Clinical trials: Multiple Congenital Anomaly
Hemophilia B
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
- Trial name
- NCT02396342
- Testing
- AAV5-hFIX
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 10 people
- Sponsor
- CSL Behring
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Autosomal Dominant Polycystic Kidney, Hypertension
Effect of Beetroot Juice on Reducing Hypertension in Autosomal Dominant Polycystic Kidney Disease
People with autosomal dominant polycystic kidney disease (ADPKD) develop high blood pressure and kidney disease. Previous studies have shown that a commonly occurring chemical, nitric oxide (NO), is reduced in ADPKD, and may…
- Conditions
- Autosomal Dominant Polycystic Kidney, Hypertension, Endothelial Dysfunction
- Trial name
- BEET-PKD (NCT05401409)
- Testing
- Beetroot juice, Nitrate-depleted beetroot juice
- Where
- 2 sites in Australia
- Volunteers
- 60 people
- Sponsor
- Western Sydney Local Health District
Atopic Dermatitis
Dose-ranging Study of Nemolizumab in Atopic Dermatitis
Assess the efficacy of several subcutaneous doses of nemolizumab in moderate-to-severe atopic dermatitis (AD) subjects with severe pruritus receiving TCS, who were not adequately controlled with topical treatments.
- Trial name
- NCT03100344
- Testing
- Nemolizumab
- Where
- 67 sites in United States, Australia and 4 more
- Volunteers
- 226 people
- Sponsor
- Galderma R&D
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.
- Trial name
- NCT00775528
- Testing
- Pancrelipase Delayed Release
- Where
- 12 sites in United States
- Volunteers
- 19 people
- Sponsor
- Solvay Pharmaceuticals
Healthy
Using Magnetic Resonance (MR) to Understand the Effect of Erythromycin on Bowel Motility
Magnetic Resonance Imaging (MRI) has proven to be a valuable imaging technique for suspected small bowel disease. This technique depends, in part, on adequate distension of the small bowel.
- Trial name
- NCT01379183
- Testing
- Erythromycin, Magnetic Resonance Imaging, Barium Sulfate Solution
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic