Acute Lymphoblastic Leukemia RecurrentEarly Phase 1INA03NCT03957915
Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia
A Early Phase 1 study testing INA03 administration in people with Acute Lymphoblastic Leukemia Recurrent and Acute Lymphoblastic Leukemia, in Relapse, run by Institut Paoli-Calmettes at 3 sites in France.
Trial summary
This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Lymphoblastic Leukemia Recurrent, Acute Lymphoblastic Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapse
- What is being tested?
- INA03 administration
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 3 sitesFrance
- Study dates
- 29 May 2020to 24 Feb 2025
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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
- . Patient with
- cytologically confirmed and documented B-cell or T-cell ALL or de novo, secondary or therapy-related AML or Mixed Phenotype Acute Leukemia (MPAL) defined according to World Health Organization (WHO) 2016 classification28 AND
- with 20% or more CD71 positive blast cells
- in relapse after- or refractory to registered therapies or ineligible to standard treatments
- with circulating blasts ≤ 20 000/mm3. For eligible patients with AML/ALL with blasts > 20000/mm3, a treatment with hydroxyurea is allowed to maintain tumor cells ≤ 20000/mm3 2. Male or female age ≥ 18 years 3. WHO performance status 0-2 4. Following laboratory values unless considered due to the leukemia:
- AST and or ALT ≤ 2.5 ULN
Show all 13Show fewer
- Total bilirubin level < 1.5 ULN (except Gilbert disease)
- Serum creatinine ≤ 1.5 ULN
- LDH < 3-5 ULN
- Uric acid ≤8 mg/dl
- Electrolyte panel within normal range
- Urine Dipstick Reading negative for proteinuria or, if documentation of +1 results for protein on dipstick reading, then total urinary protein ≤ 500 mg and measured creatinine clearance ≥50mL/min/1.73m2 from a 24-hour urine collection
- Patients who have recovered at least CTCAE grade <2 5. Life expectancy greater than 3 months 6. Women of child bearing potential must be willing to use birth control method during the study duration (W4 or early termination) plus 30 days. Male partner of women must use condom; in case of male patient, he must agree to use condom during the study duration (W4 or early termination) plus 30 days; 7. Pregnancy test (females of childbearing potential): negative 8. Signed informed consent indicating that they have been informed of the procedures to be followed, an is willing and able to comply the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts 9. Patient affiliated to the national "Social Security" regimen or beneficiary of this regi-men
Who cannot join
- Patients with acute promyelocytic leukemia
- Patients with more than 30% marrow erythroid cells
- Patients who have been treated with any anti-TfR antibody
- Allogeneic stem cell transplantation in the last 6 months or with persistent active GVHD. Autologous bone marrow transplant in the last 3 months
- Last dose of prior chemotherapy, immunotherapy or investigational agent within 14 days or within 5 half-lives before baseline receipt of study medication, except for hydroxyurea and corticosteroids
- 7. Patients who have had a prior anaphylactic or other severe infusion reaction such that the patient is unable to tolerate human immunoglobulin or monoclonal antibody administration 8. Patients who have history or clinical evidence of central nervous system (CNS), meningeal, or epidural disease from any cause and/or peripheral neuropathy 9. Impaired cardiac function or clinically significant cardiac disease, including any one of the following:
Show all 11Show fewer
- New York Heart Association Class III or IV cardiac disease, including preexisting clinically significant arrhythmia, congestive heart failure, or cardiomyopathy b. Angina pectoris ≤ 3 months prior to starting study drug c. Acute myocardial infarction ≤ 3 months prior to starting study drug d. Other clinically significant heart disease (e.g., uncontrolled hypertension, history of labile hypertension, or history of poor compliance with an antihypertensive regimen) e. Left ventricular Ejection Fraction <45% 11. Uncontrolled infection 12. Acute and chronic liver disease 13. Other severe, acute, or chronic medical or psychiatric condition or laboratory abnor-mality that may increase the risk associated with study participation or study drug administra-tion or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for this study.
