Find Clinical Trials & Research Studies

Cabaletta Bio is conducting a phase 1/2 clinical trial (RESET-SLE^TM) to evaluate the effects of the investigational cell treatment CABA-201 for the treatment of systemic lupus erythematosus (SLE) in patients with active lupus nephritis (LN) or active SLE without renal involvement.

Novartis Pharmaceuticals is sponsoring a phase 2, open-label study is to evaluate the safety and efficacy of rapcabtagene autoleucel in treating patients with active, refractory systemic lupus erythematosus (SLE) or active, refractory lupus nephritis (LN).

Alexion Pharmaceuticals, Inc. is sponsoring a study to evaluate the safety and efficacy of ravulizumab administered by intravenous (IV) infusion compared to placebo and demonstrate proof-of-concept of the efficacy of terminal complement inhibition in participants with lupus nephropathy.

Alexion Pharmaceuticals, Inc. is sponsoring a study to evaluate evaluate the efficacy and safety of ALXN2050 in adult participants with proliferative lupus nephritis (LN) or immunoglobulin A nephropathy (IgAN).

Protara Therapeutics is conducting a phase 2a/b single arm, open label study to evaluate the safety and efficacy of intracystic administration of TARA-002 in participants between 6 months to less than 18 years of age for the treatment of Macrocystic and Mixed Cystic Lymphatic Malformations

Ono Pharmaceutical Co. Ltd is sponsoring a phase 3, multi-regional, open-label, randomized study to evaluate the safety and efficacy of tirabrutinib alone compared with rituximab and temozolomide (R-TMZ) combination therapy in patients with Relapsed/Refractory Primary Central Nervous System Lymphoma (PCNSL).

Ono Pharmaceutical Co. Ltd is sponsoring a non-randomized, open-label, phase 2 study to evaluate the safety, efficacy, and pharmacokinetics of tirabrutinib in patients with primary central nervous system lymphoma (PCNSL).

Ono Pharmaceutical Co. Ltd is sponsoring a study to investigate the safety, tolerability, pharmacokinetics and preliminary efficacy of ONO-4685 in patients with relapsed or refractory T cell lymphoma.

The University of California, San Francisco is collaborating with Duke University on a study to determine maternal and fetal safety and feasibility of prenatal enzyme replacement therapy in fetuses with LSDs.

PrECOG, LLC is sponsoring a study to evaluate the effectiveness and potential side effects of ixazomib combined with ibrutinib chemotherapy in treating patients who have relapsed or refractory mantle cell lymphoma.

PrECOG, LLC is sponsoring a study to evaluate the effectiveness and potential side effects of venetoclax combined with bendamustine and rituximab chemotherapy in treating patients ages 60+ who have mantle cell lymphoma.

Blueprint Medicines Corporation is sponsoring a randomized, double-blind, placebo-controlled phase 2/3 study to evaluate the safety and efficacy of elenestinib in patients with Indolent Systemic Mastocytosis

Cogent Biosciences, Inc. is sponsoring a global phase 2 study to determine the safety, efficacy, pharmacokinetic and pharmacodynamic profiles of an investigational therapy bezuclastinib. Patients with systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) who are receiving or who are appropriate to receive treatment with azacitidine are currently being recruited for this study. Patients will receive the investigational therapy, bezuclastinib, while taking azacitidine for their AHN.

Cogent Biosciences, Inc. is sponsoring an expanded access program to provide the investigational therapy bezuclastinib to patients with a nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who have received and failed or been intolerant to at least one standard approved therapy and/or have no comparable or satisfactory alternative therapy options.Cogent Biosciences, Inc. is sponsoring an expanded access program to provide the investigational therapy bezuclastinib to patients with a nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who have received and failed or been intolerant to at least one standard approved therapy and/or have no comparable or satisfactory alternative therapy options.

Ionis Pharmaceuticals, Inc. is sponsoring a study to evaluate the safety and tolerability of ION440 in children and adults with MDS.

Incyte Corpopration is conducting a study to assess the clinical activity and safety of INCMGA00012 in patients with metastatic merkel cell carcinoma. For more information, visit: ClinicalTrials.gov:NCT035997

The National Cancer Institute is conducting a phase III study to determine how well pembrolizumab works compared to standard of care observation in treating patients with stage I-III Merkel cell cancer that has been completely removed by surgery.

Shire is conducting a study to evaluate the effects of intrathecal (IT) administration of SHP611 on gross motor function, using the gross motor function classification in metachromatic leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD.

ModernaTX, Inc. is sponsoring a study to evaluate the safety, pharmacokinetics and pharmacodynamics of mRNA-3705 in patients with MMA due to methylmalonyl-coenzyme A mutase deficiency. Estimated enrollment is 33 participants.

The National Human Genome Research Institute is conducting this study to evaluate patients with MMA to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it.

ModernaTX, Inc. is conducting a first-in-human phase 1/2 study to evaluate mRNA-3704 in patients 1-18 years of age with MMA due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have elevated plasma methylmalonic acid.

PTC Therapeutics is sponsoring a parallel-arm, double-blind, placebo-controlled study to evaluate the efficacy and safety of vatiquinone to treat mitochondrial disease in patients with refractory epilepsy.

Rare Thyroid Therapeutics International AB is sponsoring a phase 3, double-blind, randomized, multicenter, placebo-controlled study of male patients with MCT8 deficiency to determine if removal of tiratricol will lead to an increase of serum total T3 concentration compared to those who continue to receive tiratricol.

Rare Thyroid Therapeutics International AB is sponsoring an expanded access program to tiratricol as a treatment option in the U.S. for eligible patients with MCT8 deficiency.

Researchers at the University of British Columbia are conducting a survey to collect data about the natural history of Morquio B disease and late-onset GM1. Questions pertain to health, medical interventions and treatments, quality of life, presenting symptoms during life span, and diagnostic history.

REGENXBIO Inc. is conducting a phase I, first-in-human, multicenter, open-label, dose escalation study of RGX-111 gene therapy in patients with MPS I. Two, one time doses of RGX-111 will be studied in approximately 5 patients age 6 years or older. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period) and study participants will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-111.

REGENXBIO Inc. is conducting a clinical trial to study RGX-121 as a gene therapy for mucopolysaccharidosis type II (MPS II or Hunter syndrome). This is a Phase I/II, first-in-human, multicenter, open-label, dose escalation study of RGX-121. Two, one time doses of RGX-121 will be studied in approximately 6 children who have severe MPS II. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period) and then the children will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-121.

Genentech, Inc. is sponsoring a study to evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamic effects of ocrelizumab in pediatric and adolescent patients with relapsing-remitting multiple sclerosis.

Genentech, Inc. is sponsoring a phase 3 study to evaluate the safety and efficacy of ocrelizumab in comparison with fingolimod in pediatric and adolescent patients with relapsing-remitting multiple sclerosis.

Ono Pharmaceutical Co. Ltd is sponsoring a phase 2, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple doses of ONO-2808 in patients with multiple system atrophy.

Janssen Research & Development, LLC is sponsoring a randomized, open-label, phase 3b study to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis.

Janssen Research & Development, LLC is sponsoring an open-label, uncontrolled, multicenter study to evaluate the pharmacokinetics, pharmacodynamics, safety, and efficacy of nipocalimab in treating children between the ages of two and eighteen years old with generalized myasthenia gravis.

Janssen Research & Development, LLC is conducting a phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of nipocalimab administered to adults with generalized myasthenia gravis

Celgene is conducting a clinical trial of fedratinib in patients with DIPSS (dynamic international prognostic scoring system)-intermediate or high- risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF) and previously treated with Ruxolitinib to evaluate the percentage of patients with at least a 35% reduction of spleen volume and to evaluate the safety of fedratinib.

