Pediatric Clinical Trials: Open, Ongoing and Recruiting Studies

To inquire about current studies please contact us at:

NCRI.recruitment@nicklaushealth.org

Cardiology Clinical Trials

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). (CEDAR-HCM)

Study ID:
CAR25047 (20241212)

Principal Investigator:
Ronald J Kanter, MD

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Percutaneous Intervention Versus Observational Trial of Arterial Ductus in Low Weight Infants (PIVOTAL)

Study ID:
CAR25008 (PIVOTAL)

Principal Investigator:
Daniel Duarte Caceres, MD

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PREEMIE: Study for Treatment of PDA in Premature Infants (PREEMIE)

Study ID:
CAR25032 (PDA-P3-23-01, PREEMIE)

Principal Investigator:
Shyam K Sathanandam, MD

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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)

Study ID:
DER99037 (PS0007)

Principal Investigator:
Ana Margarita Duarte, MD

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A Study to Evaluate the Safety and Efficacy of Ruxolitinib Cream in Pediatric Participants With Nonsegmental Vitiligo

Study ID:
DER24083 (INCB 18424-312)

Principal Investigator:
Ana Margarita Duarte, MD

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Endocrinology Clinical Trials

A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature

Study ID:
END24024 (BMN 111-210)

Principal Investigator:
Adriana Carrillo Iregui, MD

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Universal Screening for Type 1 Diabetes in the Primary Clinical Setting

Study ID:
END25055 (RDC92)

Principal Investigator:
Adriana Carrillo Iregui, MD

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Gastroenterology Clinical Trials

A prospective study evaluating serial intestinal ultrasound (IUS) use in pediatric Inflammatory Bowel Disease (IBD)

Study ID:
GAS24086

Principal Investigator:
Lina Maria Felipez, MD

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A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis (MIGHTY)

Study ID:
GAS24065 (M19-751)

Principal Investigator:
Lina Maria Felipez, MD

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

Study ID:
GAS99042 (OVATION)

Principal Investigator:
Lina Maria Felipez, MD

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Microbiome and Genetic Profile [MGP] of Very Early-Onset Pediatric Inflammatory Bowel Disease and Pediatric Inflammatory Bowel Disease

Study ID:
GAS23122

Principal Investigator:
Lina Maria Felipez, MD

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Hematology/Oncology Clinical Trials

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

Study ID:
ONC99099 (ACNS2021)

Principal Investigator:
Ziad A Khatib, MD

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

Study ID:
ONC99274 (AREN1921)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC25006 (ACNS2321)

Principal Investigator:
Ziad A Khatib, MD

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

Study ID:
ONC99074 (ACNS1833)

Principal Investigator:
Ziad A Khatib, MD

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A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss

Study ID:
ONC99270 (ACNS2031)

Principal Investigator:
Ziad A Khatib, MD

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A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations

Study ID:
ONC99096 (AAML1831)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99271 (AHOD2131)

Principal Investigator:
Maggie Eidson Fader, MD

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A Study to Test the Addition of the Drug Cabozantinib to Chemotherapy in Patients With Newly Diagnosed Osteosarcoma

Study ID:
ONC99273 (AOST2032)

Principal Investigator:
Maggie Eidson Fader, MD

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A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)

Study ID:
ONC24032 (ARAR2221)

Principal Investigator:
Maggie Eidson Fader, MD

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A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

Study ID:
ONC23006 (ANHL2121)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99078 (AGCT1532)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99077 (AGCT1531)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99276 (ARST2032)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC24008 (BCC016)

Principal Investigator:
Ossama M Maher, MD

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

Study ID:
ONC24030 (ANBL2131)

Principal Investigator:
Maggie Eidson Fader, MD

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Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Study ID:
ONC99139 (Ex Vivo 3)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99062 (AALL1732)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99060 (AALL1621)

Principal Investigator:
Maggie Eidson Fader, MD

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Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

Study ID:
ONC23118 (BCC018)

Principal Investigator:
Maggie Eidson Fader, MD

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Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)

Study ID:
ONC99113 (NMTRC012)

Principal Investigator:
Guillermo R De Angulo, MD

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Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors

Study ID:
ONC24057 (BCC021)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC23150 (ACCL1931)

Principal Investigator:
Maggie Eidson Fader, MD

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

Study ID:
ONC99116 (PTCTC ONC1701)

Principal Investigator:
Jorge Ricardo Galvez Silva, MD

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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 & Lymphoma Society and Children's Oncology Group Study

Study ID:
ONC99101 (APAL2020SC)

Principal Investigator:
Maggie Eidson Fader, MD

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Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

Study ID:
ONC99100 (AOST2031)

Principal Investigator:
Ziad A Khatib, MD

View Study Details

Nephrology Clinical Trials

A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB (ACTION3)

Study ID:
NEP24046 (DMX-200-301)

Principal Investigator:
Ana L Paredes, MD

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Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (APPARENT)

Study ID:
NEP23144 (CLNP023B12302)

Principal Investigator:
Ana L Paredes, MD

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

Study ID:
NEP23121 (CLNP023B12301)

Principal Investigator:
Ana L Paredes, MD

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

Study ID:
NEP99184 (RTRX-RE021-201)

Principal Investigator:
Ana L Paredes, MD

View Study Details

Neurology Clinical Trials

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

Study ID:
NEU24048 (STK-001-DS-301)

Principal Investigator:
Matt S Lallas, MD

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

Study ID:
NEU99205 (BT005)

Principal Investigator:
John Ragheb, MD

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A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

Study ID:
NEU24088 (LP352-302)

Principal Investigator:
Matt S Lallas, MD

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A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)

Study ID:
NEU24061 (LP352-301)

Principal Investigator:
Matt S Lallas, MD

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

Study ID:
NEU24007 (EP0224)

Principal Investigator:
Anuj Jayakar, MD

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

Study ID:
NEU23134 (BT016)

Principal Investigator:
Toba N Niazi, MD

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Focal Cerebral Arteriopathy Steroid Trial (FOCAS)

Study ID:
NEU23170 (FOCAS)

Principal Investigator:
Anuj Jayakar, MD

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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Study ID:
NEU24071 (ION582-CS2)

Principal Investigator:
Paula Padua Schleifer, MD

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Orthopedics Clinical Trials

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

Study ID:
ORT23136 (2022-4916_COMET)

Principal Investigator:
Monica M Payares-Lizano, MD

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Developmental Dysplasia of the Hip: Observation Vs. Bracing

Study ID:
ORT23125 (Brace v. Observational)

Principal Investigator:
Kevin S Horowitz, MD

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Distal Radius Interventions for Fracture Treatment (DRIFT)

Study ID:
ORT23139 (2021-4883_DRIFT)

Principal Investigator:
Monica M Payares-Lizano, MD

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Primary Care Centers Clinical Trials

Food for Salud Medically Tailored Grocery Intervention

Study ID:
PCC25017 (Food for Salud)

Principal Investigator:
Joanna E Perdomo, MD

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Psychology Clinical Trials

The Pediatric Precision Sleep Network

Study ID:
PSC24011 (PPSN)

Principal Investigator:
Sara Rivero-Conil, PsyD

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Wise Girls Workshop Study

Study ID:
PSC24069 (Wise Girls)

Principal Investigator:
Marina G Villani Capo, PsyD

View Study Details

Pediatric Clinical Trials: Open, Ongoing and Recruiting Studies

To inquire about current studies please contact us at:

Cardiology Clinical Trials

Dermatology Clinical Trials

Endocrinology Clinical Trials

Gastroenterology Clinical Trials

Hematology/Oncology Clinical Trials

Nephrology Clinical Trials

Neurology Clinical Trials

Orthopedics Clinical Trials

Primary Care Centers Clinical Trials

Psychology Clinical Trials

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