Pediatric Brain TumorsPhase 1NCT02947373
Pilot Study of Safety and Toxicity of Acquiring Hyperpolarized Carbon-13 Imaging in Children With Brain Tumors
A Phase 1 study testing Hyperpolarized Pyruvate in people with Pediatric Brain Tumors, run by University of California, San Francisco at 1 site in United States.
Trial summary
This is a single arm pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The pilot study will look at the safety and toxicity of acquiring hyperpolarized carbon-13 imaging in children with brain tumors. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Pediatric Brain Tumors
- What is being tested?
- Hyperpolarized Pyruvate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jan 2017to 31 Jul 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: PNOC 011: Pilot Study of Safety and Toxicity of Acquiring Hyperpolarized Carbon-13 Imaging in Children With Brain Tumors
This is a single arm pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC).
The pilot study will look at the safety and toxicity of acquiring hyperpolarized carbon-13 imaging in children with brain tumors.
Read the detailed description
This is an open label trial to assess the safety and tolerability of the adult tolerated dose of Hyperpolarized Pyruvate (HP) for metabolic imaging in children with brain tumors who do not require sedation for their MR imaging. Nine 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.
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.
- Conditions
- Pediatric Brain Tumors
- Being tested
- Hyperpolarized Pyruvate
- Keywords
- brain tumor, children
- Medical subject headings
- Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hyperpolarized PyruvateDrug
Hyperpolarized Pyruvate
| Study group | Role | What they receive |
|---|---|---|
| Imaging Arm | Other | Patients will receive a one-time injection of Hyperpolarized Pyruvate prior to a single MR imaging examination that includes the acquisition of HP carbon-13 metabolic data. Receives: Drug: Hyperpolarized Pyruvate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events
Dose Limiting Toxicities will be assessed by monitoring for adverse events, scheduled laboratory assessments, vital sign measurements, ECGs, and physical examinations. The severity of the toxicities will be graded according to the NCI CTCAE v4.0. Adverse events and clinically significant laboratory abnormalities will be summarized by maximum intensity and relationship to study drug. Safety will be assessed during the infusion and at least for one hour after completion of the infusion as well as by phone 24 hours after the infusion. Descriptive statistics will be utilized to display the data on toxicity seen. Analyses will be performed for all patients having received at least one dose of study drug.
Measured: 9 months
Secondary outcomes (1)
Secondary analyses will include assessment of imaging quality, which will be descriptive in nature.
13C data will be acquired using the following sequence parameters;
- a volumetric acquisition from a 4-5cm slice with 2D phase encoding and 1-D Echo-Planar Spectroscopic Imaging (EPSI) encoding, field of view (FOV) 20x24x10cm (1cc voxel size),
- 1H scout; A further set of scout images will be obtained to re-establish appropriate landmarks.
- High resolution anatomic imaging: These require a 3D localizing scan to define the graphical prescription;
- Diffusion Images: This will be followed by diffusion tensor spin echo single shot echo planar images, 6 gradient directions, 22cm FOV, 128x128 matrix,
- Lactate edited 3D magnetic resonance spectroscopic imaging (MRSI): water suppressed 1H magnetic resonance spectroscopy (MRS) with PRESS volume selection, out-of-voxel suppression with very spatially selective rf pulses, echo planar encoding in the signal intensity (SI) direction and 2D in-plane phase encoding
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2016 (estimated)
- Study start25 Jan 2017
- Primary completion30 Apr 2020
- Study completion31 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Brain TumorsStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sabine Mueller, MD, PhD, University of California, San Francisco
- Investigators
- Sabine Mueller, MD, PhD, MAS, Study chair, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02947373
- Sponsor's protocol ID
- PNOC 011
- Other IDs
- 16088
- Status last verified
- Jun 2021
- First submitted
- 24 Oct 2016
- Last updated
- 24 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PNOC 011: Pilot Study of Safety and Toxicity of Acquiring Hyperpolarized Carbon-13 Imaging in Children With Brain Tumors
- ClinicalTrials.gov ID
- NCT02947373
- Acronym / sponsor ID
- PNOC 011
- Conditions
- Pediatric Brain Tumors
- Keywords
- brain tumor, children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Study start
- 25 Jan 2017 (actual)
- Primary completion
- 30 Apr 2020 (actual)
- Study completion
- 31 Jul 2020 (actual)
- First posted
- 27 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single arm pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC).
The pilot study will look at the safety and toxicity of acquiring hyperpolarized carbon-13 imaging in children with brain tumors.
Detailed description
This is an open label trial to assess the safety and tolerability of the adult tolerated dose of Hyperpolarized Pyruvate (HP) for metabolic imaging in children with brain tumors who do not require sedation for their MR imaging. Nine 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.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Imaging Arm | Other | Drug: Hyperpolarized PyruvatePatients will receive a one-time injection of Hyperpolarized Pyruvate prior to a single MR imaging examination that includes the acquisition of HP carbon-13 metabolic data. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hyperpolarized PyruvateHyperpolarized Pyruvate | Drug | Imaging Arm |
Primary outcome
Number of Participants With Adverse Events
Time frame: 9 months
Dose Limiting Toxicities will be assessed by monitoring for adverse events, scheduled laboratory assessments, vital sign measurements, ECGs, and physical examinations. The severity of the toxicities will be graded according to the NCI CTCAE v4.0. Adverse events and clinically significant laboratory abnormalities will be summarized by maximum intensity and relationship to study drug. Safety will be assessed during the infusion and at least for one hour after completion of the infusion as well as by phone 24 hours after the infusion. Descriptive statistics will be utilized to display the data on toxicity seen. Analyses will be performed for all patients having received at least one dose of study drug.
1 secondary outcome
Secondary analyses will include assessment of imaging quality, which will be descriptive in nature.
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sabine Mueller, MD, PhD, MAS study chair
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Hyperpolarized Pyruvate in people with Pediatric Brain Tumors, run by University of California, San Francisco at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2021.