Clinical trials: Prion Disease
Prion Disease
A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD)
This study will test a Regeneron drug called ALN-6457 ("study drug") to see if it can help treat Prion Disease (PrD). In PrD, the prion protein folds into an abnormal shape.
- Trial name
- NCT07832370
- Testing
- ALN-6457
- Volunteers
- 60 people
- Sponsor
- Regeneron Pharmaceuticals
Prion Disease
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
- Trial name
- NCT06153966
- Testing
- ION717
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures