Can a Gene-Targeted pill turn the tide against a rare blood cancer?

NCT ID NCT03503409

Disease control Sponsor: Groupe Francophone des Myelodysplasies Source: ClinicalTrials.gov ↗

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This trial is testing an oral drug called AG-120 (ivosidenib) in adults with myelodysplastic syndrome (MDS) or a related early form of leukemia that carries a specific IDH1 gene mutation. The drug is designed to block the mutated enzyme that drives abnormal blood cell growth. Researchers are looking to see if the treatment can improve blood cell counts and slow the progression of the disease in patients who have not responded to or have relapsed after standard therapy.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: AG-120 (ivosidenib), an oral drug that targets cancer cells with a specific IDH1 gene mutation
What this could lead to: If successful, this could offer a new targeted treatment option for people with myelodysplastic syndrome who have an IDH1 mutation and have not responded to standard therapy.
What could go wrong: This is a phase II trial, so the drug may not prove effective or safe in a larger population. Side effects are possible, and the benefit may be limited to certain patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 68 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2019

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients must meet all of the following criteria to participate in the study: * Age ≥ 18 years * Myelodysplastic syndrome according to WHO classification including non-proliferative AML up to 29% of BM blast * Belonging to one of the following categories : * higher risk (IPSS high or int 2 ) MDS without response to azacitidine (CR,PR, stable disease with HI) after at least 6 cycles , or relapsing after a response but without overt progression (defined by at least doubling of marrow blasts, compared to pre azacitidine bone marrow, or AML progression beyond 30% blasts) * Untreated higher risk MDS (IPSS int-2, high) without life threatening cytopenia including ANC \<500/mm3 or any recent severe infections and /or platelets below 30,000/mm3 or any bleeding symptom * lower risk MDS with resistance or loss of response to a previous treatment with epoetin alpha/ beta (≥60000 U/w) or Darbopoetin (≥250 ug/w) given for at least 12 weeks and RBC transfusion requirement at least 2 U/8 weeks in the previous 16 weeks * Presence of IDH1 mutation in either blood or marrow prior to start of therapy; * Normal renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance (Modification of diet in renal disease) creatinine clearance ≥ 50 mL/min; * Normal liver function, defined by total bilirubin and transaminases less than 1.5 times the upper limit of normal; * Adequate cardiac ejection fraction (\>40%); * Patient is not known to be refractory to platelet transfusions; * Written informed consent; * Patient must understand and voluntarily sign consent form. * Patient must be able to adhere to the visit schedule as outlined in the study and follow protocol requirements; * ECOG performance status 0-2 at the time of screening; * Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy. Subjects with reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy or tubal occlusion or who have not been naturally postmenopausal (i.e., who have not menstruated at all) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months). Females of reproductive potential as well as fertile men and their partners who are female of reproductive potential must agree to abstain from sexual intercourse or to use two highly effective forms of contraception from the time of giving informed consent, during the study and for 3 months (females and males) following the last dose of AG-120. A highly effective form of contraception is defined as hormonal oral contraceptives, injectables, patches, intrauterine devices. * Male patients must : * Agree the need for the use of a condom if engaged in sexual activity with a woman of childbearing potential during the entire period of treatment, even if disruption of treatment and during 3 months after end of treatment. * Agree to learn about the procedures for preservation of sperm before starting treatment Exclusion Criteria: * A patient meeting any of the following criteria is not eligible to participate in the study: * Severe infection or any other uncontrolled severe condition. * Significant cardiac disease - NYHA Class III or IV or having suffered a myocardial infarction in the last 6 months. * Less than 14 days since prior treatment with growth factors (EPO, G-CSF). * Use of investigational agents within 30 days or any anticancer therapy within 2 weeks before the study entry with the exception of hydroxyurea. The patient must have recovered from all acute toxicity from any previous therapy. * Subject has a heart-rate corrected QT interval using Fridericia's method (QTcF) ≥ 470 msec or any other factor that increases the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome). Subjects with prolonged QTcF interval in the setting of bundle branch block may participate in the study. * Subject is taking known strong cytochrome P450 (CYP) 3A4 inducers or inhibitors or sensitive CYP3A4 substrate medications with a narrow therapeutic window, unless they can be transferred to other medications within ≥ 5 half-lives prior to dosing. * Subject is taking P-glycoprotein (P-gp) transporter-sensitive substrate medications with a narrow therapeutic window, unless they can be transferred to other medications within ≥ 5 half-lives prior to administration of study treatment * Active cancer or cancer during the year prior to trial entry other than basal cell carcinoma, or carcinoma in situ of the cervix or breast. * Patient already enrolled in another therapeutic trial of an investigational drug. * Known HIV infection or active hepatitis B or C. * Women who are or could become pregnant or who are currently breastfeeding. * Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form. * Patient eligible for allogeneic stem cell transplantation. * Known allergies to AG 120 or any of its excipients. * The study does not provide for the inclusion of persons referred to in Articles L. 1121-5 to L. 1121-9 and L. 1122-1-2 of the Public Health Code (e.g. minors, protected adults, etc.) * No affiliation to a health insurance system.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute myeloid leukemia Myelodysplastic syndromes

