NCT03564821 is a Phase 1 clinical trial of Ivosidenib in IDH1 Mutation Myeloid Neoplasms, sponsored by Massachusetts General Hospital.

Who is sponsoring NCT03564821?

NCT03564821 is sponsored by Massachusetts General Hospital.

What drugs are being tested in NCT03564821?

Interventions in NCT03564821: Ivosidenib.

What condition does NCT03564821 study?

NCT03564821 studies IDH1 Mutation Myeloid Neoplasms.

Is NCT03564821 still recruiting?

NCT03564821 is currently completed. Last updated 4 November 2025.

Last reviewed 2025-11-04 · How we verify

NCT03564821

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

IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation

Completed Phase 1 Last updated 4 November 2025

What this trial tests

Phase 1 trial testing Ivosidenib in IDH1 Mutation Myeloid Neoplasms in 18 participants. Completed in 6 June 2023.

Timeline

16 January 2019

Primary endpoint
22 May 2022

6 June 2023

Quick facts

Lead sponsor	Massachusetts General Hospital
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	16 January 2019
Primary completion	22 May 2022
Estimated completion	6 June 2023
Sites	4 locations across United States

Drugs / interventions tested

Ivosidenib (IVOSIDENIB) — full drug profile →

Conditions studied

IDH1 Mutation Myeloid Neoplasms — all drugs for IDH1 Mutation Myeloid Neoplasms →

Sponsor

Massachusetts General Hospital

Who can join

18 and older, any sex, with IDH1 Mutation Myeloid Neoplasms. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This research study is studying a drug as a possible treatment for IDH1-mutant myeloid neoplasms. -The drug involved in this study is ivosidenib (AG-120)

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
Recent advances in targeted therapies in acute myeloid leukemia.
Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
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
The Graft-Versus-Leukemia Effect in AML.
Sweeney C, Vyas P. · · 2019 · cited 86× · PMID 31803612 · DOI 10.3389/fonc.2019.01217
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

Verify or expand the search:

Other trials of Ivosidenib

Trials testing the same drug.

NCT06707493 — Ivosidenib as Post-HSCT Maintenance for AML · Phase 2 · recruiting
NCT06501625 — Ivosidenib Plus Durvalumab and Gemcitabine/Cisplatin as First-Line Therapy in Participants With Locally Advanced or Meta · Phase 1, PHASE2 · recruiting
NCT06465953 — Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelod · Phase 3 · recruiting
NCT07131059 — MRD-positive AML Clinical Study · NA · recruiting
NCT07282262 — An Exploratory Study on the Use of Ivosidenib for the Precise Treatment of Advanced Biliary Tract Malignancies With IDH1 · Phase 2 · recruiting

Other Massachusetts General Hospital trials

Trials by the same sponsor.

NCT03585946 — Outcomes in Stevens Johnsons Syndrome and Toxic Epidermal Necrolysis · withdrawn
NCT07214831 — A Feasibility and Acceptability Study of a Large Language Model-based Chatbot for Brief Alcohol Intervention Among Emerg · NA · not yet recruiting
NCT06686901 — A Randomized Controlled Trial of a Smartphone Delivered Treatment for Suicidal Thoughts and Behavior · NA · not yet recruiting
NCT05854212 — Behavioral Economics to Implement Nutrition Ranking in Food Pantries · NA · not yet recruiting
NCT07323446 — The MIND Study: The MGH/MIT Investigation of NAC on Dysregulation · EARLY_PHASE1 · not yet recruiting

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 NCT03564821 (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 Massachusetts General Hospital
Last refreshed: 4 November 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/NCT03564821.

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