Find Clinical Trials & Research Studies

NORD shares information about opportunities to participate in clinical trials and other studies so that patients and their physicians may decide whether specific studies are appropriate for them. We do not endorse or recommend participation in any specific clinical trials or studies. The following is a brief sampling of current clinical trials listed at www.clinicaltrials.gov.


Current Clinical Trials & Studies

Thyroid Eye Disease (TED)

Viridian Therapeutics, Inc. is sponsoring a randomized, parallel assignment, multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of VRDN-001 in normal healthy adults and adult patients with TED.

More

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.

More

Viridian Therapeutics, Inc. is sponsoring a phase 3, randomized, double-masked, placebo-controlled study to evaluate the safety, tolerability and efficacy of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in patients with chronic TED.

More
Transverse Myelitis (including Acute Flaccid Myelitis)

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.

More
Trichotillomania

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.

ClinicalTrials.gov:NCT03797521
von Willebrand Disease (VWD)

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.

ClinicalTrials.gov - NCT02932618
Waldenström’s Macroglobulinemia

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.

 

More

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.

More
Warm Autoimmune Hemolytic Anemia (wAIHA)

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.

More

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.

More
WHIM Syndrome

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.

More
X-Linked Retinoschisis (XLRS)

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.

More