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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04730349




Registration number
NCT04730349
Ethics application status
Date submitted
26/01/2021
Date registered
29/01/2021
Date last updated
24/03/2023

Titles & IDs
Public title
A Study of Bempegaldesleukin (BEMPEG: NKTR-214) in Combination With Nivolumab in Children, Adolescents and Young Adults With Recurrent or Treatment-resistant Cancer
Scientific title
Phase 1/2 Study of Bempegaldesleukin in Combination With Nivolumab in Children, Adolescents, and Young Adults With Recurrent or Refractory Malignancies (PIVOT IO 020)
Secondary ID [1] 0 0
2020-000854-85
Secondary ID [2] 0 0
CA045-020
Universal Trial Number (UTN)
Trial acronym
PIVOT IO 020
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ependymoma 0 0
Ewing Sarcoma 0 0
High-grade Glioma 0 0
Leukemia and Lymphoma 0 0
Medulloblastoma 0 0
Miscellaneous Brain Tumors 0 0
Miscellaneous Solid Tumors 0 0
Neuroblastoma 0 0
Relapsed, Refractory Malignant Neoplasms 0 0
Rhabdomyosarcoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Cancer 0 0 0 0
Sarcoma (also see 'Bone') - soft tissue
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other
Cancer 0 0 0 0
Children's - Brain

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Nivolumab
Treatment: Other - NKTR-214

Experimental: A1W Dosing schema -

Experimental: A1F Dosing schema -

Experimental: A2W Dosing schema -

Experimental: A2F Dosing schema -

Experimental: Part B: Cohort B1 Neuroblastoma -

Experimental: Part B: Cohort B2 Ewing sarcoma -

Experimental: Part B: Cohort B3 Rhabdomyosarcoma -

Experimental: Part B: Cohort B4 Miscellaneous solid tumors -

Experimental: Part B: Cohort B5 NHL/leukemia -

Experimental: Part B: Cohort B6 High-grade glioma -

Experimental: Part B: Cohort B7 Medulloblastoma and Embryonal Tumors -

Experimental: Part B: Cohort B8 Ependymoma -

Experimental: Part B: Cohort B9 Miscellaneous brain tumors -


Treatment: Other: Nivolumab
Specified dose on specified days

Treatment: Other: NKTR-214
Specified dose on specified days
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Dose-Limiting Toxicities (DLTs) - Part A
Timepoint [1] 0 0
From first dose to 42 days after first dose
Primary outcome [2] 0 0
Number of Participants With Adverse Events (AEs) - Part A
Timepoint [2] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [3] 0 0
Number of Participants With Serious Adverse Events (SAEs) - Part A
Timepoint [3] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [4] 0 0
Number of Participants With Drug-Related Adverse Events - Part A
Timepoint [4] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [5] 0 0
Number of Participants With Adverse Events Leading to Discontinuation - Part A
Timepoint [5] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [6] 0 0
Number of Participants Who Died - Part A
Timepoint [6] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [7] 0 0
Maximum Observed Plasma Concentration (Cmax) - Part A
Timepoint [7] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [8] 0 0
Trough Observed Concentration (Ctrough) - Part A
Timepoint [8] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)
Primary outcome [9] 0 0
Area Under the Plasma Concentration (AUC) - Part A
Timepoint [9] 0 0
From first dose to 30 days after last dose (up to approximately 6 months)

Eligibility
Key inclusion criteria
* Age < 18 years for Part A and Part B
* Age up to 30 years for Part B Cohorts B2, B3 and B4
* Must have received standard of care therapy and there must be no potentially curative treatment available
* Histologically confirmed with malignant neoplasms that are refractory, relapsed, or curative treatments are lacking
* Must have measurable or evaluable disease based on Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 for solid tumors, Response Assessment in Neuro-Oncology (RANO) or Response Assessment in Pediatric Neuro-Oncology (RAPNO) for central nervous system tumors, International Pediatric Non-Hodgkin Lymphoma Response Criteria for non-Hodgkin lymphoma (NHL), revised International Neuroblastoma Response Criteria (INRC) for neuroblastoma, modified National Comprehensive Cancer Network (NCCN) Criteria for acute lymphoblastic leukemia, and modified Cheson et al International Working Group criteria for acute myeloid leukemia
* Lansky play score for age = 16 years or Karnofsky performance score for age > 16 years assessed within 2 weeks of enrollment must be = 60
Minimum age
No limit
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Osteosarcoma, T-cell/Natural Killer (NK) cell leukemia/lymphoma, and Hodgkin's lymphoma
* Need for > 2 antihypertensive medications for management of hypertension (including diuretics)
* Known cardiovascular history, including unstable or deteriorating cardiac disease, within the previous 12 months prior to screening
* Inadequately treated adrenal insufficiency
* Active, known, or suspected autoimmune disease
* Active infection requiring systemic therapy within 14 days prior to first dose
* Condition requiring systemic treatment with either corticosteroids or other immunosuppressive medications within 14 days of start of study treatment
* Prior allogeneic stem cell transplant
* Previous severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection either suspected or confirmed within 4 weeks prior to screening

Other protocol-defined inclusion/exclusion criteria apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/06/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/06/2022
Sample size
Target
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Local Institution - 0001 - Randwick
Recruitment hospital [2] 0 0
Local Institution - South Brisbane
Recruitment hospital [3] 0 0
Local Institution - 0002 - Parkville
Recruitment hospital [4] 0 0
Local Institution - 0003 - Perth
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment postcode(s) [4] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
Missouri
Country [3] 0 0
France
State/province [3] 0 0
Val-de-Marne
Country [4] 0 0
France
State/province [4] 0 0
Lyon
Country [5] 0 0
France
State/province [5] 0 0
Marseille
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
Germany
State/province [7] 0 0
Hamburg
Country [8] 0 0
Germany
State/province [8] 0 0
Tuebingen
Country [9] 0 0
Germany
State/province [9] 0 0
Wuerzburg
Country [10] 0 0
Italy
State/province [10] 0 0
Milan
Country [11] 0 0
Spain
State/province [11] 0 0
Madrid, Comunidad De
Country [12] 0 0
Spain
State/province [12] 0 0
Barcelona
Country [13] 0 0
Spain
State/province [13] 0 0
Sevilla
Country [14] 0 0
Spain
State/province [14] 0 0
València

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bristol-Myers Squibb
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Nektar Therapeutics
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to first, in Part A, assess the safety, tolerability and drug levels of Bempegaldesleukin (BEMPEG) in combination with nivolumab and then, in Part B, to estimate the preliminary efficacy in children, adolescents and young adults with recurrent or treatment-resistant cancer.
Trial website
https://clinicaltrials.gov/study/NCT04730349
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bristol-Myers Squibb
Address 0 0
Bristol-Myers Squibb
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04730349