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 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.
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 ↗
Enrollment
Prader-Willi Syndrome · Target enrollment decreased from 150 to 111
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment · Rhythm Pharmaceuticals, Inc. · NCT06239116 ↗
New sites
Polycythemia Vera · Added 22 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 19 more
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Pheochromocytoma · Added 1 new site: Orlando, Florida
Targeted Alpha-Particle Therapy for Advanced Somatostatin Receptor Type 2 (SSTR2) Positive Tumors · Perspective Therapeutics · NCT05636618 ↗
New sites
Phelan-McDermid Syndrome · Added 2 new sites: Chicago, Illinois, Charlottesville, Virginia
An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome · Neuren Pharmaceuticals Limited · NCT07593391 ↗
Tuesday, October 20th
New sites
Myelofibrosis · Added 1 new site: Hamburg, Germany
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Myelodysplastic Syndromes · Added 155 new sites: Birmingham, Alabama, Phoenix, Arizona, Los Angeles, California and 152 more
Lisaftoclax (APG-2575) Combined With Azacytidine (AZA) in the Treatment of Patients With Higher-risk Myelodysplastic Syndrome (GLORA-4). · Ascentage Pharma Group Inc. · NCT06641414 ↗
MDS collectively is not rare; Trial Friend includes it because many MDS subtypes are individually rare and closely tied to rare blood cancers.
New sites
Myelodysplastic Syndromes · Added 3 new sites: Bismarck, North Dakota, Fargo, North Dakota, Fargo, North Dakota
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) · National Cancer Institute (NCI) · NCT05564390 ↗
MDS collectively is not rare; Trial Friend includes it because many MDS subtypes are individually rare and closely tied to rare blood cancers.
New sites
Multiple Sclerosis · Added 1 new site: Münster, Germany
A Study to Investigate Effects of Ocrelizumab Treatment on Neurofilament Light Chain (NfL) Levels and Participant Satisfaction in Participants With Multiple Sclerosis (MS) · Hoffmann-La Roche · NCT06780150 ↗
MS is not rare; Trial Friend includes it because its trial-search challenges and treatment research overlap heavily with rare neurological diseases.
New sites
Multiple Sclerosis · Added 3 new sites: Orlando, Florida, Warsaw, Poland, Ivano-Frankivsk, Ukraine
A Study of Orelabrutinib in Patients With Primary Progressive Multiple Sclerosis · Zenas BioPharma (USA), LLC · NCT07067463 ↗
MS is not rare; Trial Friend includes it because its trial-search challenges and treatment research overlap heavily with rare neurological diseases.
New sites
Paraganglioma · Added 1 new site: Orlando, Florida
Targeted Alpha-Particle Therapy for Advanced Somatostatin Receptor Type 2 (SSTR2) Positive Tumors · Perspective Therapeutics · NCT05636618 ↗
Enrollment
Neuromyelitis Optica · Target enrollment increased from 122 to 200
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies · Alexion Pharmaceuticals, Inc. · NCT05966467 ↗
New sites
Myotonic Dystrophy · Added 1 new site: Barcelona, Spain
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 · Dyne Therapeutics · NCT07486934 ↗
New sites
Myotonic Dystrophy · Added 1 new site: Westmead, Australia
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping · Avidity Biosciences, Inc. · NCT07587242 ↗
New sites
Myotonic Dystrophy · Added 1 new site: Winston-Salem, North Carolina
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I · Vertex Pharmaceuticals Incorporated · NCT06926621 ↗
New sites
Myelofibrosis · Added 21 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 18 more
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
Enrollment
Myelodysplastic Syndromes · Target enrollment increased from 160 to 210
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) · Takeda · NCT04419649 ↗
MDS collectively is not rare; Trial Friend includes it because many MDS subtypes are individually rare and closely tied to rare blood cancers.