Study of Nivolumab and Ipilimumab in Children and Young Adults With INI1-Negative Cancers

STATUS

Recruiting
End date

Oct 5, 2025
participants needed

45
sponsor

Dana-Farber Cancer Institute

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Updated on 19 February 2024

See if I qualify

cancer

blood transfusion

renal function

measurable disease

neutrophil count

nivolumab

monoclonal antibodies

growth factor

sarcoma

small molecule

karnofsky performance status

immunohistochemistry

cancer treatment

ipilimumab

kidney function tests

monoclonal protein

cancer therapy

epithelioid sarcoma

biological therapy

lipase

myelosuppressive chemotherapy

rhabdoid tumor

pulse oximetry

biologics

hcg pregnancy test

dyspnea at rest

serum lipase

chordoma

samarium

Summary

This clinical trial is studying two immunotherapy drugs (nivolumab and ipilimumab) given together as a possible treatment for INI1-negative tumors.

Description

This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug or drug combination to learn whether the drug or drug combination works in treating a specific disease. "Investigational" means that the drug combination is being studied.

The names of the study drugs involved in this study are:

Nivolumab (OPDIVO)
Ipilimumab (YERYOY)

This trial is studying whether nivolumab and ipilimumab work to treat INI1-negative cancers.

The U.S. Food and Drug Administration (FDA) has not approved combination nivolumab and ipilimumab for the specific diseases in this study but it has been approved for other diseases. Nivolumab and ipilimumab have been tested in children to find out a safe dose of this combination.

Details

Condition	Connective and Soft Tissue Neoplasm, Solid Tumors, Rhabdoid Tumor, Sarcoma, Sarcoma, Germ cell tumor, Chordoma, Chordoma, Solid Tumors, Rhabdoid Tumor of the Kidney, Atypical Teratoid/Rhabdoid Tumor, Other INI1 Negative Malignant Tumors (With PI Approval)
Age	1years - 30years
Treatment	Nivolumab, Ipilimumab
Clinical Study Identifier	NCT04416568
Sponsor	Dana-Farber Cancer Institute
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	All participants must have one of the following histologically confirmed tumors at original diagnosis or relapse
	Stratum 1
	Malignant rhabdoid tumor (MRT)
	Rhabdoid tumor of the kidney (RTK)
	Epithelioid sarcoma
	Chordoma (poorly differentiated or de-differentiated)
	Other INI1-negative malignant tumors (with PI approval)
	Stratum 2
	Atypical teratoid rhabdoid tumor (ATRT)
	Other INI1-negative primary CNS malignant tumors (with PI approval)
	All participants must have tumor assessment at original diagnosis or relapse showing the following
	Loss of INI1 confirmed by immunohistochemistry (IHC), OR
	Molecular confirmation of tumor bi-allelic SMARCB1 (INI1) loss or mutation when INI1 IHC is equivocal or unavailable
	Relapsed or refractory disease and no standard treatment options as determined by locally or regionally available standards of care and treating physician's discretion
	Measurable disease as defined by RECIST v1.1 (Stratum 1) or RANO criteria (Stratum 2)
	Karnofsky performance status 50% for participants 16 years of age and Lansky performance status 50% for participants <16 years of age
	Participants must have fully recovered from the acute toxic effects of all prior anti-cancer therapy. Participants must meet the following minimum washout periods prior to first day of study treatment
	Myelosuppressive chemotherapy: At least 14 days after the last dose of myelosuppressive chemotherapy
	Radiotherapy
	At least 14 days after local palliative XRT (small port)
	At least 90 days must have elapsed after prior TBI, craniospinal XRT or if >50% radiation of pelvis
	At least 42 days must have elapsed if other substantial BM radiation
	At least 42 days must have passed since last radionuclide therapy (e.g. samarium or radium)
	Small molecule biologic therapy: At least 7 days following the last dose of a nonmonoclonal biologic agent
	Monoclonal antibody: At least 21 days after the last dose
	Myeloid growth factors: At least 14 days following the last dose of long-acting growth factor (e.g. Neulasta) or 7 days following short-acting growth factor
	Stem Cell or Autologous T Cell Infusion: At least 42 days must have elapsed after stem cell or autologous T cell infusion
	Participants must have adequate organ function as defined below
	Bone Marrow Function
	Absolute neutrophil count 500/uL
	Hemoglobin 8 g/dL and transfusion independent
	Platelets 50,000/uL and transfusion independent
	Hepatic Function
	Total bilirubin 1.5 x upper limit of normal for age
	ALT (SGPT) 3 x upper limit of normal
	Renal function
	A serum creatinine within protocol limits based on age/sex. OR
	Creatinine clearance 70 mL/min/1.73 m2 for participants with creatinine levels above institutional normal
	Adequate Pulmonary Function Defined as: no evidence of dyspnea at rest, no exercise intolerance due to pulmonary insufficient and a pulse oximetry > 92% while breathing room air
	Adequate pancreatic function defined as serum lipase ULN at baseline
	Negative B-HCG pregnancy test in females of childbearing potential (must be drawn within 24 hours prior to initial administration of nivolumab)
	Women of childbearing potential (WOCBP) receiving nivolumab agree to adhere to contraception for a period of 5 months after the last dose of nivolumab. Men receiving nivolumab and who are sexually active with WOCBP will agree to adhere to contraception for a period of 7 months after the last dose of nivolumab
	Ability to understand and/or the willingness of the patient (or parent or legally authorized representative, if minor) to provide informed consent using an institutionally approved informed consent procedure
Exclusion Criteria
	Participants who are receiving any other investigational agents
	Participants must not be receiving concomitant systemic steroid medications The use of physiologic doses of corticosteroids (up to 5 mg/m2/day prednisone equivalent) may be approved after consultation with the PI (treatment with topical, inhaled or ophthalmic corticosteroid is acceptable)
	Participants with a known history of HIV, hepatitis B, and/or hepatitis C
	Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection or any other concurrent disease which in the judgment of the Investigator would make the subject inappropriate for enrollment on this study
	Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis
	Has active autoimmune disease that has required systemic treatment in the past 12 months, or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Subjects with vitiligo or resolved childhood asthma/atopy are exceptions. Intermittent use of bronchodilators or local steroid injections are not excluded. Replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment. Autoimmune diagnoses not listed must be approved by the Principal Investigator
	Patients who have received prior solid organ transplantation are not eligible
	Pregnancy or Breast-Feeding. Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal
	Has received prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-L2 or anti-CTLA4 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (e.g. OX-40, CD137)
	Participants who have received live / attenuated vaccine within 30 days of first dose of study treatment

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Study Definition

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Study of Nivolumab and Ipilimumab in Children and Young Adults With INI1-Negative Cancers