Incyte Corporation is sponsoring a study to compare the efficacy and safety of parsaclisib when combined with ruxolitinib versus placebo combined with ruxolitinib in patients with myelofibrosis who have suboptimal response while receiving ruxolitinib monotherapy.

Incyte Corporation is sponsoring a study to compare the efficacy of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in patients with myelofibrosis.

Incyte Corporation is sponsoring a study to assess the safety, tolerability, and efficacy of itacitinib immediate release tablets in patients with primary or secondary myelofibrosis who have received prior ruxolitinib and/or fedratinib monotherapy.

Lynk Pharmaceuticals is sponsoring a multicenter, open-label, phase 1 study designed to evaluate safety and tolerability of multi-kinase inhibitor LNK01002 in patients with primary myelofibrosis, or myelofibrosis due to polycythemia vera.

Cabaletta Bio is conducting a phase 1/2 clinical trial (RESET-Myositis^TM) to evaluate the effects of the investigational cell treatment CABA-201 in patients with dermatomyositis (DM), antisynthetase syndrome (ASyS) and immune-mediated necrotizing myopathy (IMNM) who have active disease.

Genentech, Inc. and the University of Pittsburgh, along with collaborating centers across the United States, are conducting a research study to examine the effectiveness of tocilizumab in adult myositis patients. Although tocilizumab is FDA-approved for use in rheumatoid arthritis, it is considered experimental when used for myositis. Basic inclusion criteria are: 1) medical diagnosis of polymyositis (PM) or dermatomyositis (DM); 2) symptoms of active disease, such as weakness of the large muscles closest to the trunk of the body, and/or abnormal muscle enzymes, and/or rash of DM; 3) have experienced little or no improvement of symptoms in response to taking steroids (prednisone) or other immunosuppressive (IS) medications, or could not tolerate other IS medications. Participants will be randomized (like a flipping a coin) to receive either intravenous tocilizumab or placebo (an inactive agent, like a sugar pill). The study includes 10 visits over a 48 week period (but study drug will only be given for monthly for 6 visits). Study procedures include: questionnaires, laboratory studies (blood draw), manual muscle testing, and a brief physical exam. Participants will receive the study drug and all other study procedures at no cost.

Novartis Pharmaceuticals is sponsoring a phase 2, randomized, open-label study to evaluate the safety and efficacy of rapcabtagene autoleucel in treating patients with severe refractory idiopathic inflammatory myopathies (IIM).

Takeda is conducting a study to assess the safety and tolerability of TAK-994 following multiple oral doses in patients with narcolepsy type 1.

Eloxx Pharmaceuticals, Inc. is conducting a phase 2 open label study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple dose levels of subcutaneously administered ELX-02 in patients with cystinosis who have one or more nonsense mutations in the CTNS (cystinosin) gene.

Exelixis is sponsoring a study to assess the effectiveness of zanzalintinib compared to everolimus in patients with previously treated, unresectable, locally advanced or metastatic neuroendocrine tumors.

Florida State University College of Medicine is conducting the NF1 Cognitive Aging Study to investigate cognitive patterns and predictors within middle-aged and older adults with NF1. Participants must have a diagnosis of NF1, be 40 years old or older, be able to read and speak English and reside in the U.S. Participation includes a virtual Zoom interview (1/2 hour) and online survey (1 hour). Participants will be paid $40. If interested, please complete this brief screening survey https://redcap.link/edpbo89j. If you have questions, email [email protected].

Florida State University College of Medicine is conducting the SMART-NF1 Study to improve understanding of cognitive, behavioral, and socioeconomic function in daily lives of adolescents with NF1. Participants must have a diagnosis of NF1, be 13-17 years old, have a third grade or higher level of English comprehension, live in the U.S. and have access to a smart phone. Participants and parents will be paid. If interested, please complete this brief screening survey https://redcap.link/SMART-NF1. If you have questions,  please email [email protected].

Recursion Pharmaceuticals Inc. is sponsoring a parallel-group, two-staged, phase 2/3, randomized, multi-center study to evaluate the safety and efficacy of REC-2282 in patients with progressive NF2 mutated meningiomas, with either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations.

The University of Rochester Department of Neurology is conducting a study to develop a patient reported outcome measure that will assess the health, symptoms, and disease burden of individuals with neuromyelitis optica spectrum disorder (NMOSD). This study aims to identify the symptoms that have the greatest impact on quality-of-life for individuals with NMOSD. Participants (age 18 and older) will complete an online survey asking about the symptoms of NMOSD they experience. The survey will take approximately 20-30 minutes to complete, and all responses will be strictly anonymous.

Interested individuals can click the link below to participate:

https://redcap.link/nmo-hi

Any questions or comments may also be directed to one of the study coordinators: Charlotte Engebrecht / 585-275-8605 / [email protected] or Matt Rathbun / 585-275-8585 / [email protected]

Claris Biotherapeutics, Inc. is sponsoring a study to evaluate the safety and efficacy of CSB-001 ophthalmic solution 0.1% in patients with stage 2 or 3 neurotrophic keratitis. Estimated enrollment is 108 participants.

BRIM Biotechnology Inc. is sponsoring a study to determine the efficacy of BRM424 ophthalmic solution in patients with stage 2 and stage 3 neurotrophic keratitis and evaluate safety and tolerability.

X4 Pharmaceuticals is sponsoring a study to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in patients with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections.

The Division of Genetics and Genomic Medicine at Washington University School of Medicine in St. Louis is conducting a study to determine if adrabetadex (VTS-270) administered intravenously is effective in treating acute liver disease in infants with NPC. Learn more about the Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C on ClinicalTrials.gov and from Laura Linneman, RN, Manager Clinical Trials, Pediatric Newborn Medicine at 314-454-4950 (office) or 314-706-3300 (cell).

Bristol-Myers Squibb is sponsoring a phase 3 study in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

The National Cancer Institute is conducting a natural history study of children and adults with ONB to better understand the course of the disease, tumor characteristics, response to treatments and management of the treatment.

The National Cancer Institute is conducting a trial to determine if giving the immunotherapy drug bintrafusp alfa can help ONB shrink or disappear.

Arcturus Therapeutics, Inc. is sponsoring a phase 2a, open-label, multiple ascending dose study to evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescents and adults with OTC deficiency.

Prevail Therapeutics is sponsoring a phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal PR001 administration in patients with moderate to severe Parkinson disease with at least 1 pathogenic GBA1 gene mutation.

Novartis Pharmaceuticals is sponsoring a study to evaluate the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HROoL) experienced by patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). The study seeks to establish a new real-world data (RWD) source to better understand symptom variably and HROoL among patients with PNH.

Regeneron Pharmaceuticals is sponsoring a study to evaluate the effect of pozelimab and cemdisiran combination therapy versus ravulizumab therapy on hemolysis and red blood cell transfusions in patients with active PNH who have not had complement inhibitor treatment or have not recently received complement inhibitor therapy.

Amryt Pharma is sponsoring a phase 3, randomized, multicenter, double-blind, placebo-controlled study to evaluate the safety and efficacy of daily subcutaneous metreleptin in treating patients with PL.

Ionis Pharmaceuticals, Inc. is sponsoring a study to evaluate the safety and tolerability of ION356 in children with Pelizaeus-Merzbacher disease and genetic confirmation of proteolipid protein 1 (PLP1) gene duplication.

Cabaletta Bio is conducting a phase 1/2 clinical trial (RESET-PV^TM) to evaluate the effects of the investigational cell treatment CABA-201 in patients with pemphigus vulgaris (PV) who have active disease.

Synlogic is sponsoring an open-label, single-arm phase 2 study to assess the safety and efficacy of SYNB1618 in adult patients with PKU. Evaluations and assessments will be conducted at the clinical site or by a home healthcare professional at an alternative location. Estimated enrollment is 12 participants.

