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Found 157 clinical trials
Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency

development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

pah gene
deficiency
  • 0 views
  • 11 Jul, 2022
  • 3 locations
Gene Therapy Clinical Study in Adult PKU (pheNIX)

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants

pah gene
deficiency
gene therapy
  • 178 views
  • 09 Feb, 2022
  • 12 locations
Liver Cell Transplant for Phenylketonuria

Human phenylketonuria (PKU) results from phenylalanine hydroxylase (PAH) deficiency, and represents one of the most common and extensively studied single-gene Mendelian disorders in humans

sapropterin
kuvan
deficiency
psychological assessment
phenylalanine
  • 51 views
  • 22 Mar, 2022
  • 1 location
Durham Healthy Breathing in Healthy Homes Project (DHBHH)

To determine if Home-based Environmental Interventions (HEI) that improve home air-quality problems can improve asthma outcomes. Participants will benefit through home-based environmental interventions that improve home air-quality problems and improve asthma outcomes. All participants will receive a Breath Easy at Home Kit, which could help with reducing environmental exposures. The …

  • 0 views
  • 11 Jul, 2022
  • 1 location
PET/MR Pre- and Post Radiotherapy for Cardiopulmonary Dysfunction Evaluation

Radiotherapy (RT) is a well-known and established therapy or adjuvant therapy for the treatment of thoracic cancer It uses a high energy radiation from x-rays, gamma rays and other charged particles that assist in damaging the cancer DNA. PET/MR as imaging biomarkers for cardiopulmonary dysfunction with a focus on Pulmonary …

cancer
  • 0 views
  • 08 Oct, 2021
  • 1 location
A Long-term Follow-up Study of Sotatercept for PAH Treatment (SOTERIA)

This study is being conducted to assess the long-term safety, tolerability, and efficacy of sotatercept in PAH.

  • 0 views
  • 09 Aug, 2022
  • 114 locations
A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background

calcium
endothelin receptor antagonist
hypertension
right heart catheterization
endothelin
  • 6468 views
  • 05 Jul, 2022
  • 229 locations
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension (SALTO)

The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in

heart disease
pulmonary arterial hypertension
endothelin receptor antagonist
endothelin
primary pulmonary hypertension
  • 247 views
  • 25 Jul, 2022
  • 88 locations
A Study in Participants With Sarcoidosis-associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag (SPHINX)

Oral selexipag is commercially available in several countries for the treatment of a particular group of pulmonary hypertension (PH) called pulmonary arterial hypertension (PAH). The aim of the

  • 0 views
  • 29 Jul, 2022
  • 33 locations
A Study of Ralinepag to Evaluate Effects on Exercise Capacity by CPET in Subjects With WHO Group 1 PH (CAPACITY)

Study ROR-PH-302, ADVANCE CAPACITY, is designed to evaluate the effects of ralinepag therapy on exercise capacity as assessed by change in peak oxygen consumption (VO2) derived from cardiopulmonary exercise testing (CPET) after 28 weeks of treatment

ralinepag
exercise stress test
endothelin receptor antagonist
cardiopulmonary exercise testing
diuretics
  • 4 views
  • 21 Jul, 2022
  • 36 locations