Find Clinical Trials & Research Studies

Astellas Pharma Inc. is sponsoring a study to assess the safety and tolerability of ASP0367 in patients with primary mitochondrial myopathy. The effect of ASP0367 on functional improvement and fatigue will also be evaluated. Estimated enrollment is 149 participants.

Reneo Pharma Ltd is sponsoring a phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of a 24-week treatment with REN001 in patients with primary mitochondrial myopathy.

Stealth Biotherapeutics is recruiting for MMPOWER-3, a multi-center, phase 3, randomized, double-blind, placebo-controlled trial in patients with genetically confirmed and clinically diagnosed primary mitochondrial myopathy (PMM). The objectives of the study are to evaluate the effects of elamipretide therapy on functional assessments (6-Minute Walk Test), patient and clinician reported questionnaires, and safety in patients with PMM. 

Atara Biotherapeutics is sponsoring a study to evaluate the safety and tolerability of ATA188 as a monotherapy in parts 1 and 2, to determine the recommended part 2 dose of ATA188 as monotherapy in part 1, and to evaluate the effect of ATA188 treatment on biological markers of disease activity in cerebral spinal fluid in part 2 in patients with primary progressive multiple sclerosis and secondary progressive multiple sclerosis.

ModernaTX, Inc. is sponsoring a global, phase 1/2, open-label, dose optimization study to evaluate the safety, pharmacodynamics and pharmacokinetics of mRNA-3927 in patients with propionic acidemia.

Arkansas Children’s Hospital Urology Division is conducting a study to help better understand the long term surgical and medical outcomes of females with prune bell syndrome, also known as Eagle-Barrett syndrome. Female patients and caregivers of female patients are eligible to complete this survey.

Galderma is sponsoring a study to assess the efficacy and safety of the investigational drug nemolizumab in patients with prurigo nodularis (PN). The primary objective of this research study is to evaluate the efficacy of nemolizumab on clearance of PN lesions and on itch reduction. Additional objectives are to demonstrate the efficacy of nemolizumab on sleep disturbance and improvement of health-related quality of life in PN patients.

Incyte Corporation is sponsoring a study to evaluate the safety and efficacy of INCB054707 in patients with prurigo nodularis over a 16-week double-blind placebo-controlled treatment period, followed by a 24-week single blind extension period. Estimated enrollment is 140 participants.

Kiniksa Pharmaceuticals, Ltd. is sponsoring a phase 2a/b randomized, double-blind, placebo-controlled study to investigate the efficacy, safety, tolerability, pharmacokinetics and immunogenicity of vixarelimab administered subcutaneously in patients with prurigo nodularis experiencing pruritus (itching).

Regeneron Pharmaceuticals is sponsoring a study to demonstrate the efficacy of dupilumab on itch response in patients with prurigo nodularis (PN) who are inadequately controlled on topical prescription therapy or when those therapies are not advisable. Additional objectives are to demonstrate the efficacy of dupilumab on additional itch endpoints in patients with PN who are inadequately controlled on topical prescription therapy or when those therapies are not advisable; demonstrate efficacy of dupilumab on skin lesions of PN; demonstrate improvement in health-related quality of life; evaluate safety outcome measures; and evaluate immunogenicity of dupilumab.

Janssen Pharmaceuticals is sponsoring a study to determine if a 75 mg dose of macitentan is superior to a 10 mg dose in prolonging the time to the first clinical events committee -adjudicated morbidity or mortality event in patients with symptomatic PAH.

OrphAI Therapeutics Is sponsoring a phase 2a, single-arm, open-label, exploratory study to evaluate the safety and efficacy of LAM-001 as an add-on therapy for the treatment of patients with pulmonary hypertension.

Boehringer Ingelheim is sponsoring a multi-center, randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of spesolimab in treating adult patients living with ulcerative Pyoderma Gangrenosum (PG).

The University of Florida is sponsoring a phase 3 trial in collaboration with Columbia University, Medosome Biotec LLC, and Saol Therapeutics, of the investigational drug dichloroacetate to treat young children with PDCD.

Agios Pharmaceuticals, Inc. is conducting a study to evaluate the efficacy and safety of orally administered AG-348 as compared with placebo in participants with PKD who are not regularly receiving blood transfusions.

Agios Pharmaceuticals, Inc. is conducting a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40 patients with PKD who are regularly receiving blood transfusions.

The National Cancer Institute is conducting a study to determine if the molecule M7824 is effective for the treatment of recurrent respiratory papillomatosis.

Blueprint Medicines Corporation is sponsoring a phase 1and 2, open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antineoplastic activity of pralsetinib (BLU-667) administered orally in patients with medullary thyroid cancer, RET-altered non-small cell lung cancer and other RET-altered solid tumors.

