Find Clinical Trials & Research Studies

Ovid Therapeutics Inc. is sponsoring a study to investigate the effect of TAK-935 on the frequency of motor seizures in patients with 15q duplication syndrome during the maintenance period.

Ascendis Pharma, Inc. is sponsoring a natural history study to examine height velocity and comorbidities in children with ACH.

Ascendis Pharma, Inc. Is sponsoring a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating the safety, efficacy, and pharmacokinetics of TransCon CNP administered once weekly in prepubertal children with ACH.

AGTC is currently conducting a clinical study to evaluate the safety and efficacy of an investigational gene therapy in patients with achromatopsia caused by mutations in the CNGB3 gene. If you are eligible to join the study and choose to participate, all study-related treatment costs and medical testing will be provided at no charge. Compensation for your time and travel expenses may also be provided.

Ionis Pharmaceuticals is investigating the safety, tolerability, and efficacy of IONIS-GHR-LRx in up to 42 adult patients with acromegaly being treated with long-acting somatostatin receptor ligands. Clinicaltrials.gov-NCT03548415.

Ionis Pharmaceuticals, Inc. is conducting a short-term study to assess changes in serum insulin-like growth factor 1 (IGF-1) over a 16-week treatment period in patients with acromegaly being treated with long-acting somatostatin receptor ligands (SRL). Clinicaltrials.gov-NCT03548415

Camurus AB is investigating the efficacy and safety of octreotide subcutaneous depot (CAM2029) in a 24-week randomized trial in 78 patients with acromegaly and who are on treatment with long-acting somatostatin analogues (ClinicalTrials.gov Identifier: NCT04076462).

Camurus AB is conducting a 52-week open-label trial to assess the long-term safety of octreotide subcutaneous depot (CAM2029) in 140 patients with acromegaly and who are on treatment with long-acting somatostatin analogues (ClinicalTrials.gov Identifier: NCT04125836).

Astellas Pharma Global Development, Inc. is sponsoring a phase 1 and phase 2 study of gilteritinib in combination with chemotherapy in children, adolescents and young adults with FMS-like tyrosine kinase 3 (FLT3)/internal tandem duplication (ITD) positive relapsed or refractory AML. Estimated enrollment is 97 participants. More.

Rubius Therapeutics is sponsoring a phase 1, open label, multicenter, multidose, first-in-human (FIH) dose escalation and expansion to determine the safety and tolerability, pharmacology, and antitumor activity of RTX-240 in adult patients with relapsed/refractory acute myeloid leukemia. More.

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 AML. More.

Exicure, Inc. is sponsoring a phase 1b study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of intratumoral cavrotolimod injections alone and in combination with intravenous pembrolizumab or cemiplimab in patients with advanced solid tumors. A phase 2 study will evaluate cavrotolimod given in combination with pembrolizumab or cemiplimab in patients with Merkel cell carcinoma and cutaneous squamous cell carcinoma. More.

Rubius Therapeutics is sponsoring a phase 1/2, open label, multicenter, multidose, first-in-human (FIH) dose escalation and expansion to determine the safety and tolerability, recommended phase 2 dose and optimal dosing interval, pharmacology, and antitumor activity of RTX-240 in adult patients with relapsed/refractory or locally advanced solid tumors. More.

Regeneron Pharmaceuticals is sponsoring a study in collaboration with Sanofi to evaluate the efficacy of dupilumab on the annualized rate of exacerbations in patients with allergic bronchopulmonary aspergillosis. Estimated enrollment is 170 participants. More.

Dicerna Pharmaceuticals, Inc. is sponsoring a study to test the experimental drug DCR-A1AT to see if it helps people with AIATD-associated liver disease.

A study is enrolling adults diagnosed with A1PI deficiency and COPD. This study will evaluate the weekly administration of A1PI augmentation therapy in patients with A1PI and COPD. For more information, visit: ClinicalTrials.gov –  NCT02722304

Sanofi Genzyme is conducting a phase II, randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of SAR339375 in patients with Alport syndrome.

Caelum Biosciences, Inc. is sponsoring a study to determine the recommended dose of CAEL-101, to facilitate progression of further clinical trials, and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab) in patients with AL Amyloidosis.

Caelum Biosciences, Inc. is sponsoring a study to evaluate the effectiveness and safety of CAEL-101 in patients with Mayo Stage IIIa AL Amyloidosis.

Caelum Biosciences, Inc. is sponsoring a study to evaluate the effectiveness and safety of CAEL-101 in patients with Mayo Stage IIIb Amyloidosis.

