NCT03042689 is a Phase 1 clinical trial of Regorafenib in Acute Myeloid Leukemia, sponsored by Massachusetts General Hospital.

Who is sponsoring NCT03042689?

NCT03042689 is sponsored by Massachusetts General Hospital.

What drugs are being tested in NCT03042689?

Interventions in NCT03042689: Regorafenib.

What condition does NCT03042689 study?

NCT03042689 studies Acute Myeloid Leukemia.

Is NCT03042689 still recruiting?

NCT03042689 is currently completed. Last updated 8 January 2026.

Last reviewed 2026-01-08 · How we verify

NCT03042689

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

Study of Regorafenib in Patients With Advanced Myeloid Malignancies

Completed Phase 1 Last updated 8 January 2026

What this trial tests

Phase 1 trial testing Regorafenib in Acute Myeloid Leukemia in 16 participants. Completed in 27 February 2023.

Timeline

17 April 2017

Primary endpoint
1 September 2021

27 February 2023

Quick facts

Lead sponsor	Massachusetts General Hospital
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	17 April 2017
Primary completion	1 September 2021
Estimated completion	27 February 2023
Sites	1 location across United States

Drugs / interventions tested

Regorafenib — full drug profile →

Conditions studied

Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

Massachusetts General Hospital

Who can join

18 and older, any sex, with Acute Myeloid Leukemia. 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 advanced myeloid malignancies including AML (acute myeloid leukemia), MDS (myelodysplastic syndrome) and MPN (myeloproliferative neoplasms) The intervention involved in this study is: -Regorafenib (Stivarga)

Publications & conference data

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

Regorafenib combined with PD1 blockade increases CD8 T-cell infiltration by inducing CXCL10 expression in hepatocellular carcinoma.
Shigeta K, Matsui A, Kikuchi H, Klein S, et al · · 2020 · cited 118× · PMID 33234602 · DOI 10.1136/jitc-2020-001435
Downregulation of MTSS1 in acute myeloid leukemia is associated with a poor prognosis, chemotherapy resistance, and disease aggressiveness.
Grandits AM, Nguyen CH, Schlerka A, Hackl H, et al · · 2021 · cited 11× · PMID 33782537 · DOI 10.1038/s41375-021-01224-2

Verify or expand the search:

Other trials of Regorafenib

Trials testing the same drug.

NCT06902246 — Regorafenib and Yttrium-90 Radioembolization for Unresectable Hepatocellular Carcinoma · Phase 2 · recruiting
NCT07392866 — A Clinical Study of SH006 Injection in Combination Therapy Versus Regorafenib in the Treatment of Advanced Hepatocellula · Phase 2, PHASE3 · not yet recruiting
NCT06820957 — Testing a New Combination of Anti-cancer Drugs in Patients Newly Diagnosed With Ewing Sarcoma Who Have Cancer That Has S · Phase 2, PHASE3 · active not recruiting
NCT07223567 — A Trial of Regorafenib Plus Lorigerlimab in Patients With Locally Recurrent or Regrowing pMMR/MSS Localized Rectal Cance · Phase 2 · withdrawn
NCT07134205 — The Study of JMT101 Combined With Irinotecan as a ≥3rd-Line Treatment in Metastatic Colorectal Cancer · Phase 3 · not yet recruiting

Other recruiting trials for Acute Myeloid Leukemia

Currently open trials in the same condition.

NCT07020533 — A Vaccine (CMV-MVA Triplex Vaccine) for the Enhancement of CMV-Specific Immunity and the Prevention of CMV Viremia in Pa · Phase 1 · recruiting
NCT06782542 — Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for I · Phase 2 · recruiting
NCT07177079 — High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML · Phase 1 · recruiting
NCT07384715 — First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelod · Phase 1 · recruiting
NCT07107126 — Safety and Proof-of-Concept Study of RPT1G in Adults With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes · Phase 1 · 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 NCT03042689 (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: 8 January 2026

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/NCT03042689.

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