Find Clinical Trials & Research Studies

Neurocrine Biosciences is sponsoring a phase 2 study to assess the efficacy, safety and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in patients with SCN8A-DEE. Estimated enrollment is 52 participants.

X4 Pharmaceuticals is sponsoring a phase 1b/2, open-label, multicenter study to evaluate the safety, tolerability, and efficacy of mavorixafor in treating patients with congenital neutropenia and chronic neutropenia disorders.

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.

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).

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.

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 randomized, active controlled, safety and tolerability study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in patients with TED.

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.

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

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.