Clinical trial NCT05918913: Expanded Access Program for Revumenib
Official title: Expanded Access Program for SNDX-5613 in Patients With Relapsed/Refractory Acute Leukemias With Genetic Alterations Associated With HOXA Overexpression
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Relapsed/Refractory Acute Leukemia
Keywords: SNDX-5613
Acute Leukemia
Refractory leukemia
Relapsed leukemia
KMT2A
NPM1
UBTF tandem duplication
Revumenib
Revuforj
Lead sponsor: Syndax Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2023-06-26
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT

Brief summary: This expanded access program will provide an investigational treatment option in a controlled clinical setting for participants who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options.


Eligibility criteria: Key Inclusion Criteria:

* Male or female participant aged ≥6 months.
* Not eligible for participation in an ongoing clinical study and have no approved treatment options.
* Participant or participant's health care proxy is able and willing to provide written informed consent and able to follow study instructions.
* Relapsed or refractory (R/R) acute leukemia, as defined by standardized criteria, after standard of care therapy, including but not limited to 1 or 2 cycles of intensive chemotherapy, or venetoclax combinations:

  1. R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
  2. Participants with persistent leukemia after initial therapy or recurrence of leukemia at any time after achieving a response during or after the course of treatment (including allogeneic hematopoietic stem cell transplant \[HSCT\]) are eligible.
  3. Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
  4. Participants previously treated on a revumenib clinical study who are entering the expanded access program for post-transplant maintenance because they are not eligible to receive revumenib on study or because the study is closed are not required to meet the R/R status.
  5. Participants who underwent HSCT and are eligible to resume treatment with revumenib will be dosed with the last revumenib tolerated dose before transplant.
* Acute leukemia harboring a lysine (K) methyltransferase 2A gene rearrangement (KMT2Ar), nucleoporin 98 rearrangement, nucleophosmin 1 mutation (or mutated) or any other genetic alteration with overexpression of HOXA genes predicted to potentially respond to menin inhibitors.

  -- Note: As revumenib is now approved in the United States, only participants with a KMT2Ar who are not included in the United States prescribing information indication or cannot be accurately dosed (within a 20% margin) with commercial supply and require use of oral solution will be allowed into the study.
* Adequate liver, renal, and cardiac function.
* Use of highly effective methods of contraception are required for females and males of childbearing potential from the time of enrollment through 120 days following the last study intervention dose.

For participants currently being treated with revumenib in a Syndax-sponsored clinical study or Syndax investigator-sponsored trial, the following criteria must be met:

* In the opinion of the Investigator, participant demonstrated acceptable benefit from and tolerability of the study intervention.
* Participant is considered compliant with study intervention and procedures.
* Participant does not meet any criteria for study intervention discontinuation.
* Investigator and participant agree to continue study intervention treatment.
* Participant continues to experience clinical benefit.

Key Exclusion Criteria:

* Evidence of uncontrolled infection.
* Pregnant or nursing women.
* Cardiac or gastrointestinal disease.
* Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD \>Grade 1 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 1 week before enrollment, with the exception of steroids.
* History of or any concurrent condition, therapy, laboratory abnormality, or allergy to excipients that, in the Investigator's opinion, either may interfere with the participant's participation or results in the conclusion that it is not in the best interest of the participant to participate.
* Participants receiving other antileukemic therapy within 14 days of start of study drug and who have not recovered from previous adverse reactions.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Duarte
country: United States
facility: City of Hope
state: California
city: Irvine
country: United States
facility: City of Hope at Orange County Lennar Foundation Cancer Center
state: California
city: Los Angeles
country: United States
facility: UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital-Stanford
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: Center for Cancer and Blood Disorders, Colorado Children's Hospital
state: Colorado
city: Aurora
country: United States
facility: HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
state: Colorado
city: Orlando
country: United States
facility: Advent Health Orlando
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Atlanta
country: United States
facility: Winship Cancer Institute at Emory University
state: Georgia
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: New Orleans
country: United States
facility: Children's Hospital
state: Louisiana
city: Boston
country: United States
facility: Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospital-Kansas City
state: Missouri
city: Saint Louis
country: United States
facility: Siteman Cancer Center - Washington University
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Long Island City
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cincinnati
country: United States
facility: Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
state: Ohio
city: Columbus
country: United States
facility: OSU Medical Center
state: Ohio
city: Portland
country: United States
facility: Doernbecher Children's Hospital, Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Houston
country: United States
facility: Memorial Hermann-Texas Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Research Institute, Seattle Childrens Hospital
state: Washington
Study officials:
References: