Clinical trial NCT05024929: Targeted Therapy to Increase RAI Uptake in Metastatic DTC
Official title: Targeted Therapy to Increase RAI Uptake in Patients With Metastatic DifferentiatedThyroid Cancer
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Papillary Thyroid Cancer
Pediatric Cancer
Differentiated Thyroid Cancer
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
Collaborators: name: United States Department of Defense
First posted: 2021-08-27
Enrollment: 32
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will enroll children and adolescents with differentiated thyroid cancer with pulmonary metastases who are planned to receive oncogene-specific targeted therapy.

Brief summary: Papillary thyroid cancer (PTC) is a common type of differentiated thyroid cancer (DTC) in children and represents the second most common cancer in adolescent females. Recently targeted drugs that block many of the genetic drivers of DTC have become available. While Investigators know that these drugs shrink DTC tumors in many cases, the impact on radioactive iodine (RAI) avidity has not been systematically studied.

Detailed description: This is an observational cohort study that will enroll patients with DTC metastatic to the lungs who will receive oncogene-specific targeted therapy as part of routine clinical care or a separate therapeutic clinical trial. After approximately 4 weeks of therapy, patients will have a whole body scan to determine the change in RAI-avidity of their tumor from baseline. Subsequent therapy will be at the discretion of the treating physician or according to the therapeutic trial on which the patient is enrolled.

Eligibility criteria: Inclusion Criteria (Prospective Cohort):

1. Patients with a histologic diagnosis of differentiated thyroid cancer
2. Presence of an neurotrophic tyrosine kinase receptors (NTRK)-fusion, RET-fusion, anaplastic lymphoma kinase (ALK)-fusion, BRAF V600 mutation, BRAF-fusion or other targetable alteration identified in a Clinical Laboratory Improvement Amendments/College of American Pathologists (CLIA/CAP) laboratory
3. Anatomically evaluable disease on chest Computed tomography (CT) meeting oneo f the following criteria (obtained within 180 days of enrollment):

   1. multiple (10 or more) noncalcified solid pulmonary nodules visible on CT and/or
   2. enlarging, discrete pulmonary nodules visible on CT of any number consistent with metastatic disease
4. Patients for whom systemic therapy with an oncogene-specific kinase inhibitor is planned from commercial supply or as part of a separate therapeutic clinical trial (that does not include data sharing with this protocol)/compassionate access protocol/single patient investigational new drug (IND). Such agents include, but are not limited to:

   1. Larotrectinib, entrectinib, selitrectinib, and repotrectinib for NTRK fusions
   2. Selpercatinib and pralsetinib for RET fusions
   3. Crizotinib, lorlatinib, repotrectinib, and alectinib for ALK fusions
   4. Dabrafenib and/or trametinib for BRAF V600 mutations
   5. Oncogene-specific kinase inhibitors other than those specifically delineated above must be approved by the overall study PI prior to enrollment

Inclusion Criteria (Data Sharing Cohort):

1. Patients enrolled on other oncogene-specific targeted therapy trials who undergo whole body thyroid scan approximately 28 days after beginning targeted therapy and agree to data sharing as part of the consent process for that trial.

Exclusion Criteria (All Cohorts):

1. No prior oncogene-specific targeted therapy allowed. However, patients may enroll within 4 weeks of starting oncogene-specific therapy if a pre-therapy WBS is available. Prior therapy with non-oncogene specific multi-thyrosine kinase inhibitors (such as sorafenib, lenvatinib, and/or cabozantanib) is allowed.
2. Females who are pregnant or breastfeeding are excluded due to the potential risks of the RAI used in the WBS to the fetus/neonate.
3. Patients who require sedation/general anesthesia to complete a WBS are excluded.
4. U.S. Military Personnel are excluded due to the Children's Hospital of Philadelphia (CHOP) Institutional Review Board (IRB) requirements.

Primary outcomes:
measure: Proportion of patients with increased tumor RAI-avidity after receiving oncogene-specific, targeted therapy
timeFrame: up to 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Sydney
country: Australia
facility: Children's Hospital Westmead
state: New South Wales
Study officials:
References: