The Importance of Clinical Trials in Rare Cancer Types
In the realm of oncology, clinical trials are essential for advancing treatment options, particularly for rare cancer types. While clinical research has greatly improved the treatment of more common cancers, rare cancers often do not receive the same level of attention. This is where the importance of clinical trials becomes particularly evident. Clinical trials help researchers understand the unique characteristics of rare cancers, develop targeted therapies, and improve patient outcomes.

Rare cancers, by definition, affect a small population of individuals, which makes it difficult to gather large-scale data through traditional methods. However, through well-designed clinical trials, researchers can gain critical insights into these cancers’ biology, progression, and response to treatments. Clinical trials can lead to the identification of new biomarkers and therapeutic targets that are crucial for more personalized and effective care. Moreover, they provide access to leading-edge treatments that may not yet be available through standard protocols.
Moffitt is uniquely positioned to treat rare and complex cancers, with physicians focused on specific and rare disease. Our commitment to expanding clinical trials and improving outcomes for rare cancer patients is evident through our collaborative research efforts, which bring together clinicians and scientists to develop novel therapeutic approaches.
The research teams here have pioneered advancements in rare cancer treatment, focusing on cancers such as mesothelioma, sarcoma, and rare neuroendocrine tumors. Our institution's robust clinical trial infrastructure allows for rapid evaluation of emerging therapies and provides patients with access to potentially life-saving treatments. Moffitt is also at the forefront of integrating precision medicine into clinical trials, using genomic information to design individualized treatment plans for patients with rare cancers.
We deeply value our partnerships with referring providers and are committed to working together to ensure the best possible outcomes for patients, especially those diagnosed with rare cancers. Our goal is not to extend treatment unnecessarily but to provide leading-edge, innovative therapies that are most appropriate for each patient's needs. When the time is right, we work diligently to return patients to care closer to home, ensuring they continue to receive the necessary treatments without disruption.
By collaborating on clinical trials and sharing our expertise, we can jointly advance treatment options and improve the quality of life and prognosis for both current and future patients with rare cancers.
Some of the Rare Cancer Trials Currently Open:
Cancer Type: Cutaneous
An Open-Label, Multicenter, Phase 1b/2 Study of RP1 in Solid Organ Transplant Recipients with Advanced Cutaneous Malignancies
Clinical Trial 21996 | CTOCutaneous@moffitt.org
Cancer Type: BMT
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Haploidentical Related, Partially HLA-Mismatched, or Matched Unrelated Bone Marrow for Newly Diagnosed Patients with Severe Aplastic Anemia
Clinical Trial 23284 | CTOBMT@moffitt.org
Cancer Type: Gastrointestinal Tumor
A Prospective, Randomized, Controlled, Open-label, Multicenter Trial to Evaluate Efficacy, Safety and Patient-reported Outcomes of Peptide Receptor Radionuclide Therapy (PRRT) with Lutetium (177Lu) Edotreotide compared to Best Standard of Care in Patients with Well-differentiated Aggressive Grade 2 and Grade 3, Somatostatin Receptor-positive (SSTR+), Neuroendocrine Tumors of GastroEnteric or Pancreatic Origin (COMPOSE)
Clinical Trial 21383 | CTOGI@Moffitt.org
Cancer Type: Genitourinary
A Phase 2 Study of the Combination Dostarlimab with Niraparib In Patients with Penile Carcinoma Who Have Progressed Following Chemotherapy
Clinical Trial 21192 | CTOGU@moffitt.org
Cancer Type: Gynecological Tumor
Phase 2 Study of Alpelisib and Fulvestrant for PIK3CA-mutated Estrogen Receptor (ER)-positive Endometrioid Endometrial Cancers
Clinical Trial 23067 | CTOGYN@Moffitt.org
Cancer Type: Malignant Hematology – Lymphoma
A Single-Arm, Open-Label, Pilot Study of Concurrent Phototherapy and POTELIGEO (mogamulizumab-kpkc) in Early Stage Mycosis Fungoides (PLIGHT)
Clinical Trial 21143 | CTOHemeLymphomaMyeloma@Moffitt.org
Cancer Type: Malignant Hematology – Myeloid
A Phase 1 Study of Avapritinib in Combination with Decitabine in Patients with Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
Clinical Trial 22318 | CTOHemeMyeloid@moffitt.org
Cancer Type: Neurology
A First in Human dose escalation of Dendritic Cell Vaccine (DCV) administered Intrathecally (IT) primed against HER2/HER3 in Patients with Leptomeningeal Disease (LMD) from Triple-Negative Breast Cancer (TNBC) or HER2+ Breast Cancer (HER2+BC)
Clinical Trial 21262 | CTOneuro@Moffitt.org
Cancer Type: Radiation Oncology
Phase I/II Study of Radiation Therapy Followed by Intrathecal Trastuzumab/Pertuzumab in the Management of HER2+ Breast Leptomeningeal Disease
Clinical Trial 20487 | CTORadOnc@moffitt.org
Cancer Type: Sarcoma
An International, Phase 3, Randomized, Multicenter, Open-label Study of Ripretinib vs Sunitinib in Patients with Advanced Gastrointestinal Stromal Tumor (GIST) with KIT Exon 11 and Co-occurring KIT Exons 17 and/or 18 Mutations Who Were Previously Treated with Imatinib (INSIGHT)
Clinical Trial 23228 | CTOSarcoma@Moffitt.org
Cancer Type: Thoracic
A Multicenter, Open-label, Phase II Study to Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC (Uncommon EGFR Mutations Only)
To inquire more about clinical trials for rare cancer types or to refer a patient with cancer or a suspicious tumor finding, please complete our online form or contact a physician liaison for assistance. As part of our efforts to shorten referral times as much as possible, online referrals are typically responded to within 24 - 48 hours.