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Children's Hospital Los Angeles | Neurology Research

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Active trials

17 of 130 total trials

A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lympho...

Active, not recruiting
Leukemia
Drug: Methotrexate
Drug: Thioguanine

Phase 1 will evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or me...

Enrolling
Locally Advanced Solid Tumors
Primary CNS Tumors
Drug: Oral repotrectinib (TPX-0005)

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive...

Enrolling
Cardiomyopathy, Hypertrophic
Drug: Placebo
Drug: Mavacamten

The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to...

Active, not recruiting
Crohn Disease
Drug: Ozanimod

This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at...

Active, not recruiting
Severe Hemophilia A
Drug: Emicizumab

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Active, not recruiting
X-Linked Retinitis Pigmentosa
Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose
Biological: Genetic: AAV5-hRKp.RPGR Low Dose

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Active, not recruiting
X-Linked Retinitis Pigmentosa
Biological: Genetic: AAV5-hRKp.RPGR

This NANT trial will determine the maximum tolerated dose (MTD) of autologous expanded natural killer (NK) cells when combined with standard dosing o...

Active, not recruiting
Neuroblastoma
Drug: Lenalidomide
Biological: NK Cells

Migraine is a common neurological disorder typically characterized by attacks of throbbing, moderate to severe headache, often associated with nausea...

Invitation-only
Migraine
Drug: Ubrogepant

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based...

Enrolling
Spinal Muscular Atrophy
Drug: Risdiplam

131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disea...

Active, not recruiting
Neuroblastoma
Radiation: 131I-MIBG
Drug: Vorinostat

Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS)...

Active, not recruiting
Cholestatic Liver Disease
Drug: Maralixibat

Lorlatinib is a novel inhibitor across ALK variants, including those resistant to crizotinib. In this first pediatric phase 1 trial of lorlatinib, th...

Active, not recruiting
Neuroblastoma
Drug: Lorlatinib
Drug: Filgrastim/pegfilgrastim
Status recently updated

This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.

Not yet enrolling
Enrolling
Neuroblastoma
Drug: Irinotecan
Drug: Temozolomide

This is a multicenter trial of the Optune device to examine the feasibility and to describe the device-related toxicity in children with supratentori...

Enrolling
Malignant Glioma
Ependymoma
Combination Product: Concurrent Optune and RT followed by Optune System alone
Device: Optune System (NovoTTF-200A System, Tumor Treating Fields, TTFields)

This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor...

Active, not recruiting
Ependymoma, Not Otherwise Specified (NOS)
Glioblastoma Multiforme
Biological: APX005M treatment for newly diagnosed DIPG patients
Biological: APX005M treatment for recurrent or refractory primary malignant CNS tumor patients

Migraine is a common neurological disorder typically characterized by attacks of throbbing, moderate to severe headache, often associated with nausea...

Enrolling
Migraine
Drug: Placebo-Matching Ubrogepant
Drug: Ubrogepant

Trial sponsors

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Children's Hospital Los Angeles logo
Pediatric Brain Tumor Consortium logo
AbbVie logo
Roche logo
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Janssen (J&J Innovative Medicine) logo

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