Pilot study of the safety and toxicity of acquiring hyperpolarized carbon-13 imaging in children with brain tumors

Trial Requirement/Treatment

This is an open label trial to assess the safety and tolerability of the adult-tolerated dose of hyperpolarized pyruvate for metabolic imaging in children with brain tumors who do not require sedation for their MR imaging. Patients will receive a single MR imaging examination that includes the acquisition of hyperpolarized 13C metabolic data in combination with anatomic, diffusion, perfusion and lactate edited 1H spectroscopic imaging data. The data will be processed using custom designed software to estimate changes in levels of lactate/pyruvate and to relate them to abnormalities observed in the data from other MR modalities.

Rationale for Study

The results of this study will provide the safety data required to move this type of metabolic imaging into therapeutic trials to assess the utility of HP 13C lactate/pyruvate as a new surrogate marker of drug tumor penetration and early response to therapy in children with brain tumors. The availability of such imaging markers for determining whether treatment is successfully modulating the tumor or whether alternative approaches should be considered will be critical for a disease area with limited therapy options.

Primary Objectives

The primary objective of this study is to determine the safety and tolerability of the established adult dose of hyperpolarized 13C in children ≥ 3 years and ≤ 18 years of age with brain tumors who do not require sedation for MR imaging.

Secondary Objectives

To assess imaging quality and define the most appropriate imaging parameters for obtaining HP 13C data from pediatric brain.

Eligibility Criteria

Patients must have baseline evaluations performed prior to the first dose of study drug and must meet all inclusion and exclusion criteria. In addition, the patient must be thoroughly informed about all aspects of the study, including the study visit schedule and required evaluations and all regulatory requirements for informed consent. The written informed consent must be obtained from the patient prior to enrollment. The following criteria apply to all patients enrolled onto the study unless otherwise specified.

Inclusion Criteria

• Children ≥ 3 years and ≤ 18 years of age with a diagnosis of a brain tumor and who do not require sedation for MR imaging

• Karnofsky ≥ 70 for patients ≥ 16 years of age, and Lansky ≥ 70 for patients < 16 years of age (See Appendix 1 Performance Status Criteria)

• Patients must not have any significant medical illnesses that in the investigator’s opinion cannot be adequately controlled with appropriate therapy, would compromise the patient’s ability to tolerate the imaging examination or any disease that will obscure toxicity or dangerously alter response to the imaging agent

• Patients must not be pregnant or breast feeding. Women of childbearing potential are required to obtain a negative pregnancy test within 14 days of starting treatment. Effective contraception (men and women) must be used in subjects of child-bearing potential

• Ability to understand and the willingness of the patient, parent or legal guardian to provide informed consent

Exclusion Criteria

• Patients who are not able to comply with study and/or follow-up procedures

• Patients receiving active therapy on an investigational trial at the time of enrollment should consult with the study chair regarding potential interactions with other study agents. Patients who are enrolled in a clinical trial but are off- therapy and in follow up are eligible

• Patients with history or evidence of cardiac dysfunction

How to Enroll

If you believe your child or patient is eligible for this trial, contact the closest participating site for more information or contact us at info@pnoc.us.