Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors
A Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS Tumors
5 other identifiers
interventional
29
1 country
12
Brief Summary
This phase I trial studies the side effects and best dose of pomalidomide in treating younger patients with tumors of the brain or spine (central nervous system) that have come back or are continuing to grow. Pomalidomide may interfere with the ability of tumor cells to grow and spread and may also stimulate the immune system to kill tumor cells.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Jul 2015
Longer than P75 for phase_1
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 13, 2015
CompletedFirst Posted
Study publicly available on registry
April 14, 2015
CompletedStudy Start
First participant enrolled
July 14, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
May 27, 2020
CompletedApril 25, 2022
April 1, 2022
4 years
April 13, 2015
April 22, 2022
Conditions
Outcome Measures
Primary Outcomes (2)
Maximum tolerated dose/recommended phase II dose of pomalidomide
Will be defined as the highest dose level at which 6 patients have been treated with at most 1 experiencing dose-limiting toxicity and the next higher dose level has been determined to be too toxic (\>= 2 dose-limiting toxicity). All safety data will be presented by dose cohort (intended dose) within each stratum separately. Adverse events will be tabulated by grade and attribution to the study agent.
Up to 28 days
Pharmacokinetics parameters of pomalidomide
Population estimates of pharmacokinetics parameters for pomalidomide will be estimated, and intra- and inter-subject variability of these parameters will be characterized. The effect of demographics/covariates (e.g., age, body weight, gender, prior treatment and use of concomitant medications, etc.) on the pharmacokinetics of pomalidomide will be evaluated. Descriptive statistics provided as appropriate. Plasma drug concentrations and pharmacokinetic parameters will be presented in tabular and graphical form.
Pre-dose, 0.5, 1, 2, 4, 8, and 24 hours post-dose day 1 of course 1; 1 sample pre-dose any day between days 3-21 of course 1
Secondary Outcomes (3)
Response rate (complete response, partial response, and stable disease)
Up to 2 years
Duration of response
The time from the initial documented response (complete response, partial response or long-term stable disease) to the first confirmed progressed disease, assessed up to 2 years
Event-free survival
The time from study enrollment until the time of progressive disease, second-(ary) malignancy or death from any cause on study treatment, assessed up to 2 years
Other Outcomes (2)
Change in levels of biologic correlates
Baseline to up to day 21
Change in levels of immunologic correlates
Baseline to up to day 21
Study Arms (1)
Treatment (pomalidomide)
EXPERIMENTALPatients receive pomalidomide PO QD on days 1-21. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.
Interventions
Given PO
Eligibility Criteria
You may qualify if:
- Patients must have received standard therapy (or generally accepted upfront therapy if no standard exists) and have no known curative therapy
- Patients with a histologically confirmed diagnosis of a primary CNS tumor that is recurrent, progressive or refractory to standard therapy; refractory disease will be defined as the presence of persistent abnormality on conventional magnetic resonance imaging (MRI) imaging that is further distinguished by histology (biopsy or sample of lesion) or advanced imaging, OR as determined by the treating physician and discussed with the primary investigator prior to enrollment; all tumors must have histological verification at either the time of diagnosis or recurrence except for patients with diffuse intrinsic brain stem tumors or optic pathway gliomas; patients with neurofibromatosis type-I (NF-1) associated CNS tumors are eligible if they meet all other eligibility criteria
- Patients must have evaluable disease on MRI imaging
- Patients must have body surface area (BSA) \> 0.55 m\^2 at the time of enrollment
- In the event of de-escalation from dose level 1 to dose level 0, patients with BSAs \< 0.67 m\^2 are not eligible
You may not qualify if:
- Agents that potentially fit into more than one category or do not clearly fit into any category listed above should be discussed with the study principal investigator (PI) prior to enrollment
- Patients must have received their last dose of known myelosuppressive anticancer therapy greater than 28 days prior to study enrollment or \> 42 days if nitrosourea
- Patients must have received their last dose of any other investigational agent greater than 28 days prior to enrollment (with exception of fluorothymidine F-18 \[FLT\])
- Patients must have received their last dose of any other biologic agent greater than 7 days prior to enrollment
- Patients must have received their last dose of any other biologic agent greater than 7 days prior to enrollment
- For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur and discussed with the PI
- Monoclonal antibody treatment and agents with known prolonged half-lives: at least three half-lives must have elapsed prior to enrollment
- Immunomodulatory therapy: greater than 28 days must have elapsed since last dose of an immune modulating agent, including vaccine therapy
- Administration of the radioisotope, 18-FLT, which is being concurrently investigated on an imaging study within the Pediatric Brain Tumor Consortium (PBTC), is allowed \> 72 hours prior to initiation of pomalidomide on this study; any adverse events related to the FLT must have resolved completely
- Patients must have had their last fraction of:
- Craniospinal irradiation, total body irradiation (TBI), or \>= 50% radiation of pelvis \> 3 months prior to enrollment
- Focal irradiation \> 6 weeks prior to enrollment
- Local palliative radiation therapy (XRT) (small port) \>= 4 weeks
- Patient must be:
- \>= 6 months since allogeneic bone marrow transplant prior to enrollment
- +44 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611, United States
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
National Cancer Institute
Rockville, Maryland, 20850, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Jason R Fangusaro
Pediatric Brain Tumor Consortium
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 13, 2015
First Posted
April 14, 2015
Study Start
July 14, 2015
Primary Completion
July 1, 2019
Study Completion
May 27, 2020
Last Updated
April 25, 2022
Record last verified: 2022-04