ACADIA Pharmaceuticals Inc. is sponsoring a study to investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome.

ACADIA Pharmaceuticals Inc. is sponsoring a study to investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome.

The Worldwide Sarcoidosis Research Study (WISE) is designed to gain more information about the characteristics and clinical course of sarcoidosis in order to help physicians and researchers better understand how to treat the disease. Researchers at the University of Iowa Institute for Clinical and Translational Science will be gathering information from a large number of sarcoidosis patients located all around the world using a web-based survey.

VectivBio AG is sponsoring an international, multicenter, double-blind, randomized, placebo-controlled trial to evaluate the safety and efficacy of apraglutide as a treatment for adult patients with SBS-IF.

Seton Hall University is conducting a study to help understand how a mobile health technology app can assist adolescents and young adults with SCD with their transition to adult care and help them manage the disease as adults. Individuals with SCD age 18-30 are invited to participate in a short anonymous survey:

Genentech, Inc. is conducting a phase 1b randomized study to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of crovalimab for the management of acute uncomplicated vaso-occlusive episodes (VOE) in patients with SCD.

Genentech, Inc. is conducting a phase 2a randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of crovalimab as adjunct treatment in prevention of vaso-occlusive episodes (VOE) in SCD.

Biogen, Inc. is sponsoring a study to examine the clinical efficacy, safety and tolerability of nusinersen administered intrathecally at higher doses to patients with SMA. More.

Biogen, Inc. is sponsoring a study to evaluate the clinical outcomes, safety and tolerability following treatment with nusinersen in patients with SMA who have previously received onasemnogene abeparvovec.

Brandeis University is conducting a study to explore the experiences of people with SMA regarding transition planning and transfer of care between pediatric and adult care settings. Participation involves a 15-minute online survey that asks about patient autonomy in different care settings, access to non-SMA-related care, and barriers to transfers of care. Participants will be entered in a raffle for the chance to win one of four $50 Amazon gift cards.

Novartis Pharmaceuticals is sponsoring a study to evaluate the safety, tolerability and efficacy of intrathecal OAV101 as a treatment for patients with type 2 SMA who are between the ages of 2 and 18, can sit but are not ambulatory and have not had any SMN-targeting therapies.

Biohaven Pharmaceuticals is conducting a 48-week phase 3 clinical study to determine if troriluzole can slow down and improve symptoms in subjects with hereditary spinocerebellar ataxia. (SCA). The study will focus mainly on disease-types SCA1 and SCA2.

Novartis Pharmaceuticals is sponsoring an open-label, phase 2 study designed to evaluate the safety, efficacy, and tolerability of MAS825 in pediatric and adult patients living with Still’s disease.

Sobi is conducting a Phase III clinical trial to assess the efficacy and to evaluate the safety, pharmacokinetics (PK) and immunogenicity of anakinra in patients with newly diagnosed Still’s disease, including SJIA (Systemic juvenile idiopathic arthritis) and AOSD (Adult-onset Still’s disease).

argenx is conducting a randomized, double-blinded, placebo-controlled, phase 2, parallel-group study to evaluate the safety and efficacy of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months.

Cabaletta Bio is conducting a phase 1/2 clinical trial (RESET-SSc^TM) to evaluate the effects of the investigational cell treatment CABA-201 for the treatment of systemic sclerosis (SSc)

Genentech, Inc. is sponsoring a double-blind, randomized, placebo-controlled, multicenter study to evaluate the safety, efficacy, tolerability and pharmacokinetics of multiple-ascending doses of RO7303509 in patients with systemic sclerosis.

Novartis Pharmaceuticals is sponsoring a phase 2, multi-part, five year, open-label, multicenter study is to evaluate the safety, efficacy, and tolerability of rapcabtagene autoleucel in treating patients with severe refractory diffuse cutaneous systemic sclerosis (DcSSc).

Novartis Pharmaceuticals is sponsoring a randomized, double-blind, parallel group, placebo-controlled, multicenter study is to evaluate safety, efficacy, and tolerability of ianalumab in treating patients with diffuse cutaneous systemic sclerosis.

Deciphera Pharmaceuticals LLC is sponsoring a multicenter, randomized, placebo-controlled, double-blind, phase 3 clinical study to evaluate the safety and efficacy of an investigational drug, vimseltinib for the treatment of TGCT in patients where surgical removal is not an option.

Deciphera Pharmaceuticals LLC is conducting a multicenter, open-label phase 1/2 study of DCC-3014 in patients with malignant solid tumors and TGCT. Phase 1 (dose escalation) will enroll both malignant solid tumor and TGCT patients. Phase 2 (expansion) will comprise two cohorts and will only enroll TGCT patients.

