Dr. Gerard Vockley, MD, PhD
Claim this profileUPMC Children's Hospital of Pittsburgh
Studies Phenylketonuria
Studies Lipid disorders
14 reported clinical trials
17 drugs studied
Affiliated Hospitals
Clinical Trials Gerard Vockley, MD, PhD is currently running
ELAPRASE + Prophylactic Therapy
for Hunter Syndrome
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR. Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously \[IV\], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle). Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR. Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).
Recruiting3 awards Phase 48 criteria
PTC923
for Phenylketonuria
This trial is testing the safety of a medication called PTC923 in people with phenylketonuria (PKU). PKU patients need to manage their blood phenylalanine levels carefully. PTC923 aims to help control these levels, making it easier for patients to manage their condition. PTC923 is a newer treatment for PKU, following earlier treatments like sapropterin and pegvaliase.
Recruiting2 awards Phase 34 criteria
More about Gerard Vockley, MD, PhD
Clinical Trial Related2 years of experience running clinical trials · Led 14 trials as a Principal Investigator · 5 Active Clinical TrialsTreatments Gerard Vockley, MD, PhD has experience with
- Placebo
- Neonate WGS Testing
- Triheptanoin
- Sodium Phenylbutyrate
- None
- HMI-103
Breakdown of trials Gerard Vockley, MD, PhD has run
Phenylketonuria
Lipid disorders
Medium-Chain Acyl-CoA Dehydrogenase Deficiency
Post-Implantation Syndrome
Genetic Disorders
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