KiTZ Clinical Trial Unit

Head: Prof. Dr. med. Olaf Witt
Deputy head: Prof. Dr. med. Stefan Pfister

Im Neuenheimer Feld 430
D-69120 Heidelberg

Combination of Niraparib and Dostarlimab for Solid Tumors

"A Phase 1, Multicentre, Open-label, Dose-Escalation and Cohort Expansion Study of Niraparib und Dostarlimab in Paediatric Patients with Recurrent or Refractory Solid Tumors (SCOOP)"

This study will evaluate the combination of a poly (adenosine diphosphate-ribose) polymerase (PARP) inhibitor, niraparib, with the programmed cell death protein 1 (PD-1) inhibitor, dostarlimab in the pediatric population. This study will be conducted to determine the recommended Phase 2 dose (RP2D) and evaluate the pharmacokinetics (PK), safety, and efficacy of niraparib in combination with dostarlimab in pediatric participants with recurrent or refractory solid tumors (excluding brain tumors).

Niraparib inhibits the function of the enzyme PARP (poly [adenosine diphosphate ribose] polymerase) in the body. As part of the study (part 1), tumors are therefore treated whose genetic information carries a specific signature called "BRCAness." Dostarlimab is a monoclonal antibody from the group of checkpoint inhibitors and is intended to strengthen the body's immune response to the tumor tissue and thus contribute to reduced tumor growth.

Niraparib is taken daily in the form of tablets or liquid suspension, dostarlimab is given as an infusion, always on the 1st day of a 21-day treatment cycle.

Inclusion of patients in Heidelberg has been possible since 03 March 2022.

Important inclusion criteria

For Part 1:

Participant has recurrent or refractory osteosarcoma, neuroblastoma, adrenocortical carcinoma, Ewing sarcoma, rhabdomyosarcoma, or any other solid tumor (excluding tumors of the central nervous system [CNS]) found to have a “BRCAness” signature
Age ≥ 6 months to less than < 18 years old
Karnofsky-/Lansky-Score > 60

For Part 2 (osteosarcoma expansion cohort):

recurrent or refractory osteosarcoma
Age ≥ 6 months to less than < 18 years old
Karnofsky-/Lansky-Score > 60
radiographically measurable disease

For Part 2 (neuroblastoma expansion cohort):

recurrent or refractory neuroblastoma
Age ≥ 6 months to less than < 18 years old
Karnofsky-/Lansky-Score > 60
radiographically measurable disease at the time of study enrolment; participants with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine-positive (+) evaluable disease are eligible

Further information

For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (ClinicalTrials.gov).
See NCT04544995.

EudraCT-number: 2020-002359-39 (see EU clinical trial register).