsor. Steroids taken as short-term therapy (≤ 7 days) for antiemesis are permissible.
  • Known additional malignancy that is progressing or has required active treatment within the past 5 years, even if currently inactive or unapparent.
  • Known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, i.e., without evidence of progression for at least 4 weeks by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment.
  • Known hypersensitivity to Montanide or vaccine adjuvants.
  • Previous clinically significant systemic allergic reaction to Montanide, sargramostim (GM-CSF), or filgrastim (G-CSF).
  • Active autoimmune disease that has required systemic treatment in past 2 years (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment and is allowed.
  • Active life threatening infection requiring systemic therapy.
  • 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 participant to participate, in the opinion of the treating investigator. This includes any serious, intercurrent, chronic, or acute illness, such as cardiac disease (New York Heart Association [NYHA] class III or IV), hepatic disease, or other illness considered by the investigator as an unwarranted high risk for investigational drug treatment.
  • Known psychiatric or substance abuse disorder that would interfere with the participant's ability to cooperate with the requirements of the study.
  • Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 4 to 6 months (depending on treatment) after the last dose of study treatment.
  • Has had an allogeneic tissue/solid organ transplant.

Trial locations and contacts

73 sites in 10 countries.

73 sites in 10 countries

United States17 sites

  • Augusta University

    Augusta, Georgia, 30912

  • O'Neal Comprehensive Cancer Center

    Birmingham, Alabama, 35205

  • Rush University Cancer Center

    Chicago, Illinois, 60612

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106

  • Baylor Scott and White Research Institute

    Dallas, Texas, 75246

  • Colorado Blood Cancer Institute - SCRI - PPDS

    Denver, Colorado, 80218

  • Virginia Cancer Specialists

    Gainesville, Virginia, 20155

  • Bon Secours St. Francis Cancer Center

    Greenville, South Carolina, 29607

  • University of Texas - MD Anderson Cancer Center

    Houston, Texas, 77030

  • Mayo Clinic Jacksonville Florida

    Jacksonville, Florida, 32224

  • Northwell Health Cancer Institute

    Lake Success, New York, 11042

  • UCLA Medical Hematology and Oncology

    Los Angeles, California, 90095

  • Tulane Cancer Center - Liberty

    New Orleans, Louisiana, 70112

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • Swedish Cancer Institute

    Seattle, Washington, 98109

  • New York Medical College

    Valhalla, New York, 10532

  • The Oncology Institute of Hope and Innovation

    Whittier, California, 90603

Other trials in United States

Greece10 sites

  • University General Hospital of Alexandroupoli

    Alexandroupoli, 68100

  • General Hospital of Athens "Evaggelismos"

    Athens, 10676

  • General Hospital of Athens "Laiko"

    Athens, 11526

  • General Hospital of Athens "G. Gennimatas"

    Athens, 11527

  • General Hospital of Athens "Ηippokration"

    Athens, 11527

  • University General Hospital "Attikon"

    Chaïdári, 12462

  • General Hospital of Thessaloniki "G. Papanikolaou"

    Chortiatis, 57010

  • University General Hospital of Ioannina

    Ioannina, 45500

  • University General Hospital of Patras

    Rio, 26504

  • University General Hospital of Thessaloniki "Ahepa"

    Thessaloniki, 54636

Other trials in Greece

Poland10 sites

  • Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy

    Bydgoszcz, 85-168

  • Uniwersyteckie Centrum Kliniczne Klinika Hematologii i Transplantologii

    Gdansk, 80-952

  • Szpitale Pomorskie Sp. z o.o.

    Gdynia, 81-519

  • Swietokrzyskie Centrum Onkologii

    Kielce, 25-734

  • Wojewodzki Szpital Specjalistyczny w Legnicy

    Legnica, 59-220

  • Zaklad Opieki Zdrowotnej MSW z Warminsko-Mazurskim Centrum Onkologii

    Olsztyn, 10-228

  • Szpital Wojewodzki w Opolu

    Opole, 45-372

  • SP ZOZ Szpital Uniwersytecki w Krakowie

    Słomniki, 32-090

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

    Wroclaw, 50-367

Other trials in Poland

France9 sites

  • CHU Amiens-Picardie - Hopital Sud

    Amiens, 80000

  • CHU Angers

    Angers, 49000

  • CHU de Caen

    Caen, 14000

  • CHU de Grenoble

    Grenoble, 38043

  • Hôtel Dieu - Nantes

    Nantes, 44000

  • Hôpital Saint Antoine

    Paris, 75571

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69310

  • CHU de Poitiers

    Poitiers, 86000

  • Institut Gustave Roussy

    Villejuif, 94805

Other trials in France

Spain9 sites

  • Hospital de San Pedro de Alcantara

    Cáceres, 10003

  • C.H. Regional Reina Sofia

    Córdoba, 14004

  • Hospital General Universitario Gregorio Marañon

    Madrid, 28007

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital Universitario Central de Asturias

