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 recruitingHereditary Spastic Paraplegia
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
First Affiliated Hospital of Wannan Medical College · Oct 27th · NCT07838883 ↗
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.
Tuesday, October 27th
New sites
Hunter Syndrome · Added 1 new site: Porto Alegre, Brazil
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 · Denali Therapeutics Inc. · NCT06075537 ↗
New sites
Hodgkin Lymphoma · Added 1 new site: Atlanta, Georgia
Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution · Children's Hospital Medical Center, Cincinnati · NCT06131801 ↗
New sites
Granulomatosis with Polyangiitis · Added 2 new sites: Calgary, Canada, Petah Tikva, Israel
Roll-over Study for Participants Who Have Completed a Previous Clinical Study With Benralizumab (Fasenra) and Benefit From Continued Treatment · AstraZeneca · NCT07444567 ↗
New sites
Hypoparathyroidism · Added 2 new sites: Omaha, Nebraska, Oakville, Canada
A Study to Investigate Canvuparatide Compared With Placebo in Adult Patients With Hypoparathyroidism · MBX Biosciences · NCT07699471 ↗
New sites
Hypoparathyroidism · Expanded from 8 to 9 sites
A Phase 3 Randomized Clinical Trial to Investigate the Safety and Efficacy of Palopegteriparatide at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism · Ascendis Pharma Bone Diseases A/S · NCT07081997 ↗
New sites
Huntington Disease · Added 1 new site: Parkville, Australia
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease · Sarepta Therapeutics, Inc. · NCT07536061 ↗
New sites
Homozygous Familial Hypercholesterolemia · Added 1 new site: Warsaw, Poland
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia · Arrowhead Pharmaceuticals · NCT07473843 ↗
Now recruiting
Hereditary Spastic Paraplegia · Now recruiting (was Active Not Recruiting)
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province · First Affiliated Hospital of Wannan Medical College · NCT07838883 ↗
New sites
Hereditary Hemorrhagic Telangiectasia · Added 1 new site: St Louis, Missouri
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT) · Atavistik Bio, Inc · NCT07601425 ↗
New sites
Hemophilia B · Added 2 new sites: Dallas, Texas, Oxford, United Kingdom
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 B · Added 1 new site: San Diego, California
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 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors · Pfizer · NCT05145127 ↗
New sites
Hemophilia A · Added 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors · Pfizer · NCT05145127 ↗
New sites
Hemophilia A · Added 4 new sites: Rosario, Argentina, Vienna, Austria, Campinas, Brazil and 1 more
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A · Hoffmann-La Roche · NCT07416526 ↗
New sites
Guillain-Barré Syndrome · Added 18 new sites: Phoenix, Arizona, Loma Linda, California, Palo Alto, California and 15 more
An Open Label Clinical Study to Evaluate Tanruprubart (Also Commonly Known as ANX005) in Participants With Guillain-Barré Syndrome (FORWARD Study) · Annexon, Inc. · NCT07020819 ↗
New sites
Graves’ Disease · Added 1 new site: San Juan, Puerto Rico
Study to Determine if BHV-1300 is Effective and Safe in Adults With Graves' Disease · Biohaven Therapeutics Ltd. · NCT07661056 ↗
Graves' disease is not rare; Trial Friend includes it because its rare complication, thyroid eye disease, anchors it to rare autoimmune drug development.
New sites
Graves’ Disease · Added 1 new site: Humble, Texas
A Study to Learn About The Effects of Felzartamab on Thyroid Function and Its Safety in Adults With Graves' Disease Ages 18 to 75 Years Old · Biogen · NCT07722546 ↗
Graves' disease is not rare; Trial Friend includes it because its rare complication, thyroid eye disease, anchors it to rare autoimmune drug development.
New sites
Giant Cell Arteritis · Added 1 new site: Ottawa, Canada
Optic Nerve Sheath Ultrasound in Giant Cell Arteritis · Centre Integre Universitaire de Sante et Services Sociaux du Nord de l'ile de Montreal · NCT05749094 ↗
New sites
Geographic Atrophy · Added 3 new sites: Oxnard, California, Walnut Creek, California, Stuart, Florida
A Safety and Efficacy Study of a One-time Intravitreal Injection of SAR446597 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration · Sanofi · NCT07215234 ↗
Geographic atrophy is not rare; Trial Friend includes it because its complement-targeted therapies connect directly to rare retinal and complement diseases.
Enrollment
Gastrointestinal Stromal Tumor · Target enrollment decreased from 110 to 58
A Prospective, Randomized, Multicenter, Comparative Study of the Efficacy of Imatinib Resumption Combined With Atezolizumab Versus Imatinib Resumption Alone in Patients With Unresectable Advanced Gastrointestinal Stromal Tumors (GIST) After Failure of Standard Treatments · Centre Leon Berard · NCT05152472 ↗
Monday, October 26th
New sites
Focal Epilepsy · Added 1 new site: Birmingham, Alabama
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1 · Rapport Therapeutics Inc. · NCT07563881 ↗
Focal epilepsy is not rare; Trial Friend includes it because many rare genetic epilepsies present as focal seizures and share its drug pipeline.
New sites
Essential Thrombocythemia · Added 3 new sites: Salzburg, Austria, Palermo, Italy, Bursa, Turkey (Türkiye)
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Focal Segmental Glomerulosclerosis · Added 5 new sites: Toronto, Canada, Pécs, Hungary, Szeged, Hungary and 2 more
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease · Sanofi · NCT06500702 ↗
New sites
Focal Epilepsy · Added 1 new site: Seoul, South Korea
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1 · Rapport Therapeutics Inc. · NCT07563881 ↗
Focal epilepsy is not rare; Trial Friend includes it because many rare genetic epilepsies present as focal seizures and share its drug pipeline.
New sites
Essential Thrombocythemia · Added 9 new sites: Austin, Texas, Vienna, Austria, Curitiba, Brazil and 6 more
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) · Novartis Pharmaceuticals · NCT07357727 ↗
New sites
Essential Thrombocythemia · Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia · Disc Medicine, Inc · NCT05320198 ↗
New sites
Epidermolysis Bullosa · Added 3 new sites: Modena, Italy, Gdansk, Poland, Lodz, Poland
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study · Xinnate AB · NCT06594393 ↗
New sites
Duchenne 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
Duchenne 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 ↗
Enrollment
Dravet Syndrome · Target enrollment increased from 47 to 113
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome · Encoded Therapeutics · NCT05419492 ↗
New sites
Dilated Cardiomyopathy · Added 1 new site: New York, New York
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) · Affinia Therapeutics · NCT07426419 ↗
DCM overall is not rare; Trial Friend includes it because its single-gene inherited forms are individually rare and drive the gene-therapy trial pipeline.
New sites
Diffuse Intrinsic Pontine Glioma · Added 3 new sites: Brussels, Belgium, Leuven, Belgium, Athens, Greece
Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0 · Gustave Roussy, Cancer Campus, Grand Paris · NCT05476939 ↗
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 ↗