Recent rare disease FDA decisionsAll decisions →
Approved Sep 30Camzyos (mavacamten)Obstructive hypertrophic cardiomyopathy, adolescentsApproved Sep 28Emcitate (tiratricol)MCT8 deficiencyApproved Sep 25Zilurgisertib (Atebrioz)Fibrodysplasia ossificans progressiva (FOP)Approved Sep 25Gazyva (obinutuzumab)Idiopathic nephrotic syndromeApproved Sep 23Lirafugratinib (Lyrfigtu)Cholangiocarcinoma (FGFR2 fusion)Rare disease trials opening to new patients
Studies that started recruiting again or added locations in the last 30 days.
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 10 · NCT04557462 ↗
Now recruitingFamilial Hypertrophic Cardiomyopathy
Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository
Montreal Heart Institute · Oct 9 · NCT05100420 ↗
Now recruitingAmyotrophic Lateral Sclerosis
Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS)
National Institute of Neurological Disorders and Stroke (NINDS) · Oct 7 · NCT07204977 ↗
Now recruitingFabry Disease
Anderson-Fabry Disease Fitness Improvement Training
General University Hospital, Prague · Sep 23 · NCT07495410 ↗
Now recruitingRetinitis Pigmentosa
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Octant, Inc. · Sep 18 · NCT07408232 ↗
Now recruitingMultiple Sclerosis
Cannabis (THC vs. CBD) in Multiple Sclerosis
University of Calgary · Sep 13 · NCT06261489 ↗
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.
Monday, October 12
New sites
Polycythemia Vera · Added 7 new sites: Osaka, Japan, Tokyo, Japan, Tokyo, Japan and 4 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 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Phenylketonuria · Added 1 new site: Chicago, Illinois
A Study of MZE782 in Adults With PKU · Maze Therapeutics · NCT07694440 ↗
New sites
Narcolepsy Type 1 · Added 1 new site: Dallas, Texas
A Long-Term Study of ALKS 2680 in Subjects With Narcolepsy and Idiopathic Hypersomnia · Alkermes, Inc. · NCT06767683 ↗
Enrollment
Narcolepsy Type 1 · Target enrollment increased from 256 to 786
A Long-Term Study of ALKS 2680 in Subjects With Narcolepsy and Idiopathic Hypersomnia · Alkermes, Inc. · NCT06767683 ↗
New sites
Narcolepsy Type 1 · Added 2 new sites: Ghent, Belgium, Kaohsiung City, Taiwan
A Study to Evaluate the Efficacy and Safety of ALKS 2680 in Adults With Narcolepsy Type 1 · Alkermes, Inc. · NCT07455383 ↗
New sites
Narcolepsy Type 1 · Added 3 new sites: Spokane, Washington, Diest, Belgium, Kumamoto, Japan
A Study to Evaluate the Efficacy, Safety and Tolerability of ALKS 2680 in Adults With Narcolepsy Type 1 (Brilliance NT1 - 304) · Alkermes, Inc. · NCT07540897 ↗
New sites
Myotonic Dystrophy · Added 2 new sites: Gainesville, Florida, Baltimore, Maryland
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 · Dyne Therapeutics · NCT07486934 ↗
New sites
Myelofibrosis · Added 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Myelodysplastic Syndromes · Added 5 new sites: Ruskin, Florida, Tampa, Florida, Tampa, Florida and 2 more
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
Myelodysplastic Syndromes · Added 2 new sites: Minneapolis, Minnesota, Sturgeon Bay, Wisconsin
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial) · National Cancer Institute (NCI) · NCT06577441 ↗
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 6 new sites: Monroe, Louisiana, Houston, Texas, Red Oak, Texas and 3 more
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions · Takeda · NCT06499285 ↗
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 1 new site: Yamagata, Japan
A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes · Takeda · NCT07319845 ↗
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 2 new sites: Maidstone, United Kingdom, Oxford, United Kingdom
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study) · Taiho Oncology, Inc. · NCT04256317 ↗
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
Myasthenia Gravis · Added 6 new sites: Seattle, Washington, CABA, Argentina, São Paulo, Brazil and 3 more
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG) · Amgen · NCT06987539 ↗
New sites
Myasthenia Gravis · Added 1 new site: Málaga, Spain
A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis · UCB Biopharma SRL · NCT07463521 ↗
New sites
Multiple Sclerosis · Added 3 new sites: Worcester, Massachusetts, Albuquerque, New Mexico, New York, New York
A Study of Orelabrutinib in Patients With Secondary Progressive Multiple Sclerosis · Zenas BioPharma (USA), LLC · NCT07299019 ↗
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 30 new sites: Mostar, Bosnia and Herzegovina, Haryāna, India, Raipur, India and 27 more
Study to Evaluate the Efficacy, Safety and Tolerability of IMU-838 in Patients With Relapsing Multiple Sclerosis · Immunic AG · NCT05201638 ↗
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 2 new sites: Chieti, Italy, Lviv, 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
Multiple Sclerosis · Added 6 new sites: São Paulo, Brazil, Frankfurt am Main, Germany, Bonn, Germany and 3 more
A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of RO7268489 as Add-on Therapy to Ocrelizumab, in Participants With Progressive Forms of Multiple Sclerosis (MS) · Hoffmann-La Roche · NCT07282574 ↗
MS is not rare; Trial Friend includes it because its trial-search challenges and treatment research overlap heavily with rare neurological diseases.