Alnylam Pharmaceuticals is sponsoring a study to evaluate the efficacy and safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy.

Alnylam Pharmaceuticals is sponsoring a study to evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously once every 3 months compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

Alnylam Pharmaceuticals is sponsoring a global observational study to describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients; characterize the safety and effectiveness of patisiran as part of routine clinical practice in the real-world clinical setting; and describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) mutation.

Prothena Biosciences Ltd is sponsoring a phase 3, multicenter, global, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of birtamimab in treating Mayo Stage IV patients with AL amyloidosis.

Alexion Pharmaceuticals is sponsoring a study to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.

Apellis Pharmaceuticals, Inc. is sponsoring a 24-month (including open label), phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in patients with ALS.

Biogen is sponsoring a study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) gene variant with elevated neurofilament. Secondary objectives are to evaluate the safety, tolerability and effect of tofersen on pharmacodynamics and treatment response biomarkers when initiated prior to versus at the time of emergence of symptoms of ALS.

Helixmith Co., Ltd. is sponsoring a phase 2 study to evaluate the safety of intramuscular (IM) administration of Engensis in patients with ALS as compared to placebo.

Ionis Pharmaceuticals, Inc. is sponsoring a phase 3, randomized study to evaluate the safety, efficacy, and pharmacodynamics of intrathecally administered ION363 in treating ALS patients with fused in sarcoma mutations.

PTC Therapeutics is sponsoring a study to assess the effects and safety of PTC857 to treat patients with ALS

Ionis Pharmaceuticals, Inc. is sponsoring a phase 1-2a, open-label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecally administered ION582 in treating patients with Angelman syndrome.

Ovid Therapeutics Inc. is sponsoring a study to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric patients with Angelman syndrome.

Regeneron Pharmaceuticals is sponsoring a study to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST) and to evaluate the clinical efficacy of REGN7257 in IST-refractory/relapsed patients.

Aeglea Biotherapeutics is sponsoring a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D.

Rocket Pharmaceuticals Inc. is sponsoring a phase 1 dose escalation trial to evaluate the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with arrhythmogenic cardiomyopathy who have pathogenic PKP2 gene variants.

Genentech, Inc. is conducting a phase 3 single-arm study to evaluate the efficacy and safety of crovalimab in adult and adolescent patients with aHUS.

Genentech, Inc. is conducting a phase 3 single-arm study to evaluate the efficacy and safety of crovalimab in pediatric patients with aHUS.

Rigel Pharmaceuticals is conducting a phase 3 study to assess the efficacy of fostamatinib in patients with warm antibody AIHA. For more information, visit:

Savara, Inc. is sponsoring an interventional, randomized, double-blind, 2-arm, parallel groups, placebo-controlled, multi-center, phase 3 trial of once daily treatment with inhaled molgramostim or placebo for 48 weeks in adult patients with aPAP.

Savara, Inc. is sponsoring a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of molgramostim in adult patients with aPAP.

Beigene is sponsoring a dose-escalation and expansion study of the bruton tyrosine kinase targeted protein degrader BGB-16673 in patients with B-cell malignancies including marginal zone lymphoma, follicular lymphoma, non-Hodgkin lymphoma, Waldenström’s macroglobulinemia, chronic lymphocytic leukemia, small lymphocytic lymphoma, mantle cell lymphoma and diffuse large B-cell lymphoma.

Ono Pharmaceutical Co. Ltd is sponsoring a first-in-human clinical study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of ONO-7018 in patients with relapsed or refractory non-Hodgkin lymphoma or chronic lymphocytic leukemia.

Neurogene, Inc. is sponsoring a phase 1/2 interventional, prospective, non-randomized, first in human (FIH), open-label study of a single dose administration of gene therapy in children who are 3 to 8 years old with neuronal ceroid lipofuscinosis (Batten) subtype 5 (CLN5) disease. Efficacy assessments in this study will evaluate motor, language, visual and cognitive function and each participant will be followed for safety and efficacy for 5 years after treatment.

Neurogene, Inc. is sponsoring a study to investigate the clinical characteristics and natural clinical progression of symptoms in patients with the CLN5 and CLN7 forms of Batten disease. This natural history study is being conducted to better understand disease course and to determine clinically relevant outcome measures for use in future clinical trials.

