ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma
In ReNeu, the largest multicenter NF1-PN trial reported to date, mirdametinib treatment demonstrated significant confirmed ORRs by BICR, deep and durable PN volume reductions, and early, sustained, and clinically meaningful improvement in pain and HRQOL. Mirdametinib was well-tolerated in adults and children.
Authors
- Kevin Bielamowicz (ORCID: https://orcid.org/0000-0003-0790-2931)
- Carl Koschmann (ORCID: https://orcid.org/0000-0002-0825-7615)
- Rene McNall‐Knapp (ORCID: https://orcid.org/0000-0002-0416-7364)
- Jamie K. Capal (ORCID: https://orcid.org/0000-0002-7579-375X)
- Prakash Ambady (ORCID: https://orcid.org/0000-0003-4812-0581)
- Brian Andrew Van Tine (ORCID: https://orcid.org/0000-0003-4572-6668)
- Raju Antony
- Hans Shuhaiber
- Ana Aguilar-Bonilla (ORCID: https://orcid.org/0000-0002-5327-449X)
- Miriam Bornhorst (ORCID: https://orcid.org/0000-0002-7595-7726)
- L. Mary Smith
- Maciej M. Mrugała (ORCID: https://orcid.org/0000-0002-4027-7173)
- Cynthia Campen (ORCID: https://orcid.org/0000-0002-3519-1375)
- Punita Gupta (ORCID: https://orcid.org/0000-0002-7575-884X)
- Laura J. Klesse (ORCID: https://orcid.org/0000-0003-1323-7720)
- Alpa Sidhu (ORCID: https://orcid.org/0000-0002-8980-5029)
- Stefania Maraka (ORCID: https://orcid.org/0000-0001-5617-3320)
- Nathan Robison (ORCID: https://orcid.org/0000-0001-8613-9772)
- Nicholas K. Foreman (ORCID: https://orcid.org/0000-0003-4848-4801)
- David Schiff
- Tobias Walbert (ORCID: https://orcid.org/0000-0003-0667-8106)
- Ahmed M. Raslan
- Ryan D. Roberts (ORCID: https://orcid.org/0000-0001-5028-744X)
- Armend Lokku
- John M. Slopis (ORCID: https://orcid.org/0000-0002-1847-0583)
- Michael Weber (ORCID: https://orcid.org/0000-0002-8507-2219)
- Zsila Sadighi (ORCID: https://orcid.org/0000-0003-1686-7760)
- Angela C. Hirbe (ORCID: https://orcid.org/0000-0003-1719-0771)
- Phioanh Leia Nghiemphu (ORCID: https://orcid.org/0000-0003-4064-6079)
- David H. Viskochil (ORCID: https://orcid.org/0000-0001-5364-3366)
- Lauren Weintraub (ORCID: https://orcid.org/0009-0009-4406-1255)
- Daniela A. Bota (ORCID: https://orcid.org/0000-0002-9680-9060)
- Jonathan Gill (ORCID: https://orcid.org/0000-0001-7796-8632)
- Lindsay Kilburn (ORCID: https://orcid.org/0000-0003-1478-3619)
- Laura K. Metrock (ORCID: https://orcid.org/0000-0003-2185-7735)
- Reuben Antony (ORCID: https://orcid.org/0000-0003-1396-3917)
- Julia Meade (ORCID: https://orcid.org/0000-0001-8364-1820)
- Sarah Chagnon
- Andrea Franson (ORCID: https://orcid.org/0000-0002-5361-7683)
- Radhika Dhamija (ORCID: https://orcid.org/0000-0001-8052-2586)
- Nagma Dalvi
- Dusica Babovic‐Vuksanovic (ORCID: https://orcid.org/0000-0001-6981-1352)
- Kathryn Nevel (ORCID: https://orcid.org/0000-0003-1499-837X)
- Paul L. Moots (ORCID: https://orcid.org/0000-0002-2277-802X)
- Stacie Stapleton (ORCID: https://orcid.org/0000-0001-6459-4360)
- Fouad Hajjar
- Gurcharanjeet Kaur (ORCID: https://orcid.org/0000-0003-3552-1423)
- Leia Nghiemphu
- Ryan Merrell (ORCID: https://orcid.org/0000-0002-8076-0768)
- Timothy R. Gershon (ORCID: https://orcid.org/0000-0001-7034-6400)
- Christopher L. Moertel (ORCID: https://orcid.org/0000-0002-5474-588X)
- Andrew Walter (ORCID: https://orcid.org/0000-0002-5544-435X)
- Mary Lou Schmidt
- Ziad Khatib
Institutions
- Indiana University Health (US)
- Morgan Stanley Children's Hospital (US)
- Arkansas Children's Hospital (US)
- University of Minnesota (US)
- Nationwide Children's Hospital (US)
- Lucile Packard Children's Hospital (US)
- Montefiore Medical Center (US)
- Children's National (US)
- Children's Hospital of Los Angeles (US)
- The University of Texas MD Anderson Cancer Center (US)
- Johns Hopkins University (US)
- Orlando Health (US)
- Emory University (US)
- University of California, Los Angeles (US)
- Oregon Health & Science University (US)
- University of Pittsburgh (US)
- Wayne State University (US)
- University of California, Irvine (US)
- Washington University in St. Louis (US)
- University of Utah (US)
- University of Michigan (US)
- Columbia University Irving Medical Center (US)
- University of Iowa Hospitals and Clinics (US)
- University of Alabama at Birmingham (US)
- University of Florida (US)
- University of Illinois Chicago (US)
- Children's Hospital Colorado (US)
- Johns Hopkins All Children's Hospital (US)
- AdventHealth for Children (US)
- Mayo Clinic in Arizona (US)
- St. Joseph’s University Medical Center (US)
- University of Virginia Medical Center (US)
- Children's Hospital at Montefiore (US)
- Children's Hospital of The King's Daughters (US)
- Meridien Research (US)
- Miami Children's Hospital (US)
- Indiana University School of Medicine
- University of Oklahoma Health Sciences Center (US)
- Indiana University – Purdue University Indianapolis (US)
- Albany Medical Center Hospital (US)
- University of Arkansas for Medical Sciences (US)
- University of California, Davis (US)
- The University of Texas Southwestern Medical Center (US)
- Michigan State University (US)
- Vanderbilt University Medical Center (US)
- University of Colorado Denver (US)
Publication Details
- Journal
- Journal of Clinical Oncology
- Published
- 2024-11-08
- DOI
- https://doi.org/10.1200/jco.24.01034
- Citations
- 74
- Primary Topic
- Neurofibromatosis and Schwannoma Cases
- Type
- article
- Field-Weighted Citation Impact
- 27.31
Funders
- AstraZeneca
- Alexion Pharmaceuticals
- Deutsches Krebsforschungszentrum
- Jazz Pharmaceuticals
- Springworks Therapeutics
- Servier
- SymBio Pharmaceuticals Limited
- National Cancer Institute