Recent rare disease FDA decisionsAll decisions →
Approved Oct 21stUltomiris (ravulizumab)IgA nephropathyApproved Oct 15thEnspryng (satralizumab)Thyroid eye disease (TED)Approved Sep 30thCamzyos (mavacamten)Obstructive hypertrophic cardiomyopathy, adolescentsApproved Sep 28thEmcitate (tiratricol)MCT8 deficiencyApproved Sep 25thZilurgisertib (Atebrioz)Fibrodysplasia ossificans progressiva (FOP)Rare disease trials opening to new patients
Studies that started recruiting again or added locations in the last 30 days.
Now recruitingAlpha-1 Antitrypsin Deficiency
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Wave Life Sciences USA, Inc. · Oct 24th · NCT06405633 ↗
Now recruitingSystemic Sclerosis
Phase IIa Trial of Anti-CD19 CAR T-Cells in Systemic Sclerosis Resistant to Immunosuppressive Therapy
University Hospital, Montpellier · Oct 22nd · NCT07493395 ↗
Now recruitingIgA Nephropathy
A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy
Novartis Pharmaceuticals · Oct 10th · NCT04557462 ↗
Now recruitingFamilial Hypertrophic Cardiomyopathy
Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository
Montreal Heart Institute · Oct 9th · NCT05100420 ↗
Now recruitingAmyotrophic Lateral Sclerosis
Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS)
National Institute of Neurological Disorders and Stroke (NINDS) · Oct 7th · NCT07204977 ↗
Most new trial sites by condition
Number of trials that added a location. Tap a condition to see them.
Rare disease news explained
Plain-language stories from News and Guides on the decisions and trials above.
All rare disease trial and FDA updates
Every change picked up from ClinicalTrials.gov and the FDA, newest first. A trial appears only when its own listed conditions name the disease.
Sunday, October 25th
New sites
Chronic Inflammatory Demyelinating Polyneuropathy · Added 11 new sites: Scottsdale, Arizona, Los Angeles, California, Orange, California and 8 more
A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) · Sanofi · NCT06290141 ↗
New sites
Chronic Inflammatory Demyelinating Polyneuropathy · Added 2 new sites: Montreal, Canada, Ottawa, Canada
A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases · Kite, A Gilead Company · NCT07304154 ↗
New sites
Cholangiocarcinoma · Added 2 new sites: Cologne, Germany, Düsseldorf, Germany
Durvalumab + Intraductal Radiofrequency Ablation (ID-RFA) in Extra- and Intrahepatic Cholangiocarcinoma · Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest · NCT06440993 ↗
Enrollment
Cholangiocarcinoma · Target enrollment decreased from 66 to 17
Study of Chemotherapy, With or Without Binimetinib in Advanced Biliary Tract Cancers in 2nd Line Setting (A ComboMATCH Treatment Trial) · National Cancer Institute (NCI) · NCT05564403 ↗
New sites
Cholangiocarcinoma · Added 3 new sites: Bamberg, Germany, Berlin, Germany, Freiburg im Breisgau, Germany
Ivosidenib in Locally Advanced or Metastatic Cholangiocarcinoma With IDH1 R132 Mutation After at Least One Prior Systemic Treatment - an Observational Study · iOMEDICO AG · NCT06607302 ↗
New sites
Becker Muscular Dystrophy · Added 7 new sites: Birmingham, Alabama, Los Angeles, California, Washington D.C., District of Columbia and 4 more
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy · Sarepta Therapeutics, Inc. · NCT06270719 ↗
New sites
Becker Muscular Dystrophy · Added 1 new site: Irbid, Jordan
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) · Wave Life Sciences USA, Inc. · NCT04906460 ↗
Saturday, October 24th
New sites
Autoimmune Hepatitis · Added 1 new site: Ottawa, Canada
Belimumab in Autoimmune Hepatitis · University Health Network, Toronto · NCT06381453 ↗
New sites
ATTR Amyloidosis (Transthyretin Amyloidosis) · Added 1 new site: Nangang, China
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy · Alnylam Pharmaceuticals · NCT07052903 ↗
New sites
ATTR Amyloidosis (Transthyretin Amyloidosis) · Added 3 new sites: Phoenix, Arizona, Buenos Aires, Argentina, Ottawa, Canada
CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) Amyloidosis · Novo Nordisk A/S · NCT07207811 ↗
New sites
Angelman Syndrome · Added 2 new sites: Nashville, Tennessee, Taipei, Taiwan
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. · OHB Pediatrics Ltd. · NCT07605429 ↗
Enrollment
Amyotrophic Lateral Sclerosis · Target enrollment increased from 12 to 36
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS · Vector Y Therapeutics · NCT07287397 ↗
New sites
Alpha-1 Antitrypsin Deficiency · Added 1 new site: Hanover, Germany
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) · AIRNA Corporation · NCT07431112 ↗
Now recruiting
Alpha-1 Antitrypsin Deficiency · Now recruiting (was Active Not Recruiting)
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD · Wave Life Sciences USA, Inc. · NCT06405633 ↗
Enrollment
Alpha-1 Antitrypsin Deficiency · Target enrollment increased from 24 to 48
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD · Wave Life Sciences USA, Inc. · NCT06405633 ↗
New sites
Acromegaly · Added 1 new site: Brasília, Brazil
A Study to Assess the Efficacy and Safety of Debio 4126 in Participants With Acromegaly Previously Treated With Somatostatin Analogs · Debiopharm International SA · NCT06930625 ↗
Friday, October 23rd
New sites