Modis Therapeutics, Inc. and Zogenix, Inc. are sponsoring a study to evaluate the efficacy and safety of MT1621 in patients with genetic confirmation of TK2d who have never received MT1621 or nucleos(t)ides before entering the study.

Sling Therapeutics, Inc. is sponsoring phase 2b, randomized, double-mask, placebo-controlled study to evaluate the safety, efficacy, and pharmacokinetics of linsitinib in patients with active, moderate to severe TED.

Viridian Therapeutics, Inc. is sponsoring a clinical trial assessing the safety and tolerability of an investigational drug, VRDN-003, in participants with Thyroid Eye Disease (TED).

Viridian Therapeutics, Inc. is sponsoring a clinical trial assessing the safety, tolerability and pharmacokinetics (PK) of an investigational drug, VRDN-003, in participants with TED (Thyroid Eye Disease).

The University of Texas Southwestern Medical Center is conducting a study to assess the current state of pediatric transverse myelitis (including AFM or acute flaccid myelitis) in terms of diagnosis, treatment and outcomes.

Promentis Pharmaceuticals, Inc. is conducting study is to explore the safety, tolerability and activity of SXC-2023 when dosed for 6 weeks versus placebo in adult patients with moderate to severe trichotillomania.

Rezolute, Inc. is conducting a phase 3, single-arm, open-label, pivotal study to evaluate the safety and efficacy of ersodetug compared to baseline in patients living with inadequately controlled Hypoglycemia due to Tumor Hyperinsulinism (Tumor HI).

Rezolute, Inc. is sponsoring an expanded access program to provide ersodetug, an investigational drug, to eligible participants who suffer from inadequately controlled hypoglycemia due to tumor-associated hyperinsulinism and are unable to participate in the ersodetug clinical trial. Participation is open to participants who meet the eligibility criteria and for whom access to the investigational drug is deemed appropriate by the treating physician.

Acelyrin Inc. is sponsoring a study to evaluate the efficacy and safety of izokibep in patients with non-infectious, intermediate-, posterior- or pan-uveitis.

Amgen is sponsoring a phase 3 clinical trial evaluating the safety, efficacy, and pharmacokinetics of avacopan in children aged 6 to under 18 with ANCA-associated vasculitis (AAV). Patients will receive avacopan in combination with rituximab or a cyclophosphamide-containing regimen over 52 weeks.

Amgen is sponsoring a phase 4 clinical trial to evaluate the safety and efficacy of avacopan in adult patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV).

Novartis Pharmaceuticals is sponsoring a phase 2, randomized study to evaluate the safety and efficacy of rapcabtagene autoleucel in treating patients with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA).

A study is enrolling children (< 18 years old) diagnosed with severe von Willebrand disease (VWD). This study will evaluate the use of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of bleeding episodes.

Beigene is sponsoring a study to evaluate the safety and efficacy of the BCL2 inhibitor BGB-11417 in patients with relapsed/refractory Waldenström’s macroglobulinemia.

Cellectar Biosciences, Inc. is sponsoring a two-part study in patients with Waldenstrom macroglobulinemia (WM). Part A evaluates CLR 131 in patients with select B-cell malignancies, including WM, who have been previously treated with standard therapy. Part B evaluates IV administration of CLR 131 in patients with WM who have received at least two prior lines of therapy. The estimated enrollment for this study is 120 participants.

X4 Pharmaceuticals is sponsoring a study is to establish a pharmacologically active dose of mavorixafor in combination with ibrutinib based on pooled safety, clinical response, pharmacokinetic and pharmacodynamic data to select the recommended dose for a randomized registrations trial. Patients with Waldenstrom’s macroglobulinemia whose tumors express mutations in MYD88 and CXCR4 genes are eligible.

Incyte Corporation is sponsoring a study to evaluate the safety and efficacy of parsclisib compared with placebo in patients with primary wAIHA. Estimated enrollment is 100 participants.

Janssen Research & Development, LLC is sponsoring a study to evaluate the efficacy and safety of M281 in patients with warm autoimmune hemolytic anemia. Estimated enrollment is 111 participants.

Novartis Pharmaceuticals is sponsoring a study to evaluate the efficacy and safety of ianalumab compared to placebo in patients with wAIHA who had at least one unsuccessful line of treatment.

X4 Pharmaceuticals is sponsoring  a double-blind, placebo-controlled study with a randomized period and an open-label extension period. The primary objective of the randomized period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with placebo, and relative to a clinically meaningful threshold. The primary objective of the open-label period is to evaluate the safety and tolerability of mavorixafor in participants with WHIM syndrome.

AGTC is currently conducting a clinical study to evaluate the safety and efficacy of an investigational gene therapy in patients with XLRS. 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.