1538 | Term | Boston Children's Hospital

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency As Open Label Extension

This is an open-label extension study for patients previously enrolled in the AB2 Bio Ltd. ongoing Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953). This OLE study will evaluate the long ...

A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide

The goal of this clinical trial was to compare participants with first relapse or refractory Ewing's sarcoma when treated with investigational product (Vigil) in addition to the standard treatment of ...

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous ...

A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.

Study to Evaluate Efficacy and Safety of PF-04965842 With or Without Topical Medications in Subjects Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis

B7451015 is a Phase 3 study to evaluate Abrocitinib with or without Topical Medications in patients aged 12 years and older who have moderate to severe atopic dermatitis and have completed a ...

Tacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score

The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive ...

AR101 Real-World Open-Label Extension Study

This study is enrolling participants by invitation only. This is an open-label, safety extension study for subjects who participated in the ARC007 study.

Low-Dose Atropine for Treatment of Myopia

Study Objectives The objectives for this randomized trial are: 1. To determine the efficacy of daily low-dose atropine (0.01%) for slowing myopia progression over a two-year treatment period in ...

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.

Longer-term Study of AR101 in Subjects Who Participated in a Prior AR101 Study (ARC008)

The purpose of this study is to assess AR101's safety, tolerability and efficacy over an extended dosing period.

Subscribe to PHASE3

Talk to Lesley