Clinical Trials | CHOP Research Institute
 

Clinical Trials

Research Family Partners

Research Family Partners

Research Family Partners assists study teams with community engagement and incorporating the patient and family voice in research.

Cornerstone Blog

Clinical Research Coordinator Discovers Passion for Painting

Published on
Jul 12, 2024
Clinical Research Coordinator Discovers Passion for Painting
Subecha “Suvy” Dahal shares how she discovered a natural inclination for creating whimsical oil paintings.
Cornerstone Blog

CHOP-Penn Team Tests First In-Body CRISPR Therapy to Treat Blindness

Published on
Jun 20, 2024
Jacob Peckham
Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.
Cornerstone Blog

CHOP-Penn Autism Experts Share Vision for Accelerated Discovery

Published on
Jun 3, 2024
Joseph W. St. Geme, MD, chair, Department of Pediatrics at CHOP; Lisa Guay-Woodford, MD, senior advisor for Clinical and Translational Research Initiatives at CHOP; and Benjamin Prosser, PhD, director, Center for Epilepsy and Neurodevelopmental Disorders
The CHOP-Penn Autism Research Symposium explored opportunities for breakthroughs in the understanding and treatment of autism spectrum disorder.
Cornerstone Blog

A 30-Year Journey: How CHOP Researchers Designed the Gene Therapy Playbook

Published on
May 29, 2024
How CHOP Researchers Designed the Gene Therapy Playbook
Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.
Cornerstone Blog

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.
Cornerstone Blog

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

Published on
Dec 22, 2023
In the News
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

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

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).