Contacts and locations

Locations

CH Angers

Angers, 49933, France
CH Le Mans/Service d'hématologie Oncologie

Le Mans, 72000, France
CHRU de Limoges

Limoges, 87046, France
CHU Brabois

Vandœuvre-lès-Nancy, 54511, France
CHU Hôtel Dieu/Service d'Hématologie Clinique

Nantes, 44093, France
CHU Montpellier St Eloi

Montpellier, 34295, France
CHU côte de Nacre

Caen, 14033, France
CHU de Grenoble/Clinique Universitaire d'hématologie 6e A

Grenoble, 38043, France
CHU de Haut-Lévèque/Centre François Magendie/Service des maladies du sang

Pessac, 33604, France
CHU de Poitiers/Pôle de cancérologie - secteur tertiaire-

Poitiers, 86021, France
CHU de Tours

Tours, 37044, France
CLINICA Ematologica ANCONA

Ancona, 7160126, Italy
Centre Henri Becquerel

Rouen, 76038, France
Centre Hospitalier de Versailles-Hôpital André Mignot

Versailles, 78157, France
Centre Hospitalier de la Côte Basque

Bayonne, 64109, France
Clinica Ematologica Genova

Genova, 1016132, Italy
Ematologia ALESSANDRIA

Alessandria, 16115121, Italy
Ematologia BOLOGNA

Bologna, 40138, Italy
Ematologia BRESCIA

Brescia, 125123, Italy
Ematologia FIRENZE

Florence, 350134, Italy
Ematologia GENOVA

Genova, 1016132, Italy
Ematologia LECCE

Lecce, 73100, Italy
Ematologia MILANO

Milan, 3520122, Italy
Ematologia ORBASSANO

Orbassano, 1010043, Italy
Ematologia ROMA

Roma, 1000144, Italy
Ematologia ed Immunologia Clinica PADOVA

Padova, 235128, Italy
GHU Caremeau

Nîmes, 30029, France
Hôpital Archet 1/Service d'Hématologie Clinique

Nice, 06200, France
Hôpital E. Muller-GHR Mulhouse Sud-Alsace

Mulhouse, 68100, France
Hôpital Henri Mondor

Paris, 94010, France
Hôpital Necker

Paris, 75743, France
Hôpital Nord Franche-Comté/Service de médecine interne / Hématologie clinique

Belfort, 90015, France
Hôpital Saint Louis - Hématologie Séniors

Paris, 75010, France
Institut Paoli Calmettes/Unité d'Hématologie 3

Marseille, 13273, France
Médecine Interne/IUCT Oncopole

Toulouse, 31059, France
Reggio Calabria

Reggio Calabria, 2189124, Italy
centre hospitalier de Lyon

Lyon, 69495, France
institut de cancérologie Lucien Neuwirth

Saint-Priest-en-Jarez, 42271, France

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