- 14. Patients with prior radiation therapy
- ≤12 weeks for cranial radiation therapy
- ≤ 4 weeks for wide field radiation therapy
- ≤2 weeks for involved field radiation therapy 15. Major surgery ≤ 4weeks prior to starting study drug or who have not recovered from side effects of such therapy 16. Known diagnosis of HIV infection (HIV testing is not mandatory). 17. History of another primary malignancy that is currently clinically significant or currently requires active intervention 18. Pregnant or breastfeeding patient; 19. Active drug or alcohol dependence; 20. Any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
Trial locations and contacts
3 sites in 1 country.
France3 sites
Institut Paoli-Calmettes
Marseille, Bouches-du Rhône, 13009
Chu Bordeaux Hopital Haut Leveque
Pessac, 33604
IUCT
Toulouse
About this study
Official title: A Phase I, First in Human, Open-label Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia
This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL.
The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
Read the detailed description
This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL.
The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
- Conditions
- Acute Lymphoblastic Leukemia Recurrent, Acute Lymphoblastic Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukaemia Recurrent, Mixed Phenotype Acute Leukemia
- Being tested
- INA03 administration
- Keywords
- acute leukemia, refractory, in relapse, antibody drug conjugate, first in human, escalating doses, INA03
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INA03 administrationDrug
INA03 will be administered IV on Day 1, Day 14 of 28-day cycles. The administration of INA03 will begin at 0.02 mg/kg. Study Part I is a titration study to determine the dose for the first INA03 infusion. Patients will be enrolled in sequential cohorts of 2 patients to receive ascending starting doses of INA03, starting from the lowest starting dose (0.02 mg/kg), and followed by subsequent administrations of INA03 (D14 and beyond) at a fixed dose of 0.1 mg/kg. The starting dose will be increased every cohort of 2 patients until evidence of absence of marrow residual erythroblasts by D14 myelogram. This dose is referred to as the MEID and will be selected as the D1 dose for the study Part 2. Patient accumulation in Part I of the study will continue until no evidence of non-hematological DLT within 28 days post dosing
| Study group | Role | What they receive |
|---|---|---|
| INA03 | Experimental | INA03 administration Receives: Drug: INA03 administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of the minimal erythroblastopenia-inducing dose (MEID) for INA03 in adults with refractory/relapsed acute leukemia
MEID defined as the lowest dose associated with the risks of residual erythroblasts in the bone marrow at 2 weeks from initial dosing or grade 2 or above non hematologic toxicity within 2 weeks from initial dosing
Measured: within 2 weeks from initial dosing
Determination of the maximum tolerated dose (MTD) for subsequent administrations (D15 from initial dosing and beyond) for INA03 in adults with refractory/relapsed acute leukemia
the maximum tolerated dose (MTD) for subsequent administrations (D15 from initial dosing and beyond)
Measured: 28 days from the first administration of INA03
Secondary outcomes (7)
Safety of INA03: NCI-CTCAE v5.0
Safety from the findings of reports of adverse events based on incidence, severity (as graded by the NCI-CTCAE v5.0), cumulative nature of treatment-emergent adverse event (TEAEs)
Measured: Until 30 days after last dose
pharmacokinetic (PK) profile of INA03
Peak Plasma Concentration (Cmax) will be calculated, as appropriate
Measured: From initial dosing day to Day 42
pharmacokinetic (PK) profile of INA03
Area under the plasma concentration versus time curve (AUC) will be calculated, as appropriate
Measured: From initial dosing day to Day 42
pharmacokinetic (PK) profile of INA03
The terminal half-life will be calculated, as appropriate
Measured: From initial dosing day to Day 42
pharmacodynamics (PD) profile of INA03
PD according to variation of erythroblast and blasts decrease using bone marrow as-pirate (BMA), and blood samples before and under treatment
Measured: From screening to end of study visit (maximum 182 days)
Concentration of anti-INA03 antibodies
Serum concentration of anti-INA03 antibodies in micrograms per milliliter
Measured: From screening to end of study visit (maximum 182 days)
Preliminary clinical response of INA03
Clinical response as defined by European LeukemiaNet (ELN) 2017 recommendations
Measured: from the date of treatment initiation (Day-1) to the date of relapse, progression or death, whichever comes first (a maximum 182 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2019
- Study start29 May 2020
- Primary completion15 Jul 2024
- Study completion24 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (32)Papers the sponsor has linked to this study.
