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Ann & Robert H. Lurie Children's Hospital of Chicago | Gastroenterology, Hepatology and Nutrition Department

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Trials

57 of 322
Status: Active
Trial type: Interventional
Funder type: Industry

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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD...

Enrolling
Duchenne Muscular Dystrophy
Genetic: placebo
Genetic: delandistrogene moxeparvovec

The goal of this study is to evaluate the safety of the study drug PTC596 (Unesbulin) taken in combination with radiotherapy (RT) when given to pedia...

Active, not recruiting
High Grade Glioma
Diffuse Intrinsic Pontine Glioma
Drug: PTC596
Radiation: Radiotherapy

A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatm...

Enrolling
Mucopolysaccharidosis II
Drug: Idursulfase
Drug: JR-141

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with s...

Enrolling
Hemoglobinopathies
Sickle Cell Disease
Genetic: EDIT-301

The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene...

Enrolling
Canavan Disease
Biological: AAV9 BBP-812

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a ce...

Enrolling
Genetic Diseases, X-Linked
Muscular Diseases
Biological: Placebo
Biological: CAP-1002

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; I...

Active, not recruiting
Metachromatic Leukodystrophy (MLD)
Drug: SHP611

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying th...

Active, not recruiting
Spinal Muscular Atrophy
Drug: Nusinersen

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atr...

Active, not recruiting
Muscular Atrophy, Spinal
Drug: Nusinersen

This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (D...

Enrolling
Mucopolysaccharidosis II
Drug: tividenofusp alfa

A 2-part study to evaluate the safety, pharmacokinetics and efficacy of EDP-938 in children with RSV infection.

Enrolling
Respiratory Syncytial Virus (RSV)
Drug: EDP-938
Drug: Placebo

The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy...

Enrolling
Spinal Muscular Atrophy
Drug: Nusinersen

The ureter is the tube that carries urine from the kidneys to the bladder. It is difficult for surgeons to see the ureter during abdominal surgery. T...

Enrolling
Abdominolpelvic Surgery
Intraoperative Ureter Visualization
Drug: pudexacianinium chloride

This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acut...

Enrolling
Graft Versus Host Disease
Drug: Abatacept

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epi...

Enrolling
Tuberous Sclerosis Complex
Drug: Ganaxalone
Drug: Placebo

RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystro...

Enrolling
Duchenne Muscular Dystrophy
Genetic: RGX-202

Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously par...

Invitation-only
Dravet Syndrome
Drug: STK-001

Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Chan...

Active, not recruiting
Dravet Syndrome
Drug: STK-001 - Multiple Ascending Doses
Drug: STK-001 - Single Ascending Doses

This study is being conducted to test the safety and efficacy of ramucirumab in combination with other chemotherapy in the treatment of relapsed, rec...

Active, not recruiting
Desmoplastic Small Round Cell Tumor
Drug: Ramucirumab
Drug: Vinorelbine

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasm...

Enrolling
OTC Deficiency
Drug: Sodium Acetate
Genetic: DTX301

Trial conditions

Glioma (18 trials)
Syndrome (18 trials)
Cystic Fibrosis (15 trials)
Diffuse Intrinsic Pontin... (15 trials)
Fibrosis (15 trials)
Leukemia (13 trials)
Sickle Cell Anemia (13 trials)
Infections (12 trials)
Duchenne Muscular Dystro... (11 trials)
Muscular Dystrophies (11 trials)

And 90 more

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