Sunday, October 11
New sites
Mesothelioma · Added 5 new sites: Los Angeles, California, Los Angeles, California, New York, New York and 2 more
Lead-212 PSV359 Therapy for Patients With Solid Tumors · Perspective Therapeutics · NCT06710756 ↗
Enrollment
Mesothelioma · Target enrollment increased from 884 to 952
Rinatabart Sesutecan (Rina-S, PRO1184, GEN1184) for Advanced Solid Tumors (GCT1184-01/ PRO1184-001) · Genmab · NCT05579366 ↗
New sites
Mastocytosis · Added 4 new sites: Scottsdale, Arizona, Tucson, Arizona, La Jolla, California and 1 more
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations · Blueprint Medicines Corporation · NCT07143669 ↗
New sites
Mastocytosis · Added 15 new sites: La Jolla, California, Garran, Australia, Leuven, Belgium and 12 more
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis · Blueprint Medicines Corporation · NCT04910685 ↗
New sites
Mastocytosis · Added 5 new sites: Berlin, Germany, Freiburg im Breisgau, Germany, Leipzig, Germany and 2 more
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany · Blueprint Medicines Corporation · NCT07255638 ↗
Saturday, October 10
New sites
Lennox-Gastaut Syndrome · Added 10 new sites: Changsha, China, Shanghai, China, Debrecen, Hungary and 7 more
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome · Epygenix · NCT05066217 ↗
New sites
IgA Nephropathy · Added 1 new site: Metz, France
Therapeutic Effect of Hydroxychloroquine on Immunoglobulin A (IgA) Nephropathy Course QUIgAN Study · Centre Hospitalier Universitaire de Saint Etienne · NCT06350630 ↗
Now recruiting
IgA Nephropathy · Now recruiting (was Active Not Recruiting)
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 · NCT04557462 ↗
New sites
IgA Nephropathy · Added 8 new sites: Newark, Delaware, Houston, Texas, Roeselare, Belgium and 5 more
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 · NCT04557462 ↗
Enrollment
IgA Nephropathy · Target enrollment increased from 448 to 540
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 · NCT04557462 ↗
New sites
Idiopathic Pulmonary Fibrosis · Added 3 new sites: Alexandroupoli, Greece, Heraklion, Greece, Taipei, Taiwan
Study to Evaluate the Efficacy, Safety, and Tolerability of PIPE 791 in Subjects With Idiopathic Pulmonary Fibrosis · Contineum Therapeutics · NCT07284459 ↗
New sites
Hodgkin Lymphoma · Added 2 new sites: Phoenix, Arizona, Macomb, Michigan
A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab · National Cancer Institute (NCI) · NCT05675410 ↗
New sites
Hereditary Hemorrhagic Telangiectasia · Added 3 new sites: Augusta, Georgia, Indianapolis, Indiana, Salt Lake City, Utah
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT) · Atavistik Bio, Inc · NCT07601425 ↗
New sites
Hemophilia B · Added 2 new sites: Westmead, Australia, Lille, France
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B · CSL Behring · NCT07080905 ↗
New sites
Hemophilia B · Added 1 new site: Phoenix, Arizona
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B · Regeneron Pharmaceuticals · NCT06379789 ↗
New sites
Hemophilia A · Added 17 new sites: Miami, Florida, Ann Arbor, Michigan, Minneapolis, Minnesota and 14 more
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A · Hoffmann-La Roche · NCT07416604 ↗
Friday, October 9
New sites
Friedreich Ataxia · Added 1 new site: Tampa, Florida
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia · Larimar Therapeutics, Inc. · NCT07778836 ↗
New sites
Friedreich Ataxia · Added 6 new sites: San Francisco, California, Iowa City, Iowa, Columbus, Ohio and 3 more
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia · Larimar Therapeutics, Inc. · NCT06447025 ↗
Now recruiting
Familial Hypertrophic Cardiomyopathy · Now recruiting (was Enrolling By Invitation)
Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository · Montreal Heart Institute · NCT05100420 ↗
HCM is not rare; Trial Friend includes it because individually rare sarcomere gene mutations drive it and its precision-therapy pipeline.
New sites
Familial Hypertrophic Cardiomyopathy · Added 4 new sites: Winnipeg, Canada, Kingston, Canada, Montreal, Canada and 1 more
Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository · Montreal Heart Institute · NCT05100420 ↗
HCM is not rare; Trial Friend includes it because individually rare sarcomere gene mutations drive it and its precision-therapy pipeline.