Clinical Trials | CHOP Research Institute
 

Clinical Trials

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
Stephan Kadauke

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

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)

UX053 in GSD III

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a clinical drug trial that involves giving you a study drug called UX033.

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

Published on
Mar 14, 2022
in Cornerstone Blog
Patient-centered Kidney Stone Research
Pediatric KIDney Stone Care Improvement Network is all about collaborative, partnership-based, patient-centered kidney stone research.

Shifting Perspectives

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.

In the News: MRSA, EoE Clinical Trial, Rare Disease, Exploring the Hypothalamus

Published on
Nov 26, 2021
in Cornerstone Blog
MRSA, EoE Clinical Trial, Rare Disease, Exploring the Hypothalamus
Learn about novel findings and new funding from our Children’s Hospital of Philadelphia researchers.

HM15136 for the Treatment of Congenital Hyperinsulinism Research Study

The purpose of this study is to look at the safety and tolerability (how well you will react) of the study drug (HM15136) and to determine if it is effective for the treatment of Congenital Hyperinsulinism (HI).