Compass Therapeutics is sponsoring a multi-center, open-label, randomized, phase 2/3, controlled study to assess the efficacy of CTX-009 in combination with paclitaxel versus paclitaxel alone in adult patients with unresectable advanced, metastatic, or recurrent biliary tract cancers who have received one prior systemic chemotherapy regimen.

ImmunoGen, Inc. is sponsoring an open-label, multi-center, phase 1/2 study to determine the maximum tolerated dose and assess the safety, tolerability, pharmacokinetics, immunogenicity, and anti-leukemia activity of IMGN632 when administered as monotherapy to patients with CD123+ disease. The study is enrolling a pivotal cohort of frontline BPDCN patients and a cohort of relapsed/refractory BPDCN patients.

AstraZeneca is sponsoring a multinational, randomized, double-blind study to evaluate the safety and efficacy of benralizumab in treating patients with bullous pemphigoid

Regeneron Pharmaceuticals and Sanofi are conducting a study to evaluate the efficacy and safety of dupilumab in adult patients with bullous pemphigoid.

Argenx is sponsoring a randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety and efficacy of efgartigimod PH20 SC in adult patients with bullous pemphigoid.

Argenx is sponsoring a phase 3, open-label extension study to evaluate the long-term safety, tolerability and efficacy of efgartigimod PH20 SC in adult patients with bullous pemphigoid.

Achillion Pharmaceuticals is investigating the safety and efficacy of ACH-4471, an orally administered complement factor D inhibitor, in patients with C3 Glomerulopathy (C3G), including Dense Deposit Disease (DDD) and C3 Glomerulonephritis (C3GN). Two clinical studies are being conducted in multiple countries. For more information about these studies, please visit ClinicalTrials.gov: NCT03369236 and ClinicalTrials.gov: NCT03124368.

ChemoCentryx is conducting a study to evaluate the effect of avacopan treatment on renal disease activity in patients with C3G.

Aspa Therapeutics is sponsoring a study to evaluate the safety, tolerability and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric patients with Canavan disease. The estimated enrollment for this study is 18 participants.

Tenaya Therapeutics is conducting a prospective and retrospective registry and biomarker study to collect information on the natural history of pediatric patients with cardiomyopathy due to mutations in the MYBPC3 gene.

Zogenix, Inc. is sponsoring a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the safety and efficacy of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Ovid Therapeutics Inc. is sponsoring a study to investigate the effect of TAK-935 on the frequency of motor seizures in patients with CDKL5 deficiency disorder during the maintenance period.

Recursion Pharmaceuticals Inc. is sponsoring a phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, and pharmacokinetics of REC-994 compared to placebo for the treatment of patients with symptomatic CCM.

Travere Therapeutics, Inc. is sponsoring an observational, multicenter study to determine the prevalence of CTX in patients diagnosed with early-onset idiopathic bilateral cataracts.

Janssen Research & Development, LLC is sponsoring a multistage, multicenter, randomized, double-blind study to evaluate the safety and efficacy of nipocalimab in delaying relapse in adults with CIDP.

Sanofi is sponsoring a study to evaluate the efficacy of riliprubart compared to IVIg in adult patients with CIDP who are receiving maintenance treatment with IVIg.

Sanofi is sponsoring a study to evaluate the efficacy of riliprubart compared to placebo in adult patients with CIDP whose disease is refractory to standard of care

Actelion Pharmaceuticals is sponsoring a study to assess the efficacy and safety of selexipag in patients with inoperable or persistent/recurrent CTEPH.

Actelion Pharmaceuticals is sponsoring a study to evaluate the effect of macitentan 75 mg versus placebo on exercise capacity at week 28 in patients with CTEPH.

Swedish Orphan Biovitrum is sponsoring a phase 3, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of pegcetacoplan in increasing hemoglobin levels from baseline in patients with CAD in order to avoid transfusion.

Adrenas Therapeutics, Inc. is sponsoring a study to evaluate the safety, tolerability and efficacy of AAV5 based BBP-631 in adult patients with classic congenital adrenal hyperplasia.

Catalyst Pharmaceuticals, Inc. is sponsoring a randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 to 70) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.For more information visit ClinicalTrials.gov: NCT02562066

QOL Medical, LLC is sponsoring an open-label, phase 4, multi-center study to evaluate the use of a 7-day therapeutic trial dose of sarcrosidase (Sucraid) in alleviating CSID symptoms in pediatric patients with low, moderate and normal sucrase levels.

Researchers at the University of British Columbia are conducting a survey to collect data about behavior following treatment for creatine deficiency syndrome (CDS). Questions for patients and their parents or caregivers pertain to diagnostic history, presenting symptoms during life span, and response to treatments with focus on patient and caregiver reported observations.

Recordati Rare Diseases is conducting a phase II, multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing’s disease.

The University of Maryland School of Medicine is conducting a study to evaluate the process that Latine-identifying caregivers of children with cystic fibrosis have gone through to access genetic testing and/or genetic counseling services. Participation involves an anonymous online survey that will take approximately 15 minutes to complete and is available in English and Spanish.

ModernaTX, Inc. is sponsoring a study to evaluate the efficacy of mRNA 1647 vaccine in healthy CMV-seronegative female participants and the safety and reactogenicity of mRNA-1647 vaccine in all healthy participants. Estimated enrollment is 6900 participants.

ModernaTX, Inc. is sponsoring a study to evaluate the safety, reactogenicity and immunogenicity of the mRNA-1647 vaccine administered according to a 3-study injection schedule in healthy cytomegalovirus-seronegative and cytomegalovirus-seropositive Japanese adults 18 to 40 years of age in the United States. Estimated enrollment is 48 participants.

Shire is sponsoring study is to compare the efficacy and safety of maribavir to valganciclovir for the treatment of CMV infection in asymptomatic hematopoietic stem  cell transplant recipients. For more information visit:

Shire is sponsoring a study is to determine if an investigational treatment (Maribavir) is safe and effective in treating transplant recipient subjects with CMV infections that are refractory or resistant to treatment with ganciclovir, valganciclovir, foscarnet, or cidofovir. For more information visit:

Rocket Pharmaceuticals Inc. is sponsoring a non-randomized open-label phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon disease.

Rocket Pharmaceuticals Inc. is sponsoring a phase 2 gene therapy trial to evaluate the efficacy and safety of RP-A501 in male patients with Danon disease.

Rocket Pharmaceuticals Inc. is sponsoring an international observational study to learn about the natural history of Danon disease in male patients >8 years of age

Caliway Biopharmaceuticals Co., Ltd. is sponsoring a study to evaluate the efficacy and safety of CBL-514 compared to placebo in patients with Dercum’s disease lipomas.

SpringWorks Therapeutics, Inc. is offering an individual patient compassionate use program for nirogacestat for patients with desmoid tumor or NOTCH gene mutation positive tumors.

LEO Pharma is sponsoring a double-blind, multi-center, randomised, vehicle-controlled, within-subject trial designed to establish the efficacy and safety of delgocitinib cream in the treatment of adults with DLE.

Researchers at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at the NIH are conducting two studies of people who have reproductive disorders. The studies will look into how genes and production of gonadotropin-releasing hormone affect puberty and infertility. Individuals 14 years of age and older with low testosterone or estrogen may be eligible. For more information visit

Ovid Therapeutics Inc. is sponsoring a study is to investigate the effect on the frequency of all seizures (convulsive and drop) in patients with Dravet syndrome treated with TAK-935 compared to placebo.

AstraZeneca Pharmaceuticals is sponsoring a phase 3 study is to investigate the use of benralizumab as a treatment for patients with EoE.

AstraZeneca is sponsoring a phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of tezepelumab in adult and adolescent patients with EoE.

AstraZeneca Pharmaceuticals is sponsoring a randomized, double blind, active-controlled, parallel group, multicenter 52-week phase 3 study to compare the efficacy and safety of benralizumab 30 mg versus mepolizumab 300 mg administered by subcutaneous injection in patients with relapsing or refractory EGPA on corticosteroid therapy with or without stable immunosuppressive therapy.

GlaxoSmithKline is sponsoring a study to investigate the efficacy and safety of depemokimab compared with mepolizumab in adults with relapsing or refractory EGPA.

Atara Biotherapeutics is sponsoring a study to evaluate tabelecleucel in patients with EBV-associated diseases.

Viracta Therapeutics, Inc. is sponsoring an open-label, multicenter phase 1b/2 study to evaluate the efficacy of nanatinostat in combination with valganciclovir in patients with relapsed/refractory EBV-associated lymphomas.

Viracta Therapeutics, Inc. is sponsoring an open-label, multicenter phase 1b/2 study to evaluate the safety and efficacy of nanatinostat in combination with valganciclovir in patients with relapsed/refractory EBV-associated solid tumors and in combination with pembrolizumab in patients with recurrent/metastatic nasopharyngeal carcinoma.