- Other trials for Acute Lymphoblastic Leukemia RecurrentStudies of the same condition, open and under way.
- More trials run by Institut Paoli-CalmettesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL. The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut Paoli-Calmettes
- Sponsor type
- University, hospital or other
- Collaborators
- INATHERYS
- Responsible party
- Sponsor
- Investigators
- Norbert VEY, Pr, Principal investigator, Institut Paoli-Calmettes
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03957915
- Sponsor's protocol ID
- INA03-IPC 2018-008
- Other IDs
- 2019-000814-13, 2019
- Acronym
- INA03
- Status last verified
- Dec 2024
- First submitted
- 6 May 2019
- Last updated
- 6 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kell J. Considerations and challenges for patients with refractory and relapsed acute myeloid leukaemia. Leuk Res. 2016 Aug;47:149-60. doi: 10.1016/j.leukres.2016.05.025. Epub 2016 Jun 11.
PubMed 27371910Dohner H, Dolnik A, Tang L, Seymour JF, Minden MD, Stone RM, Del Castillo TB, Al-Ali HK, Santini V, Vyas P, Beach CL, MacBeth KJ, Skikne BS, Songer S, Tu N, Bullinger L, Dombret H. Cytogenetics and gene mutations influence survival in older patients with acute myeloid leukemia treated with azacitidine or conventional care. Leukemia. 2018 Dec;32(12):2546-2557. doi: 10.1038/s41375-018-0257-z. Epub 2018 Oct 1.
PubMed 30275526Eleni LD, Nicholas ZC, Alexandros S. Challenges in treating older patients with acute myeloid leukemia. J Oncol. 2010;2010:943823. doi: 10.1155/2010/943823. Epub 2010 Jun 10.
PubMed 20628485Dombret H, Seymour JF, Butrym A, Wierzbowska A, Selleslag D, Jang JH, Kumar R, Cavenagh J, Schuh AC, Candoni A, Recher C, Sandhu I, Bernal del Castillo T, Al-Ali HK, Martinelli G, Falantes J, Noppeney R, Stone RM, Minden MD, McIntyre H, Songer S, Lucy LM, Beach CL, Dohner H. International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts. Blood. 2015 Jul 16;126(3):291-9. doi: 10.1182/blood-2015-01-621664. Epub 2015 May 18.
PubMed 25987659Breems DA, Lowenberg B. Acute myeloid leukemia and the position of autologous stem cell transplantation. Semin Hematol. 2007 Oct;44(4):259-66. doi: 10.1053/j.seminhematol.2007.08.002.
PubMed 17961725Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Dohner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Dohner H. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. doi: 10.1056/NEJMoa1614359. Epub 2017 Jun 23.
PubMed 28644114Perl AE, Altman JK, Cortes J, Smith C, Litzow M, Baer MR, Claxton D, Erba HP, Gill S, Goldberg S, Jurcic JG, Larson RA, Liu C, Ritchie E, Schiller G, Spira AI, Strickland SA, Tibes R, Ustun C, Wang ES, Stuart R, Rollig C, Neubauer A, Martinelli G, Bahceci E, Levis M. Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study. Lancet Oncol. 2017 Aug;18(8):1061-1075. doi: 10.1016/S1470-2045(17)30416-3. Epub 2017 Jun 20.
