Pediatric Clinical Trials: Open, Ongoing and Recruiting Studies

Blood and Bone Marrow Transplant Clinical Trials

• Phase I/​II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)
  Study ID: BMT23108 (ORCA-Q)
  PI: Jorge Galvez-Silva. View study details

• A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (TransIT)
  Study ID: BMT23109 (TransIT)
  PI: Jorge Galvez-Silva. View study details

• 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)
  PI: Jorge Galvez-Silva. View study details

• HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide (ACCESS)
  Study ID: BMT23119 (CIBMTR/NMDP_ACCESS)
  PI: Jorge Galvez-Silva. View study details

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)
  PI: Ana Margarita M Duarte. View study details

Gastroenterology Clinical Trials

• Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis
  Study ID: GAS99042 (OVATION)
  PI: Lina Felipez. View study details

• ABTECT-2 - ABX464 Treatment Evaluation for Ulcerative Colitis Therapy -2
  Study ID: GAS23135 (ABX464-106)
  PI: Lina Maria Felipez. View study details

Hematology and Oncology Clinical Trials

• A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
  Study ID: ONC99274 (AREN1921)
  PI: Maggie Fader. View study details

• 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 Other Places in the Body (ON-TRK)
  Study ID: ONC99126 (ON-TRK)
  PI: Ossama Maher. View study details

• DFMO as Maintenance Therapy for Molecular High/​Very High Risk and Relapsed Medulloblastoma
  Study ID: ONC24008 (BCC016)
  PI: Ossama Maher. View study details

• Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)
  Study ID: ONC99078 (AGCT1532)
  PI: Maggie Fader. View study details

• PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma
  Study ID: ONC23174 (CONNECT1906)
  PI: Ziad Khatib. View study details

• A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
  Study ID: ONC23006 (ANHL2121)
  PI: Maggie Fader. View study details

• MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
  Study ID: ONC23127 (CONNECT2108)
  View study details

• ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
  Study ID: ONC23173 (CONNECT1905)
  PI: Ziad Khatib. View study details

• 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)
  PI: Maggie Fader. View study details

• Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)
  Study ID: ONC99113 (NMTRC012)
  PI: Guillermo De Angulo. View study details

• A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy
  Study ID: ONC23162 (I3Y-MC-JPEH)
  PI: Ziad Khatib. View study details

• Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma
  Study ID: ONC23150 (ACCL1931)
  PI: Maggie Fader. View study details

• A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab
  Study ID: ONC99271 (AHOD2131)
  PI: Maggie Fader. View study details

• Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
  Study ID: ONC99276 (ARST2032)
  PI: Maggie Fader. View study details

• A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma
  Study ID: ONC25006 (ACNS2321)
  PI: Ziad Khatib. View study details

• Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
  Study ID: ONC23118 (BCC018)
  PI: Maggie Fader. View study details

• Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
  Study ID: ONC24030 (ANBL2131)
  PI: Maggie Fader. View study details

• Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
  Study ID: ONC99077 (AGCT1531)
  PI: Maggie Fader. View study details

• 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)
  PI: Maggie Fader. View study details

• A Study of the Drugs Selumetinib Versus Carboplatin/​Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
  Study ID: ONC99073 (ACNS1831)
  PI: Ziad Khatib. View study details

• A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
  Study ID: ONC99074 (ACNS1833)
  PI: Ziad Khatib. View study details

• Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
  Study ID: ONC99100 (AOST2031)
  PI: Ziad Khatib. View study details

• A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
  Study ID: ONC99099 (ACNS2021)
  PI: Ziad Khatib. View study details

• A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)
  Study ID: ONC24032 (ARAR2221)
  PI: Maggie Fader. View study details

• A Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia
  Study ID: ONC24004 (AALL2121)
  PI: Maggie Fader. View study details

• Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia
  Study ID: ONC99060 (AALL1621)
  PI: Maggie Fader. View study details

Nephrology Clinical Trials

• Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)
  Study ID: NEP99184 (RTRX-RE021-201)
  PI: Ana Paredes. View study details

• Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (APPARENT)
  Study ID: NEP23144 (CLNP023B12302)
  PI: Ana Paredes. View study details

• Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)
  Study ID: NEP23121 (CLNP023B12301)
  PI: Ana Paredes. View study details

Neurology Clinical Trials

• Randomized Study in Children and Adolescents With Migraine: Acute Treatment
  Study ID: NEU23013 (C4951002-BHV3000-311)
  PI: Wilson Heredia-Nunez. View study details

• Focal Cerebral Arteriopathy Steroid Trial (FOCAS)
  Study ID: NEU23170 (FOCAS)
  PI: Anuj Jayakar. View study details

• Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric Diffuse Intrinsic Pontine Gliomas (DIPG)
  Study ID: NEU23134 (BT016)
  PI: Toba Niazi. View study details

• 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)
  Study ID: NEU23003 (N01269)
  PI: Anuj Jayakar. View study details

• 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)
  Study ID: NEU23103 (JZP926-402)
  PI: Paula Schleifer. View study details

• Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects with AS (Aspire)
  Study ID: NEU24001 (GTX-102-CL301)
  PI: Paula Schleifer. View study details

• Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
  Study ID: NEU99201 (NBI-98854-DCP3018)
  PI: Migvis Monduy. View study details

• 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)
  PI: Anuj Jayakar. View study details

Orthopedics Clinical Trials

• Distal Radius Interventions for Fracture Treatment (DRIFT)
  Study ID: ORT23139 (2021-4883_DRIFT)
  PI: Monica Payares-Lizano. View study details

• Cast or Operation for Medial Epicondyle Fracture Treatment in Children (COMET)
  Study ID: ORT23136 (2022-4916_COMET)
  PI: Monica Payares-Lizano. View study details

• Developmental Dysplasia of the Hip: Observation Vs. Bracing
  Study ID: ORT23125 (Brace v. Observational)
  PI: Kevin Horowitz. View study details