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New treatment option for Rhabdomyosarcoma
Official title FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma
ClinicalTrials.gov ID: NCT06865664
What this study is testing
What is fludarabine?
fludarabine is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for rhabdomyosarcoma.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: Rhabdomyosarcoma (RMS) is a cancer of soft tissues. It is the most common soft tissue sarcoma seen in children.
- Phase 1: an early, usually small safety study
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 39
You may be able to join if
- Histologically confirmed rhabdomyosarcoma by the NCI Department of Pathology. Note: Since FGFR4 expression is universal in rhabdomyosarcoma...
- Relapsed or refractory rhabdomyosarcoma after at least two (2) cancer treatment regimens i.e., participants should have relapsed or progressed after...
- No available alternative curative therapies per standard of care.
- Participants must have measurable disease per RECIST 1.1 or non-measurable disease on imaging.
- Age \>= 3 and \<= 39 years old.
You likely can't join if
- Prior therapy with the following prior to apheresis:
- tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen within \<= 1 week
- systemic chemotherapy within \<= 2 weeks
- antineoplastic antibody therapy, checkpoint inhibitors, or vaccine therapy, within \<= 3 weeks or 5 half-lives (whichever is shorter)
- radiation within \ =50% of bony pelvis and \<=12 weeks in case of total body irradiation). Note: There is no time restriction if the volume of bone...
- any investigational agents within \<= 4 weeks
See the full eligibility criteria
Who can join
- Histologically confirmed rhabdomyosarcoma by the NCI Department of Pathology. Note: Since FGFR4 expression is universal in rhabdomyosarcoma, confirmation of FGFR4 expression is not required.
- Relapsed or refractory rhabdomyosarcoma after at least two (2) cancer treatment regimens i.e., participants should have relapsed or progressed after upfront therapy (that includes any systemic chemotherapy with or...
- No available alternative curative therapies per standard of care.
- Participants must have measurable disease per RECIST 1.1 or non-measurable disease on imaging.
- Age \>= 3 and \<= 39 years old.
- Weight \>=15 kg.
- Performance status: Karnofsky \>= 50% (\>= 16 years) or Lansky \>= 50% (\< 16 years). Note: Participants who are unable to walk because of paralysis, but who are upright in a wheelchair will be considered ambulatory for...
- Participants must be willing to accept blood transfusions.
- Adequate organ and marrow function as defined below:
- Organ: Bone Marrow Function\
- Laboratory Element: Absolute neutrophil count; Minimum Requirement \>= 500/mcL
- Laboratory Element: Platelets; Minimum Requirement \>= 50,000/mcL \ Transfusion independent (defined as no transfusion in the prior 7 days) for participants without bone marrow involvement. Participants who have bone...
- Organ: Liver Function
- Laboratory Element: Aspartate aminotransferase (AST); Minimum Requirement \<= 5 x upper limit of normal (ULN)
- Laboratory Element: Alanine aminotransferase (ALT); Minimum Requirement \<= 5 x ULN
- Laboratory Element: Total bilirubin; Minimum Requirement \<= 2 x ULN (Note: Participants with Gilbert's syndrome and/or bilirubin elevation due to tumor involvement are allowed to have \<= 5 x ULN) Note: Adult values...
- Age: 3 to \< 6 years; Maximum serum creatinine (mg/dL): Male - 0.8, Female - 0.8
- Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): Male - 1, Female - 1
- Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): Male - 1.2, Female - 1.2
- Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): Male - 1.5, Female - 1.2
- Age: \>= 16 years; Maximum serum creatinine (mg/dL): Male - 1.7, Female - 1.4 OR
- Measured or calculated creatinine clearance or glomerular filtration rate (GFR); Minimum Requirement: \>= 60mL/min/1.73 m\^2 --Organ: Cardiac Function
- Laboratory Element: Cardiac status; Minimum Requirement: Cardiac ejection fraction \>= 45% or shortening fraction \>= 28%, pericardial effusion \<= grade 2 as determined by an echocardiogram (ECHO)
- Organ: Pulmonary Function
- Laboratory Element: Pulmonary status; Minimum Requirement: Pleural effusion \ =92% on room air at rest --Organ: Neurological Function
- Laboratory Element: Neurologic status; Minimum Requirement: No acute neurotoxicity greater than grade 2 per CTCAE v.5.0 with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible.
- Individuals of child-bearing potential (IOCBP) must agree to use highly effective contraception (hormonal, intrauterine device [IUD], abstinence, surgical sterilization) at the study entry and up to 12 months after the...
- Nursing participants must be willing to discontinue nursing from study treatment initiation through 4 months after completion of chemotherapy preparative administration or 6 months after FGFR4-CAR T cells infusion...
- Participants with previous central nervous system (CNS) tumor involvement that has been treated and is stable for at least 6 weeks following completion of therapy as evidenced by no requirements for corticosteroids, no...
- Participants must be willing to be enrolled into protocol 15C0028 "Follow-Up Evaluation for Gene-Therapy Related Delayed Adverse Events after Participation in Pediatric Oncology Branch Clinical Trials" after 5 years on...
- The ability of participant or parent/guardian to understand and the willingness to sign a written informed consent document.
What rules you out
- Prior therapy with the following prior to apheresis:
- tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen within \<= 1 week
- systemic chemotherapy within \<= 2 weeks
- antineoplastic antibody therapy, checkpoint inhibitors, or vaccine therapy, within \<= 3 weeks or 5 half-lives (whichever is shorter)
- radiation within \ =50% of bony pelvis and \<=12 weeks in case of total body irradiation). Note: There is no time restriction if the volume of bone marrow treated is less than 10% and the participant has...
- any investigational agents within \<= 4 weeks
- autologous stem cell infusion following myeloablative therapy within \<= 6 weeks
- genetically modified T cell, NK cell, or dendritic cell therapy within \<= 6 weeks
- allogeneic stem cell transplant/infusion within \<=12 weeks or evidence of active graft versus host disease (GVHD)
- Participants receiving more than physiologic dosing of systemic steroids (3 mg/m\^2/day of prednisone equivalent).
- History of severe, immediate hypersensitivity reaction attributed to any agents used in the study or in the manufacturing of the cells.
- Second malignancy at any time.
- Primary immunodeficiency.
- Seropositive for human immunodeficiency virus (HIV) antibody.
- Seropositive for hepatitis C (HCV) or positive for Hepatitis B (HBV) surface antigen (HbsAg).
- Pregnancy confirmed with beta-HCG serum or urine pregnancy test performed in IOCBP at screening.
- Uncontrolled intercurrent illness or social situations that would limit compliance with study requirements.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 39 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.