PubMed 28645776DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, Swords R, Collins RH, Mannis GN, Pollyea DA, Donnellan W, Fathi AT, Pigneux A, Erba HP, Prince GT, Stein AS, Uy GL, Foran JM, Traer E, Stuart RK, Arellano ML, Slack JL, Sekeres MA, Willekens C, Choe S, Wang H, Zhang V, Yen KE, Kapsalis SM, Yang H, Dai D, Fan B, Goldwasser M, Liu H, Agresta S, Wu B, Attar EC, Tallman MS, Stone RM, Kantarjian HM. Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML. N Engl J Med. 2018 Jun 21;378(25):2386-2398. doi: 10.1056/NEJMoa1716984. Epub 2018 Jun 2.
PubMed 29860938Castaigne S, Pautas C, Terre C, Raffoux E, Bordessoule D, Bastie JN, Legrand O, Thomas X, Turlure P, Reman O, de Revel T, Gastaud L, de Gunzburg N, Contentin N, Henry E, Marolleau JP, Aljijakli A, Rousselot P, Fenaux P, Preudhomme C, Chevret S, Dombret H; Acute Leukemia French Association. Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): a randomised, open-label, phase 3 study. Lancet. 2012 Apr 21;379(9825):1508-16. doi: 10.1016/S0140-6736(12)60485-1. Epub 2012 Apr 5.
PubMed 22482940Cortes JE, Heidel FH, Hellmann A, Fiedler W, Smith BD, Robak T, Montesinos P, Pollyea DA, DesJardins P, Ottmann O, Ma WW, Shaik MN, Laird AD, Zeremski M, O'Connell A, Chan G, Heuser M. Randomized comparison of low dose cytarabine with or without glasdegib in patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Leukemia. 2019 Feb;33(2):379-389. doi: 10.1038/s41375-018-0312-9. Epub 2018 Dec 16.
PubMed 30555165Vey N, Keating M, Giles F, Cortes J, Beran M, Estey E. Effect of complete remission on survival in patients with acute myelogenous leukemia receiving first salvage therapy. Blood. 1999 May 1;93(9):3149-50. No abstract available.
PubMed 10336269Estey EH. Treatment of relapsed and refractory acute myelogenous leukemia. Leukemia. 2000 Mar;14(3):476-9. doi: 10.1038/sj.leu.2401568.
PubMed 10720145Siegel R, Ma J, Zou Z, Jemal A. Cancer statistics, 2014. CA Cancer J Clin. 2014 Jan-Feb;64(1):9-29. doi: 10.3322/caac.21208. Epub 2014 Jan 7.
PubMed 24399786Rowe JM, Buck G, Burnett AK, Chopra R, Wiernik PH, Richards SM, Lazarus HM, Franklin IM, Litzow MR, Ciobanu N, Prentice HG, Durrant J, Tallman MS, Goldstone AH; ECOG; MRC/NCRI Adult Leukemia Working Party. Induction therapy for adults with acute lymphoblastic leukemia: results of more than 1500 patients from the international ALL trial: MRC UKALL XII/ECOG E2993. Blood. 2005 Dec 1;106(12):3760-7. doi: 10.1182/blood-2005-04-1623. Epub 2005 Aug 16.
PubMed 16105981O'Brien S, Schiller G, Lister J, Damon L, Goldberg S, Aulitzky W, Ben-Yehuda D, Stock W, Coutre S, Douer D, Heffner LT, Larson M, Seiter K, Smith S, Assouline S, Kuriakose P, Maness L, Nagler A, Rowe J, Schaich M, Shpilberg O, Yee K, Schmieder G, Silverman JA, Thomas D, Deitcher SR, Kantarjian H. High-dose vincristine sulfate liposome injection for advanced, relapsed, and refractory adult Philadelphia chromosome-negative acute lymphoblastic leukemia. J Clin Oncol. 2013 Feb 20;31(6):676-83. doi: 10.1200/JCO.2012.46.2309. Epub 2012 Nov 19.
PubMed 23169518
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, First in Human, Open-label Study of Escalating Doses of INA03 Administered Intravenously as Single Agent in Adult Patients With Relapse/Refractory Acute Leukemia
- ClinicalTrials.gov ID
- NCT03957915
- Acronym / sponsor ID
- INA03 / INA03-IPC 2018-008
- Conditions
- Acute Lymphoblastic Leukemia Recurrent, Acute Lymphoblastic Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukaemia Recurrent, Mixed Phenotype Acute Leukemia
- Keywords
- acute leukemia, refractory, in relapse, antibody drug conjugate, first in human, escalating doses, INA03
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Institut Paoli-CalmettesUniversity, hospital or other
- Collaborators
- INATHERYS
- Study start
- 29 May 2020 (actual)
- Primary completion
- 15 Jul 2024 (actual)
- Study completion
- 24 Feb 2025 (actual)
- First posted
- 21 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL.
The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
Detailed description
This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL.
The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This Phase 1 Study is an open-label, non-randomized, dose escalation, safety, efficacy, pharmacokinetic, and pharmacodynamic evaluation study of INA03 administered as a single agent IV infusion every 2 weeks to patients ≥18 years of age with R/R AML, MLL, or ALL. The study will be performed in 2 parts: a Dose Titration for Day 1 study (Part 1) followed by a Dose Escalation Part (Part 2) of INA03 used as monotherapy.
| Arm | Type | Receives |
|---|---|---|
| INA03 | Experimental | Drug: INA03 administrationINA03 administration |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- . Patient with
- cytologically confirmed and documented B-cell or T-cell ALL or de novo, secondary or therapy-related AML or Mixed Phenotype Acute Leukemia (MPAL) defined according to World Health Organization (WHO) 2016 classification28 AND
- with 20% or more CD71 positive blast cells
- in relapse after- or refractory to registered therapies or ineligible to standard treatments
- with circulating blasts ≤ 20 000/mm3. For eligible patients with AML/ALL with blasts > 20000/mm3, a treatment with hydroxyurea is allowed to maintain tumor cells ≤ 20000/mm3 2. Male or female age ≥ 18 years 3. WHO performance status 0-2 4. Following laboratory values unless considered due to the leukemia:
Key exclusion criteria (11 listed)
- 7. Patients who have had a prior anaphylactic or other severe infusion reaction such that the patient is unable to tolerate human immunoglobulin or monoclonal antibody administration 8. Patients who have history or clinical evidence of central nervous system (CNS), meningeal, or epidural disease from any cause and/or peripheral neuropathy 9. Impaired cardiac function or clinically significant cardiac disease, including any one of the following:
- 14. Patients with prior radiation therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| INA03 administrationINA03 will be administered IV on Day 1, Day 14 of 28-day cycles. The administration of INA03 will begin at 0.02 mg/kg. Study Part I is a titration study to determine the dose for the first INA03 infusion. Patients will be enrolled in sequential cohorts of 2 patients to receive ascending starting doses of INA03, starting from the lowest starting dose (0.02 mg/kg), and followed by subsequent administrations of INA03 (D14 and beyond) at a fixed dose of 0.1 mg/kg. The starting dose will be increased every cohort of 2 patients until evidence of absence of marrow residual erythroblasts by D14 myelogram. This dose is referred to as the MEID and will be selected as the D1 dose for the study Part 2. Patient accumulation in Part I of the study will continue until no evidence of non-hematological DLT within 28 days post dosing | Drug | INA03 |
Primary outcome
Determination of the minimal erythroblastopenia-inducing dose (MEID) for INA03 in adults with refractory/relapsed acute leukemia
Time frame: within 2 weeks from initial dosing
MEID defined as the lowest dose associated with the risks of residual erythroblasts in the bone marrow at 2 weeks from initial dosing or grade 2 or above non hematologic toxicity within 2 weeks from initial dosing
Determination of the maximum tolerated dose (MTD) for subsequent administrations (D15 from initial dosing and beyond) for INA03 in adults with refractory/relapsed acute leukemia
Time frame: 28 days from the first administration of INA03
the maximum tolerated dose (MTD) for subsequent administrations (D15 from initial dosing and beyond)
7 secondary outcomes
Safety of INA03: NCI-CTCAE v5.0
Time frame: Until 30 days after last dose
pharmacokinetic (PK) profile of INA03
Time frame: From initial dosing day to Day 42
pharmacokinetic (PK) profile of INA03
Time frame: From initial dosing day to Day 42
pharmacokinetic (PK) profile of INA03
Time frame: From initial dosing day to Day 42
pharmacodynamics (PD) profile of INA03
Time frame: From screening to end of study visit (maximum 182 days)
Concentration of anti-INA03 antibodies
Time frame: From screening to end of study visit (maximum 182 days)
Preliminary clinical response of INA03
Time frame: from the date of treatment initiation (Day-1) to the date of relapse, progression or death, whichever comes first (a maximum 182 days)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2025.
