Clinical Trials | CHOP Research Institute

First Two Gene Therapies for Sickle Cell Disease Supported by CHOP Clinical Research

Published on

Jan 3, 2024

Two Gene Therapies Tested at CHOP Approved for Sickle Cell Disease

After decades of limited progress in treating sickle cell disease, we have reached an historical moment with two new gene therapies.

Sickle Cell Disease, Concussion Prevention, Firearm Injury, Epilepsy Disorders

Published on

Dec 22, 2023

in Cornerstone Blog

Our research news roundup highlights newly approved gene therapies, insights into common genetic epilepsy disorders, and head injury prevention for young athletes.

MAS825 Trial for NLRC4-GOF

The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF).

VOCAL Trial

This is a study to measure the effectiveness of the medication voclosporin compared to placebo in adolescent patients with active lupus nephritis.

Pimavanserin for the Treatment of Irritability Associated With ASD

The purpose of this study is to test an investigational medication in children and adolescents aged 5-17 years old with irritability associated with autism spectrum disorder (ASD).

Stephan Kadauke, MD, PhD

Dr. Kadauke is the associate director of the Cell and Gene Therapy Laboratory at CHOP and the medical director of the Cell and Gene Therapy Informatics team. His research focuses on developing novel ways to manufacture cell therapies with the goal of accelerating and broadening patient access to these life-saving therapies.

E-mail:

kadaukes [at] chop.edu

Novel Therapeutics for Bleeding Disorders

Combining our proven clinical expertise in hemophilia gene therapy and care of bleeding disorders with our research expertise to solidify Children’s Hospital of Philadelphia as the premier center for the therapeutic development and advanced clinical care of inherited bleeding disorders.

DTX401 Gene Therapy in Glycogen Storage Disease (GSD) Type Ia

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)

Knowing What Matters Most to Patients is Key to Pediatric Kidney Stone Research

Published on

Mar 14, 2022

in Cornerstone Blog

Pediatric KIDney Stone Care Improvement Network is all about collaborative, partnership-based, patient-centered kidney stone research.

Shifting Perspectives

This Shifting Perspectives study explores whether we can improve treatment outcomes in teens with anorexia nervosa by adding Adolescent Focused Cognitive Remediation Therapy (CRT) to Family Based Treatment (FBT). CRT focuses on improving cognitive and behavioral flexibility in adolescents.