    Oviedo, 33011

  • Clinica Universidad Navarra

    Pamplona, 31008

  • Complejo Asistencial Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026

Other trials in Spain

India5 sites

  • Yashoda Hospital

    Hyderabad, 500084

  • Malabar Cancer Centre

    Kannur, Kerala, 670103

  • Fortis Hospital

    Ludhiāna, 141015

  • All India Institute of Medical Sciences

    New Delhi, 110029

  • State Cancer Institute, Indira Gandhi Institute of Medical Sciences

    Patna, 800014

Other trials in India

Taiwan5 sites

  • Changhua Christian Hospital

    Chang-hua, 50006

  • Kaohsiung Medical University Hospital

    Kaohsiung City, 807

  • Taichung Veterans General Hospital

    Taichung, 40705

  • National Cheng Kung University Hospital

    Tainan, 704

  • National Taiwan University Hospital

    Taipei, 100

Other trials in Taiwan

Germany3 sites

  • Klinikum Chemnitz gGmbH

    Chemnitz, 09116

  • Universitatsklinikum Leipzig

    Leipzig, 04103

  • Universitätsklinik Rostock

    Rostock, 18057

Other trials in Germany

Hungary3 sites

  • Semmelweis Egyetem

    Budapest, 1088

  • Petz Aladár Egyetemi Oktató Kórház

    Győr, 9028

  • Pécsi Tudományegyetem

    Pécs, 7624

Other trials in Hungary

Serbia2 sites

  • University Clinical Center of Serbia

    Belgrade, 11000

  • Clinical Centre of Vojvodina

    Novi Sad, 402007

Other trials in Serbia

About this study

Official title: A Randomized, Open-Label Study of the Efficacy and Safety of Galinpepimut-S (GPS) Maintenance Monotherapy Compared to Investigator's Choice of Best Available Therapy in Subjects With Acute Myeloid Leukemia Who Have Achieved Complete Remission After Second-Line Salvage Therapy

To assess the safety and efficacy of galinpepimut-S (GPS) compared with investigator's choice of best available therapy (BAT) on overall survival (OS) in subjects with acute myeloid leukemia (AML) who are in second or later complete remission (CR2) or second or later complete remission with incomplete platelet recovery (CRp2).

Read the detailed description

This is an open-label, multicenter, randomized, parallel groups study of galinpepimut-S (GPS) vs. best available treatment (BAT) in patients with AML in second complete remission (CR2) or in second complete remission with incomplete platelet recovery (CRp2). All patients will have their historical bone marrow samples and/or peripheral blood drawn during screening stained for WT1 via IHC and/or analyzed via PCR by central pathology review. The primary goal of the study will be to demonstrate an advantage for GPS in overall survival in these patient populations. The study will enroll approximately 140 patients and will be conducted at about 110 investigational sites. Patients will be randomized 1:1 to GPS or BAT stratified by whether they are in CR2 or CRp2, their cytogenetic risk at diagnosis (poor vs all other), whether they harbor minimal residual disease (MRD), and the duration of CR1 of less than one year or one year or more.

Patients on the BAT arm may be treated with 1. observation (whereby palliative management with hydroxyurea is allowed), 2. a hypomethylating agent (decitabine or azacitidine), 3. venetoclax and/or 4. low-dose ara-C. Patients whose remission in CR2 can be maintained with molecularly targeted agents (e.g. FLT-3 or IDH inhibitors) per investigator's determination will not be eligible. However, there are no restrictions on prior use of any agents in the CR1 setting. Patients cannot receive GPS as an adjunct therapy to any other agents.

Patients on the GPS arm will receive 70 μg of sargramostim (GM-CSF) on Day -2 and Day 1 before each injection of GPS. The first two administrations of GM-CSF will take place at the same anatomical site as the planned administration of GPS within each treatment cycle. GPS will be administered as an immunization induction every 2 weeks for 6 administrations (Weeks 0 - 10); this will be followed by a 4-week period of no treatment. Treatment will then resume for 6 administrations as an initial booster phase every 4 weeks (Weeks 14 - 34) which will again be followed by a period of no treatment lasting 6 weeks. GPS will be resumed after this period as a second booster phase and will be administered every 6 weeks (Weeks 40 - 52). Patients who remain in remission after 52 weeks will receive treatment every 2 months (Q2M) in the second year of treatment. Patients who remain in remission after 2 years will be treated every 3 months (Q3M) until disease relapse. Following each administration of GM-CSF or GPS, patients will be observed for approximately 30 minutes. An End of Treatment visit will be conducted 30 days following the last dose of GPS. Patients will then enter the long-term follow-up portion of the trial where they will be followed for recurrence of leukemia and overall survival.

