Locations

Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients

Posted on

This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy.

MIBG for Refractory Neuroblastoma and Pheochromocytoma

Posted on

This is a best available therapy/compassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who are not eligible for therapies of higher priority. Patients may receive a range of doses depending on stem cell availability and tumor involvement of bone marrow. Response rate, toxicity, and time to progression and death will be evaluated.

Myeloablative Consolidation Therapy and Tandem Autologous Stem Cell Rescue in Patients With High-Risk Neuroblastoma

Posted on

This is a phase II single center study to administer two courses of myeloablative consolidation chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in patients with high-risk neuroblastoma who have completed induction chemotherapy (independent of this study). Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #5; however it is strongly recommended to begin consolidation within 4-6 weeks after the start of Induction Cycle #5.

Phase 2 STIR Trial: Haploidentical Transplant and Donor Natural Killer Cells for Solid Tumors (STIR)

Posted on

The investigators hypothesize that this Phase 2 cellular and adoptive immunotherapy study using human leukocyte antigen (HLA)-haploidentical hematopoietic cell transplantation (HCT) followed by an early, post-transplant infusion of donor natural killer (NK) cells on Day +7 will not only be well-tolerated in this heavily-treated population (safety), but will also provide a mechanism to treat high-risk solid tumors, leading to improved disease control rate (efficacy). Disease control rate is defined as the combination of complete (CR) and partial (PR) response and stable disease (SD). The investigators further propose that this infusion of donor NK cells will influence the development of particular NK and T cell subtypes which will provide immediate/long-term tumor surveillance, infectious monitoring, and durable engraftment. Patients with high-risk solid tumors (Ewings Sarcoma, Neuroblastoma and Rhabdomyosarcoma) who have either measurable or unmeasurable disease and have met eligibility will be enrolled on this trial for a goal enrollment of 20 patients over 4 years.

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Posted on

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Posted on

This is a standard of care treatment guideline for allogeneic hematopoietic stem cell transplant (HSCT) in patients with primary immune deficiencies.
NOTE: many likely fall in the eligibility category of “CID” (see up to date “Combined immunodeficiencies, Table 1)

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Posted on

This is a standard of care treatment guideline for allogeneic hematopoietic stem cell transplant (HSCT) in patients with primary immune deficiencies.