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Blood and Bone Marrow Transplant Clinical Trials

A Trial Comparing Unrelated Donor BMT with IST for Pediatric and Young Adult Patients with Severe Aplastic Anemia (TransIT, BMT CTN 2202) (TransIT)

Conditions: Aplastic Anemia
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT05600426.


Phase I/​II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)

Conditions: Leukemia
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT05322850.


HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide (ACCESS)

Conditions: Leukemia
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT04904588.


CIBMTR Research Database

Interventions: Cancers
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT01166009.


Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Abo

Conditions: Chronic Graft versus Host disease
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT05567406.


Dermatology Clinical Trials

A Study to Evaluate the Pharmacokinetics, Safety, and Effectiveness of Certolizumab Pegol in Children With Moderate to Severe Chronic Plaque Psoriasis (CIMcare)

Conditions: Psoriasis
Principal Investigator: Ana Duarte
Learn more at https://clinicaltrials.gov/study/NCT04123795.


Gastroenterology Clinical Trials

Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis

Conditions: Ulcerative Colitis
Eligibility:
 
Principal Investigator: Lina Maria Felipez
Learn more at https://clinicaltrials.gov/study/NCT04624230.


ABTECT-2 - ABX464 Treatment Evaluation for Ulcerative Colitis Therapy -2

Conditions: Ulcerative Colitis
Principal Investigator: Lina Maria Felipez
Learn more at https://clinicaltrials.gov/study/NCT05507216.


Hematology and Oncology Clinical Trials

Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia

Conditions: B-cell Acute Lymphoblastic Leukemia (B-ALL)
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT02981628.


Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT03959085.


Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04203316.


The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lympho

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04726241.


A Study of the Drugs Selumetinib Versus Carboplatin/​Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

Conditions: Glioma, Neurofibromatosis
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT03871257.


A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma

Conditions: Glioma, Astrocytoma
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT04166409.


A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT

Conditions: Non-germinomatous germ cell tumors (NGGCT)
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT04684368.


Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

Conditions: Osteosarcoma, Metastatic Malignant Neoplasm in the Lung
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT05235165.


Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors

Conditions: Germ Cell Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT03067181.


Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)

Conditions: Germ Cell Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT02582697.


The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALL

Conditions: B-cell Acute Lymphoblastic Leukemia (B-ALL)
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT03509961.


Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)

Conditions: Neuroblastoma
Principal Investigator: Guillermo De Angulo
Learn more at https://clinicaltrials.gov/study/NCT02559778.


A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT

Conditions: Wilms Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04322318.


Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma

Conditions: Sarcoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05304585.


Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Conditions: Cancers
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05857969.


Study to Learn More About the Safety and Effectiveness of the Drug VITRAKVI During Routine Use in Patients With TRK Fusion Cancer Which is Locally Advanced or Spread From the Place Where it Started to

Conditions: Advanced or Metastatic Solid Tumor
Principal Investigator: Ossama Maher
Learn more at https://clinicaltrials.gov/study/NCT04142437.


Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma

Conditions: Acute Lymphoblastic Leukemia (ALL)
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05602194.


A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab

Conditions: Hodgkin's Lymphoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05675410.


A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma

Conditions: Basal Ganglia Germinoma
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT06368817.


A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy

Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT06413706.


Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

Conditions: Neuroblastoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05489887.


A Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05761171.


DFMO as Maintenance Therapy for Molecular High/​Very High Risk and Relapsed Medulloblastoma

Conditions: Medulloblastoma
Principal Investigator: Ossama Maher
Learn more at https://clinicaltrials.gov/study/NCT04696029.


Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma

Conditions: Ganglioneruoblastoma, Neuroblastoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT06172296.


Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome

Conditions: Leukemia, Myelodysplastic Syndrome
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT05457556.


Nephrology Clinical Trials

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)

Conditions: Proteinuric Glomerular Diseases
Principal Investigator: Ana Paredes
Learn more at https://www.clinicaltrials.gov/study/NCT05003986.


Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)

Conditions: Complement 3 Glomerulopathy
Principal Investigator: Ana Paredes
Learn more at https://clinicaltrials.gov/study/NCT04817618.


Neurology Clinical Trials

Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy

Conditions: Cerebral Palsy, Dyskinesia
Principal Investigator: Migvis Monduy
Learn more at https://clinicaltrials.gov/study/NCT05206513.


Early Access Program With Arimoclomol in US Patients With NPC

Conditions: Niemann-Pick
Principal Investigator: Paula Schleifer
Learn more at https://clinicaltrials.gov/study/NCT04316637.


Clinical Needs of Individuals with a Chromosome 15 Condition

Conditions: Angelman Syndrome
Principal Investigator: Paula Schleifer


TSC Biosample Repository and Natural History Database

Conditions: Tuberous Sclerosis, Lymphangioleiomyomatosis
Principal Investigator: Paula Schleifer
Learn more at https://clinicaltrials.gov/study/NCT05676099.


A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Lacosamide in Neonates With Repeated Electroencephalographic Neonatal Seizures (LENS)

Conditions: Seizures
Principal Investigator: Anuj Jayakar
Learn more at https://clinicaltrials.gov/study/NCT04519645.


A Study to Test the Long-term Safety and Tolerability of Brivaracetam in Study Participants With Childhood Absence Epilepsy or Juvenile Absence Epilepsy

Conditions: Epilepsy
Principal Investigator: Anuj Jayakar
Learn more at https://clinicaltrials.gov/study/NCT06315322.


Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG

Conditions: Brain Tumor
Principal Investigator: Toba Niazi
Learn more at https://clinicaltrials.gov/study/NCT05630209.


A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex (EpiCom)

Conditions: Tuberous Sclerosis
Principal Investigator: Paula Schleifer
Learn more at https://clinicaltrials.gov/study/NCT05864846.


Efficacy and Safety of Erenumab in Pediatric Participants With Episodic Migraine (OASIS(EM))

Conditions: Migraines
Principal Investigator: Wilson Heredia Nunez
Learn more at https://clinicaltrials.gov/study/NCT03836040.


Randomized Study in Children and Adolescents With Migraine: Acute Treatment

Principal Investigator: Wilson Heredia-Nunez
Learn more at https://clinicaltrials.gov/study/NCT04649242.


Focal Cerebral Arteriopathy Steroid Trial (FOCAS)

Conditions: Stroke, Arteriopathy, Arterial Ischemic Stroke
Principal Investigator: Anuj Jayakar
Learn more at https://clinicaltrials.gov/study/NCT06040255.


A Study to Evaluate the Efficacy, Safety, and Tolerability of Brivaracetam as Monotherapy in Patients 2 to 25 Years of Age With Childhood Absence Epilepsy or Juvenile Absence Epilepsy (EXPAND)

Conditions: Absence Epilepsy
Principal Investigator: Anuj Jayakar
Learn more at https://clinicaltrials.gov/study/NCT04666610.


Orthopedics Clinical Trials

Developmental Dysplasia of the Hip: Observation Vs. Bracing

Conditions: Hip Dysplasia
Principal Investigator: Verena Schreiber
Learn more at https://clinicaltrials.gov/study/NCT05869851.


Cast or Operation for Medial Epicondyle Fracture Treatment in Children (COMET)

Conditions: Fracture Elbow
Principal Investigator: Monica Payares-Lizano
Learn more at https://clinicaltrials.gov/study/NCT05131672.


Distal Radius Interventions for Fracture Treatment (DRIFT)

Conditions: Fracture Distal Radius
Principal Investigator: Monica Payares-Lizano
Learn more at https://clinicaltrials.gov/study/NCT05131685.


You can also search for current studies Nicklaus Children's Hospital is participating in ClinicalTrials.gov