To ensure a comparable level of observation, patients randomized to the BAT arm will be seen every 4 weeks through Week 52.

All patients will undergo bone marrow aspirates and biopsies at screening, Week 12, Week 52, and end of treatment or relapse. Bone marrow examinations will then be repeated as clinically indicated. Patients will be assessed for safety at each clinic encounter. The primary endpoint will be overall survival.

Being tested
Galinpepimut-S, Azacitidine, Venetoclax, Decitabine, Cytarabine
Keywords
maintenance, complete remission, overall survival, galinpepimut-S, randomized, Wilms Tumor-1 protein
Medical subject headings
Leukemia, Myeloid, Acute, Pathologic Complete Response

What participants receive

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

  • Galinpepimut-SBiological

    Also called SLS-001, GPS

    Galinpepimut-S admixed with the adjuvant Montanide following specified schedule

  • AzacitidineDrug

    injection

  • VenetoclaxDrug

    tablet

  • DecitabineDrug

    injection

  • CytarabineDrug

    Also called Ara-C

    injection

  • ObservationOther

    palliative management

  • GM-CSFBiological

    Also called sargramostim

    subcutaneous injection

  • MontanideOther

    adjuvant

Study groups in NCT04229979
Study groupRoleWhat they receive
Galinpepimut-S + Montanide + GM-CSFExperimentalGalinpepimut-S injections will be administered as follows, until disease relapse: 1. First 6 galinpepimut-S injections: every 2 weeks (Weeks 0 - 10) followed by a 4-week period of no treatment. The first series of 6 injections of galinpepimut-S define the initial immunization induction phase. 2. Injections 7 to 12: every 4 weeks (Weeks 14 - 34) followed by a 6-week period of no treatment. The second series of injections of galinpepimut-S define the early immune booster phase. 3. Injections 13 to 15: every 6 weeks (Weeks 40 - 52). The third series of injections of galinpepimut-S define the late immune booster phase. 4. Injections 16-20: every 2 months (in Year 2). 5. Injection 21 and thereafter: every 3 months (in Year 3). Y2 and Y3 define the maintenance phase. Note: Galinpepimut-S is admixed with Montanide adjuvant before administered as a subcutaneous injection. GM-CSF is administered two days before and on the same day as the galinpepimut-S + Montanide injection.
Receives: Biological: Galinpepimut-S; Biological: GM-CSF; Other: Montanide
Best Available TherapyActive comparatorFour options, as monotherapy or as combination of agents listed below, (per treating investigator's choice): 1. Observation (whereby palliative management with hydroxyurea is allowed), or 2. HMA (decitabine or azacitidine), and/or 3. Venetoclax, and/or 4. Low-dose ara-C
Receives: Drug: Azacitidine; Drug: Venetoclax; Drug: Decitabine; Drug: Cytarabine; Other: Observation

What is being measured

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

  1. OS

    Interval between randomization and death from any cause

    Measured: 5 years

Secondary outcomes (3)

  1. LFS

    Interval between randomization and recurrence of leukemia or death from any cause

    Measured: 5 years

  2. OS rate (%)

    Percentage of patients surviving

    Measured: At 6, 9 and 12 months

  3. LFS rate (%)

    Percentage of patients surviving and being free of leukemic relapse

    Measured: At 6, 9, and 12 months

Timeline

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

  1. First posted18 Jan 2020
  2. Study start8 Feb 2021
  3. Primary completionDec 2025 (estimated)
  4. Study completionDec 2026 (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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Open-label, multicenter, randomized, parallel group study
Masking
None (open label)
Enrolment
127 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04229979
Sponsor's protocol ID
SLSG18-301
Acronym
REGAL
Status last verified
Sep 2025
First submitted
12 Jan 2020
Last updated
1 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Jamy O, Cicic D. REGAL: galinpepimut-S vs. best available therapy as maintenance therapy for acute myeloid leukemia in second remission. Future Oncol. 2025 Jan;21(1):73-81. doi: 10.1080/14796694.2024.2433935. Epub 2024 Nov 28.

    PubMed 39606837

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Galinpepimut-S, Azacitidine and Venetoclax in people with Acute Myeloid Leukemia, run by Sellas Life Sciences Group at 73 sites in 10 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 22 inclusion and 23 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: under way, not looking for participants, as of the sponsor's last update on 1 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 73 sites in United States, Greece, Poland, France, Spain and 5 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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?

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?

OS (time frame: 5 years). 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 NCT04229979, which also lists 1 linked publication.

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 1 Oct 2025.

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