Rare Disease Trial Updates: April 2026
In April 2026, Trial Friend tracked 103 rare disease clinical trial updates across 45 conditions, including 0 FDA-related actions and 14 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.
This is a permanent monthly archive. For the latest updates, see the live current events feed.
Major Updates: FDA Actions, Status Changes & Results
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
No longer enrolling new participants
Trial Site & Enrollment Activity
Added 1 new site: Seoul, South Korea
Added 8 new sites: Córdoba, Argentina, Buenos Aires, Argentina, Edegem, Belgium and 5 more
Added 8 new sites: Rochester, Minnesota, CABA, Argentina, Curitiba, Brazil and 5 more
Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more
Reduced from 36 to 30 sites
Target enrollment decreased from 66 to 42
Eligibility criteria have been updated
Added 2 new sites: Boston, Massachusetts, New York, New York
Target enrollment decreased from 30 to 23
Added 1 new site: Los Angeles, California
Added 7 new sites: New York, New York, Houston, Texas, Copenhagen, Denmark and 4 more
Added 2 new sites: Salt Lake City, Utah, Milwaukee, Wisconsin
Target enrollment decreased from 80 to 60
Added 1 new site: Boston, Massachusetts
Eligibility criteria have been updated
Added 1 new site: Los Angeles, California
Added 2 new sites: La Jolla, California, Palo Alto, California
Added 1 new site: Boston, Massachusetts
Added 7 new sites: Santa Rosa, California, Ann Arbor, Michigan, Philadelphia, Pennsylvania and 4 more
Eligibility criteria have been updated
Added 1 new site: Atlanta, Georgia
Added 2 new sites: Fairway, Kansas, Montreal, Canada
Added 2 new sites: Westerville, Ohio, Austin, Texas
Added 2 new sites: Tbilisi, Georgia, Tbilisi, Georgia
Added 1 new site: Vitoria-Gasteiz, Spain
Eligibility criteria have been updated
Eligibility criteria have been updated
Added 2 new sites: Birmingham, Alabama, Boston, Massachusetts
Added 1 new site: Rancho Cordova, California
Target enrollment increased from 50 to 95
Eligibility criteria have been updated
Target enrollment decreased from 90 to 32
Target enrollment increased from 30 to 50
Added 2 new sites: New York, New York, Seattle, Washington
Added 7 new sites: Gainesville, Florida, Tampa, Florida, Portland, Oregon and 4 more
Added 1 new site: South Brisbane, Australia
Added 1 new site: Seoul, South Korea
Added 1 new site: Chapel Hill, North Carolina
Added 4 new sites: Salvador, Brazil, Hamburg, Germany, Gurugram, India and 1 more
Added 44 new sites: Maitland, Florida, Smyrna, Georgia, Spokane, Washington and 41 more
Added 7 new sites: Porto Alegre, Brazil, Baotou, China, Changchun, China and 4 more
Added 11 new sites: Brussels, Belgium, Bron, France, Katowice, Poland and 8 more
Added 1 new site: Boston, Massachusetts
Added 1 new site: Dallas, Texas
Eligibility criteria have been updated
Added 2 new sites: Minneapolis, Minnesota, Charlotte, North Carolina
Eligibility criteria have been updated
Eligibility criteria have been updated
Eligibility criteria have been updated
Added 2 new sites: Pontiac, Michigan, Dallas, Texas
Reduced from 32 to 30 sites
Eligibility criteria have been updated
Eligibility criteria have been updated
Added 2 new sites: Shenzhen, China, Wuhan, China
Eligibility criteria have been updated
Added 1 new site: Stony Brook, New York
Added 4 new sites: Leuven, Belgium, Bydgoszcz, Poland, Katowice, Poland and 1 more
Added 5 new sites: Leuven, Belgium, Bydgoszcz, Poland, Krakow, Poland and 2 more
Added 17 new sites: Bordeaux, France, Toulouse, France, Montpellier, France and 14 more
Added 1 new site: Eugene, Oregon
Added 1 new site: Sendai, Japan
Eligibility criteria have been updated
Added 1 new site: Melbourne, Australia
Eligibility criteria have been updated
Added 1 new site: Sendai, Japan
Eligibility criteria have been updated
Added 1 new site: Melbourne, Australia
Eligibility criteria have been updated
Added 2 new sites: Nanning, China, New Delhi, India
Added 6 new sites: Tampa, Florida, Beijing, China, Shanghai, China and 3 more
Added 6 new sites: Tampa, Florida, Beijing, China, Shanghai, China and 3 more
Eligibility criteria have been updated
Eligibility criteria have been updated
Eligibility criteria have been updated
Added 4 new sites: Bochum, Germany, Bochum, Germany, Cologne, Germany and 1 more
Added 2 new sites: Kansas City, Missouri, Sosnowiec, Poland
Reduced from 117 to 111 sites
Eligibility criteria have been updated
Added 1 new site: Sendai, Japan
Eligibility criteria have been updated
Added 1 new site: Melbourne, Australia
Eligibility criteria have been updated
Added 1 new site: Houston, Texas
Added 5 new sites: Phoenix, Arizona, Boston, Massachusetts, Ann Arbor, Michigan and 2 more
Eligibility criteria have been updated
Added 2 new sites: San Jose, California, Berlin, Germany
Added 4 new sites: Budapest, Hungary, Maribor, Slovenia, Fatih, Turkey (Türkiye) and 1 more
Added 5 new sites: Gainesville, Florida, Fuzhou, China, Heraklion, Greece and 2 more
Added 2 new sites: Orlando, Florida, Billings, Montana