- Background: PubMed 27371910Kell J. Considerations and challenges for patients with refractory and relapsed acute myeloid leukaemia. Leuk Res. 2016 Aug;47:149-60. doi: 10.1016/j.leukres.2016.05.025. Epub 2016 Jun 11.
- Background: PubMed 30275526Dohner H, Dolnik A, Tang L, Seymour JF, Minden MD, Stone RM, Del Castillo TB, Al-Ali HK, Santini V, Vyas P, Beach CL, MacBeth KJ, Skikne BS, Songer S, Tu N, Bullinger L, Dombret H. Cytogenetics and gene mutations influence survival in older patients with acute myeloid leukemia treated with azacitidine or conventional care. Leukemia. 2018 Dec;32(12):2546-2557. doi: 10.1038/s41375-018-0257-z. Epub 2018 Oct 1.
- Background: PubMed 20628485Eleni LD, Nicholas ZC, Alexandros S. Challenges in treating older patients with acute myeloid leukemia. J Oncol. 2010;2010:943823. doi: 10.1155/2010/943823. Epub 2010 Jun 10.
- Background: PubMed 25987659Dombret H, Seymour JF, Butrym A, Wierzbowska A, Selleslag D, Jang JH, Kumar R, Cavenagh J, Schuh AC, Candoni A, Recher C, Sandhu I, Bernal del Castillo T, Al-Ali HK, Martinelli G, Falantes J, Noppeney R, Stone RM, Minden MD, McIntyre H, Songer S, Lucy LM, Beach CL, Dohner H. International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts. Blood. 2015 Jul 16;126(3):291-9. doi: 10.1182/blood-2015-01-621664. Epub 2015 May 18.
- Background: PubMed 17961725Breems DA, Lowenberg B. Acute myeloid leukemia and the position of autologous stem cell transplantation. Semin Hematol. 2007 Oct;44(4):259-66. doi: 10.1053/j.seminhematol.2007.08.002.
- Background: PubMed 28644114Stone RM, Mandrekar SJ, Sanford BL, Laumann K, Geyer S, Bloomfield CD, Thiede C, Prior TW, Dohner K, Marcucci G, Lo-Coco F, Klisovic RB, Wei A, Sierra J, Sanz MA, Brandwein JM, de Witte T, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Krauter J, Schlenk RF, Ganser A, Serve H, Ehninger G, Amadori S, Larson RA, Dohner H. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation. N Engl J Med. 2017 Aug 3;377(5):454-464. doi: 10.1056/NEJMoa1614359. Epub 2017 Jun 23.
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- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Norbert VEY, Pr principal investigator
Institut Paoli-Calmettes
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing INA03 administration in people with Acute Lymphoblastic Leukemia Recurrent and Acute Lymphoblastic Leukemia, in Relapse, run by Institut Paoli-Calmettes at 3 sites in France.
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 13 inclusion and 11 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 6 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Determination of the minimal erythroblastopenia-inducing dose (MEID) for INA03 in adults with refractory/relapsed acute leukemia (time frame: within 2 weeks from initial dosing). The study lists 2 primary outcomes in all. 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 NCT03957915, which also lists 32 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 6 Aug 2025.