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 ↗
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.
Thursday, October 15
Phase
Becker Muscular Dystrophy · Moved from N/A to Phase 2
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients · University of Florida · NCT07766980 ↗
New sites
Becker Muscular Dystrophy · Added 1 new site: St Louis, Missouri
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101 · Satellos Bioscience, Inc. · NCT06867107 ↗
New sites
Becker Muscular 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 ↗
Enrollment
Batten Disease · Target enrollment increased from 16 to 26
A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease · Tern Therapeutics, LLC · NCT05791864 ↗
New sites
ATTR Amyloidosis (Transthyretin Amyloidosis) · Added 3 new sites: La Jolla, California, Honolulu, Hawaii, Würzburg, Germany
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting · Alnylam Pharmaceuticals · NCT07358078 ↗
New sites
ATTR Amyloidosis (Transthyretin Amyloidosis) · Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting · Bayer · NCT07235462 ↗
New sites
ATTR Amyloidosis (Transthyretin Amyloidosis) · Added 4 new sites: Belgrano, Argentina, Salvador, Brazil, Guadalajara, Mexico and 1 more
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy · Alnylam Pharmaceuticals · NCT07223203 ↗
New sites
Angelman Syndrome · Added 1 new site: Miami, Florida
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. · OHB Pediatrics Ltd. · NCT07605429 ↗
New sites
Amyotrophic Lateral Sclerosis · Added 2 new sites: Lübeck, Germany, London, United Kingdom
FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS · Trace Neuroscience, Inc. · NCT07674667 ↗
Enrollment
Amyotrophic Lateral Sclerosis · Target enrollment increased from 30 to 60
A Study to Assess the Safety, Tolerability, and Pharmacology of Darifenacin in Patients With ALS · Oliver Blanchard · NCT06249867 ↗
Enrollment
Achondroplasia · Target enrollment increased from 6 to 13
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） · Kyowa Kirin Co., Ltd. · NCT06926491 ↗
Tuesday, October 13
New sites
Sjögren's Syndrome · Added 7 new sites: Voorhees Township, New Jersey, Wilmington, North Carolina, Sydney, Canada and 4 more
Two Arm, Double-blind, Phase III Study Assessing Efficacy and Safety of Ianalumab Versus Placebo, in Participants With Sjögren's Disease With High Symptom Burden · Novartis Pharmaceuticals · NCT07621809 ↗
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
Sjögren's Syndrome · Added 2 new sites: Clearwater, Florida, Trois-Rivières, Canada
A Phase 2a/b Study to Assess the Efficacy, Safety and Tolerability of DDY391 in Participants With Sjögren's Disease · Novartis Pharmaceuticals · NCT07737743 ↗
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
Wild-Type ATTR Cardiac Amyloidosis · Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting · Bayer · NCT07235462 ↗
New sites
Systemic Sclerosis · Added 2 new sites: Henan, China, Seoul, South Korea
A Phase 1/2 Study of PRO-203 in Healthy Volunteers and Participants With Systemic Sclerosis. · Prolium Bioscience, Inc · NCT07641634 ↗
New sites
Systemic 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
Systemic 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 ↗
New sites
Stargardt Disease · Added 1 new site: Philadelphia, Pennsylvania
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR) · Ascidian Therapeutics, Inc · NCT06467344 ↗
New sites
Stargardt Disease · Added 1 new site: Boston, Massachusetts
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) · Ascidian Therapeutics, Inc · NCT06445322 ↗
New sites
Spinal Muscular Atrophy · Added 5 new sites: Randwick, Australia, Parkville, Australia, Calgary, Canada and 2 more
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy · argenx · NCT07287982 ↗
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.