Travere Therapeutics, Inc. is currently enrolling patients with pantothenate kinase-associated neurodegeneration (PKAN) in the FOsmetpantotenate Replacement Therapy (FORT) Study, a Phase 3 clinical study assessing the safety and efficacy of fosmetpantotenate (RE-024). Fosmetpantotenate is a potential treatment designed to restore the metabolic product of the missing or dysfunctional enzyme in PKAN. Patients with PKAN who are 6-65 years of age may be eligible to participate in the study.

Protagonist Therapeutics, Inc.is sponsoring a randomized, double-blind, open-label, phase 3 study to evaluate the safety and efficacy of rusfertide in patients with PV in maintaining hematocrit control and in improving symptoms.

PharmaEssentia is sponsoring a phase 3, randomized, open-label, multicenter study to evaluate the safety, tolerability and efficacy of ropeginterferon alfa-2b-njft (P1101) in adult patients with PV.

Genzyme is currently conducting a phase 4 prospective study aimed at characterizing the pharmacokinetics of aglucosidase alfa manufactured at the 4000L scale in patients with late-onset Pompe disease. Patients between 8 and 18 years of age with a confirmed diagnosis of Pompe disease and who have not been previously treated with aglucosidase alfa may be eligible to participate in the PAPAYA study.

Spark Therapeutics is sponsoring a study to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adult patients with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy.

Recordati Rare Diseases is conducting a double-blind randomized placebo-controlled dose-finding phase II study to assess the efficacy and safety of pasireotide s.c. in patients with post-bariatric hypoglycaemia.

Pierre Fabre Pharmaceuticals is conducting a multicenter, open-label, phase 3 study to determine the safety and efficacy of tabelecleucel for patients with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease after receiving a solid organ or allogeneic hematopoietic cell transplant and failure of rituximab or rituximab and chemotherapy.

Acadia Pharmaceuticals, Inc. is sponsoring a 12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of extreme hunger (hyperphagia) in patients with Prader-Willi syndrome.  

CymaBay Therapeutics, Inc. is sponsoring a study to evaluate seladelpar as a treatment for patients with PBC who have an inadequate control to or an intolerance to ursodeoxycholic acid. Determination of biochemical improvement in cholestasis markers will be based on ALP and total bilirubin, and the safety of seladelpar treatment will be compared to placebo.

GenKyoTex Suisse SA, a subsidiary of Calliditas Therapeutics AB is conducting a 52-week phase 2b/3 trial of setanaxib with a 52-week extension phase in patients with PBC and elevated liver stiffness. The main goal of this clinical trial is to evaluate the efficacy and safety of setanaxib in patients with PBC and elevated liver stiffness who had intolerance or inadequate response to ursodeoxycholic acid (UDCA). The trial will recruit patients in the US, Europe, Israel, Australia and New Zealand.

Ipsen is sponsoring a phase III randomized, parallel-group, double-blind, placebo-controlled, two-arm study to evaluate the efficacy and safety of elafibranor on long-term clinical outcomes in adults with PBC.

Advicenne is conducting a phase III, multicenter study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis defined by serum bicarbonate level in pediatric (6 months to <18 years) and adult (18 to 65 years) individuals with primary dRTA.

Travere Therapeutics, Inc. is sponsoring a study to determine the long-term nephroprotective potential of treatment with sparsentan as compared to an angiotensin receptor blocker in patients with FSGS.

Dicerna Pharmaceuticals, Inc. is sponsoring a study to evaluate the efficacy and safety of DCR-PHXC in children and adults with PH1 and PH2.

Dicerna Pharmaceuticals, Inc. is sponsoring a natural history study of adults, adolescents, and children (≥ 2 years of age) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre-existing stones detected by renal ultrasound at screening.

Dicerna Pharmaceuticals, Inc. is sponsoring a study to evaluate DCR-PHXC in patients with primary hyperoxaluria type 1 (PH1) or type two (PH2) who have severe renal impairment, with or without dialysis.