NCT03503409 is a Phase 2 clinical trial of AG-120 in Myelodysplastic Syndromes, sponsored by Groupe Francophone des Myelodysplasies.

Who is sponsoring NCT03503409?

NCT03503409 is sponsored by Groupe Francophone des Myelodysplasies.

What drugs are being tested in NCT03503409?

Interventions in NCT03503409: AG-120.

What condition does NCT03503409 study?

NCT03503409 studies Myelodysplastic Syndromes, Acute Myeloid Leukemia.

Is NCT03503409 still recruiting?

NCT03503409 is currently active, enrolled. Last updated 18 May 2025.

Last reviewed 2025-05-18 · How we verify

NCT03503409

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic Syndrome

Active, enrolled Phase 2 Last updated 18 May 2025

What this trial tests

Phase 2 trial testing AG-120 in Myelodysplastic Syndromes in 68 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

14 May 2019

Primary endpoint
2 April 2027

2 April 2027

Quick facts

Lead sponsor	Groupe Francophone des Myelodysplasies
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	68
Start date	14 May 2019
Primary completion	2 April 2027
Estimated completion	2 April 2027
Sites	38 locations across France, Italy

Drugs / interventions tested

AG-120 — full drug profile →

Conditions studied

Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →
Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

Groupe Francophone des Myelodysplasies — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes or Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

patients with MDS (Myelodysplastic Syndrome) and mutated IDH1 patients will be treated with AG120 (IDH1 inhibitor)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Altered metabolism in cancer: insights into energy pathways and therapeutic targets.
Tufail M, Jiang CH, Li N. · · 2024 · cited 265× · PMID 39294640 · DOI 10.1186/s12943-024-02119-3
Mutant Isocitrate Dehydrogenase Inhibitors as Targeted Cancer Therapeutics.
Golub D, Iyengar N, Dogra S, Wong T, et al · · 2019 · cited 176× · PMID 31165048 · DOI 10.3389/fonc.2019.00417
Targeting multiple signaling pathways: the new approach to acute myeloid leukemia therapy.
Carter JL, Hege K, Yang J, Kalpage HA, et al · · 2020 · cited 148× · PMID 33335095 · DOI 10.1038/s41392-020-00361-x
Ivosidenib: First Global Approval.
Dhillon S. · · 2018 · cited 112× · PMID 30209701 · DOI 10.1007/s40265-018-0978-3
Cancer-associated mutation and beyond: The emerging biology of isocitrate dehydrogenases in human disease.
Tommasini-Ghelfi S, Murnan K, Kouri FM, Mahajan AS, et al · · 2019 · cited 110× · PMID 31131326 · DOI 10.1126/sciadv.aaw4543
Epigenetic regulation in hematopoiesis and its implications in the targeted therapy of hematologic malignancies.
Zhao A, Zhou H, Yang J, Li M, et al · · 2023 · cited 81× · PMID 36797244 · DOI 10.1038/s41392-023-01342-6
Current challenges and unmet medical needs in myelodysplastic syndromes.
Platzbecker U, Kubasch AS, Homer-Bouthiette C, Prebet T. · · 2021 · cited 67× · PMID 34045662 · DOI 10.1038/s41375-021-01265-7
Advances in targeted therapy for acute myeloid leukemia.
Yu J, Jiang PYZ, Sun H, Zhang X, et al · · 2020 · cited 48× · PMID 32477567 · DOI 10.1186/s40364-020-00196-2

Verify or expand the search:

Other trials of AG-120

Trials testing the same drug.

NCT03839771 — A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Mainten · Phase 3 · active not recruiting
NCT03343197 — Study of AG-120 and AG-881 in Subjects With Low Grade Glioma · Phase 1 · completed
NCT03173248 — Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute · Phase 3 · active not recruiting
NCT02989857 — Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy) · Phase 3 · completed
NCT02677922 — A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapie · Phase 1, PHASE2 · active not recruiting

Other recruiting trials for Myelodysplastic Syndromes

Currently open trials in the same condition.

NCT07566377 — Cord Blood Transplantation in Children and Young Adults With Blood Cancer · Phase 2 · recruiting
NCT06303193 — Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or · Phase 1, PHASE2 · recruiting
NCT07071155 — Momelotinib in Combination With Hypomethylating Agent for Chronic Phase Myelodysplastic Syndromes/Myeloproliferative Ove · EARLY_PHASE1 · recruiting
NCT07283900 — Ascorbate in Myelodysplastic Syndrome · Phase 2 · recruiting
NCT06487247 — HEME Home Transfusion Program · NA · recruiting

Other Groupe Francophone des Myelodysplasies trials

Trials by the same sponsor.

NCT05226455 — Venetoclax in Patients With MDS or AML in Relapse After AHSCT · Phase 1, PHASE2 · active not recruiting
NCT05181735 — Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA · Phase 1, PHASE2 · recruiting
NCT04857645 — ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients · Phase 2 · completed
NCT04827719 — BST-236 as a Single Agent in Adults With Relapsed or Refractory AML or HR-MDS · Phase 2 · completed
NCT04273802 — CPX-351 in Higher Risk Myelodysplastic Syndromes · Phase 1, PHASE2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03503409 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Groupe Francophone des Myelodysplasies
Last refreshed: 18 May 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03503409.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing