Search for Open Studies

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Bone and Blood Marrow Transplant Clinical Trials

A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) with Acute Leukemia or Myelodysplastic Syndrome (MDS)

Conditions: Leukemia
Principal Investigator: Jorge Galvez-Silva
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05457556.


Phase I/II Trial: Engineered Donor Graft (Orca-Q) for Pediatric Hematopoietic Cell Transplant (HCT) Recipients with Hematologic Malignancies (HM)

Conditions: Hematologic Malignancies
Principal Investigator: Jorge Galvez-Silva
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05322850.


A Phase II Pilot Trial to Estimate Survival After a Non-Total Body Irradiation (TBI) Based Conditioning Regimen in Patients Diagnosed With B-Acute Lymphoblastic Leukemia (ALL) Who Are Pre-Allogeneic Hematopoietic Cell Transplantation (HCT) Next-Generation-Sequence (NGS) Minimal Residual Disease (MRD) Negative

Conditions: Lymphoblastic Leukemia
Principal Investigator: Jorge Galvez-Silva
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03509961.


Dermatology Clinical Trials

Multicenter, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled (12-17 Years) Including A Single Open-Label Arm (6-11 Years) Study To Evaluate The Efficacy, Safety, And Pharmacokinetics Of Certolizumab Pegol (Czp) In Pediatric Study Participants With Moderate To Severe Chronic Plaque Psoriasis (Pso)

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


Gastroenterology Clinical Trials

A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase III Study To Evaluate The Efficacy And Safety Of Abx464 Once Daily For Induction Treatment In Subjects With Moderately To Severely Active Ulcerative Colitis

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


Open-Label Induction And Maintenance Study Of Oral Cp-690,550 (Tofacitinib) In Children With Moderately To Severely Active Ulcerative Colitis

Conditions: Ulcerative Colitis
Principal Investigator: Lina Maria Felipez
CRA: Moya Chang


Hematology and Oncology Clinical Trials

International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03007147.


A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03817398.


A Phase 3 Randomized Trial for Patients with De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 with GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients with FLT3 Mutations

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04293562.


An Open-Label Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients with Relapsed/Refractory Acute Myeloid Leukemia R/R-AML with an Isocitrate Dehydrogenase-2 IDH2 Mutation

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04203316.


Pediatric Acute Leukemia (PedAL) Screening Trial – Developing New Therapies for Relapsed Leukemias

Conditions: Leukemia
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04726241.


A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients

Conditions: Medulloblastoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT02724579.


A Phase 2 Study of Dabrafenib (NSC# 763760) with Trametinib (NSC# 763093) after Local Irradiation in Newly-Diagnosed BRAFV600-Mutant High-Grade Glioma (HGG)

Conditions: Glioma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Conditions: Glioma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1) 

Conditions: Glioma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine (IND# 149832) for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors

Conditions: Central Nervous System Tumors
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor 

Conditions: Central Nervous System Tumors
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04684368.


A Phase 3 Study of Sodium Thiosulfate for Reduction of Cisplatin-Induced Ototoxicity in Children with Average-Risk Medulloblastoma and Reduced Therapy in Children with Medulloblastoma with Low-Risk Features

Conditions: Medulloblastoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05382338.


A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients with Osteosarcoma

Conditions: Sarcoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05235165.


A Feasibility and Randomized Phase 2/3 Study of the VEFGR2/MET Inhibitor Cabozantinib in Combination with Cytotoxic Chemotherapy for Newly Diagnosed Osteosarcoma

Conditions: Sarcoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05691478.


A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy with Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) plus VINO-CPO Maintenance in Patients with High Risk Rhabdomyosarcoma (HR-RMS)

Conditions: Sarcoma
Principal Investigator: Maggie Fader
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04994132.


A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma

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


A Phase 3 Study of Active Surveillance for Low risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors 

Conditions: Germ Cell Tumor
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03067181.


Phase 3 Accelerated BEP Trial: A Randomized Phase 3 Trial of Accelerated Versus Standard BEP Chemotherapy for Patients With Intermediate and Poor-Risk Metastatic Germ Cell Tumors

Conditions: Germ Cell Tumor
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL)

Conditions: Neuroblastoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03126916.


A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma

Conditions: Neuroblastoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03794349.


Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT) – Phase 2/3

Conditions: Hepatic Malignancy
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy with Immuno-oncology Therapy for Children and Adults with Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma

Conditions: Hodgkin's Lymphoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05675410.


Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Conditions: Wilms Tumor
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT04322318.


Prospective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults with Cancer 

Conditions: Childhood Cancer
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05228275.


Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis

Conditions: Langerhans Cell Histiocytosis
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05828069.


A Single Arm, Open-Label, Phase 1b Trial of Epcoritamab in Pediatric Patients with Relapsed/ Refractory Aggressive Mature B-cell Neoplasms

Conditions: B-Cell Neioplasms
Principal Investigator: Maggie Fader
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05206357.


Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors: Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Either Single Cycle (Low-Risk Patients) Or Randomization (High Risk Patients) To Either Single-Cycle Or To Three Tandem Cycles Of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue

Conditions: Medulloblastoma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier


A Parallel-group, Two-staged, Phase 2/3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of REC-2282 in Participants with Progressive NF2 Mutated Meningiomas

Conditions: Meningioma
Principal Investigator: Ziad Khatib
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05130866.


A Study Using Molecular Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma

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


A Randomized, Open-Label, Active Controlled, Safety And Descriptive Efficacy Study In Pediatric Subjects Requiring Anticoagulation For The Treatment Of A Venous Thromboembolic Event

Conditions: Venous Thromboembolic Event
Principal Investigator: Maggie Fader
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT02464969.


Expanded Access to OKN-007 for Patients With Diffuse Midline Glioma, H3 K27-altered

Conditions: Diffuse Midline Glioma
Principal Investigator: Ossama Maher
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT05518838.


Nephrology Clinical Trials

A Phase 2, Open-Label, Single-Arm, Cohort Study To Evaluate The Safety, Efficacy, And Pharmacokinetics Of Sparsentan Treatment In Pediatric Subjects With Selected Proteinuric Glomerular Diseases

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


Neurology Clinical Trials

A Phase 3, Randomized, Double-blind, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy

Conditions: Epilepsy
Principal Investigator: Matt Lallas
CRA: Marinellie Vega
Learn more at https://clinicaltrials.gov/study/NCT05323734.


A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy

Conditions: Cerebral Palsy
Principal Investigator: Migvis Monduy
CRA: Marinellie Vega
Learn more at https://clinicaltrials.gov/study/NCT05206513.


Early Access Program with Arimoclomol for the Treatment of Niemann-Pick disease Type C in the US

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


Intrathecal 2-Hydroxypropyl-B-cyclodextin for Neurological Decline in Patients with Niemann-Pick Disease Type C1

Conditions: Niemann-Pick
Principal Investigator: Paula Schleifer
CRA: Marinellie Vega


Clinical Needs of Individuals with a Chromosome 15 Condition

Conditions: Angelman Syndrome
Principal Investigator: Paula Schleifer
CRA: Marinellie Vega


TS Alliance Tuberous Sclerosis Complex (TSC) Biosample Repository and Natural History Database

Conditions: Tuberous Sclerosis Complex
Principal Investigator: Paula Schleifer
CRA: Marinellie Vega


A Multicenter, Open-Labe, Randomized, Active Comparator Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Lacosamide in Neonates with Repeated Electroencephalographic Neonatal Seizures

Conditions: Epilepsy
Principal Investigator: Anuj Jayakar
CRA: Marinellie Vega


A double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of intravenous Ganaxolone in status epilepticus Ganaxolone

Conditions: Epilepsy
Principal Investigator: Anuj Jayakar
CRA: Marinellie Vega
Learn more at https://clinicaltrials.gov/study/NCT04391569.


A Randomized, Dose-Finding and Confirmatory, Double-Blind, Placebo-Controlled, Parallel-Group Multicenter Study with a 2-Stage Adaptive Design and Randomized Withdrawal 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

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


Predicting Seizure Responsiveness to Neuromodulation Using Connectomic Profiling (Vagus Nerve Stimulation [VNS] Study)

Conditions: Epilepsy
Principal Investigator: Shelly Wang
CRA: Marinellie Vega


Neurosurgery Clinical Trials

A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects

The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects.

Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible.

Conditions: Intracranial Tumors
Interventions: Device: ExAblate 4000 System
Eligibility: 8 Years to 22 Years
Principal Investigator: John Ragheb
CRA: Marinellie Vega
Learn more at https://clinicaltrials.gov/study/NCT03028246.


Psychology Clinical Trials

Sleep-dependent Negative Overgeneralization in Peri-pubertal Anxiety

Conditions: Anxiety
Principal Investigator: Dana McMakin
CRA: Michelin Janvier
Learn more at https://clinicaltrials.gov/study/NCT03643848.


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