Von Willebrand Disease · Added 1 new site: Kingston, Canada
A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD · Unity Health Toronto · NCT06205095 ↗
Thursday, October 22nd
New sites
Systemic Sclerosis · Added 1 new site: Valencia, Spain
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease · CRISPR Therapeutics · NCT06925542 ↗
Enrollment
Spinal Muscular Atrophy · Target enrollment decreased from 28 to 19
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy · Hoffmann-La Roche · NCT05861999 ↗
New sites
Thyroid Eye Disease · Added 17 new sites: Glendale, Arizona, Beverly Hills, California, Canoga Park, California and 14 more
Study to Assess Linsitinib in Participants With Moderate to Severe Active Thyroid Eye Disease (TED) · Sling Therapeutics, Inc. · NCT07753603 ↗
Now recruiting
Systemic Sclerosis · Now recruiting (was Active Not Recruiting)
Phase IIa Trial of Anti-CD19 CAR T-Cells in Systemic Sclerosis Resistant to Immunosuppressive Therapy · University Hospital, Montpellier · NCT07493395 ↗
New sites
Systemic Juvenile Idiopathic Arthritis · Added 4 new sites: Athens, Greece, Chamberí, Spain, Madrid, Spain and 1 more
A Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Systemic Juvenile Idiopathic Arthritis (SKYPS) · Sanofi · NCT02991469 ↗
New sites
Spinal Muscular Atrophy · Added 2 new sites: São Paulo, Brazil, Ahmedabad, India
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) · Biogen · NCT07221669 ↗
New sites
Sjögren's Syndrome · Added 6 new sites: Miami, Florida, Durham, North Carolina, Oklahoma City, Oklahoma and 3 more
Phase 3 Study of Telitacicept in Active Primary Sjögren's Disease · Vor Biopharma · NCT07404865 ↗
Sjögren's is not rare; Trial Friend includes it because it is underdiagnosed, long neglected by drug development, and its pipeline overlaps rare autoimmune disease research.
New sites
Sickle Cell Disease · Added 4 new sites: New York, New York, Ribeirão Preto, Brazil, São Paulo, Brazil and 1 more
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease · Novartis Pharmaceuticals · NCT06546670 ↗
Enrollment
Sickle Cell Disease · Target enrollment decreased from 470 to 390
Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain · University of Pittsburgh · NCT06374238 ↗
New sites
Sickle Cell Disease · Added 4 new sites: Washington D.C., District of Columbia, Yenişehir, Turkey (Türkiye), Liverpool, United Kingdom and 1 more
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis · CSL Behring · NCT06699849 ↗
New sites
Sickle Cell Disease · Added 1 new site: Davis, California
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease · Mark Walters, MD · NCT04774536 ↗
Enrollment
Sickle Cell Disease · Target enrollment decreased from 750 to 377
Cooperative Assessment of Late Effects for SCD Curative Therapies · Vanderbilt University Medical Center · NCT05153967 ↗
New sites
Sickle Cell Disease · Added 19 new sites: Clovis, California, Los Angeles, California, Washington D.C., District of Columbia and 16 more
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease · Sanofi · NCT06975865 ↗
New sites
Sarcoidosis · Added 34 new sites: Birmingham, Alabama, Phoenix, Arizona, Tucson, Arizona and 31 more
A Study of Brepocitinib in Adults With Cutaneous Sarcoidosis · Priovant Therapeutics, Inc. · NCT06978725 ↗
Wednesday, October 21st
New sites
Polycythemia Vera · Added 15 new sites: Orange, California, Columbus, Ohio, Philadelphia, Pennsylvania and 12 more
INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV) · Ono Pharmaceutical Co., Ltd. · NCT07429266 ↗
New sites
Polycythemia Vera · Added 1 new site: Charlottesville, Virginia
A Clinical Trial of Bomedemstat in Participants With Polycythemia Vera (MK-3543-025). · Merck Sharp & Dohme LLC · NCT07722611 ↗
New sites
Rett Syndrome · Added 1 new site: Tbilisi, Georgia
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome · UCB BIOSCIENCES, Inc. · NCT07503444 ↗
New sites
Pulmonary Arterial Hypertension · Added 13 new sites: New Hyde Park, New York, New York, New York, New York, New York and 10 more
A Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder (TPIP) in Participants With Pulmonary Arterial Hypertension (PAH) · Insmed Incorporated · NCT07481981 ↗
New sites
Pulmonary Arterial Hypertension · Added 1 new site: Krakow, Poland
A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY) · AllRock Bio, Inc. · NCT07175038 ↗
New sites
Progressive Supranuclear Palsy · Added 22 new sites: Boca Raton, Florida, Lexington, Kentucky, New Orleans, Louisiana and 19 more
A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy · Novartis Pharmaceuticals · NCT07498426 ↗
New sites
Progressive Supranuclear Palsy · Added 7 new sites: Sun City, Arizona, Chicago, Illinois, Indianapolis, Indiana and 4 more
The Progressive Supranuclear Palsy Clinical Trial Platform · Adam Boxer · NCT07173803 ↗
New sites
Primary Biliary Cholangitis · Added 4 new sites: Roseville, California, Milwaukee, Wisconsin, San Miguel de Tucumán, Argentina and 1 more
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis · Gilead Sciences · NCT06051617 ↗
New sites
Prader-Willi Syndrome · Added 25 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 22 more
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome · Soleno Therapeutics, Inc. · NCT07450664 ↗