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Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies
The purpose of this study is to identify genes associated with impaired development and function of the cranial nerves and brainstem, which may result in misal…
A Phase 2 Open-label Study to Evaluate Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Primary or Secondary Myelofibrosis
The purpose of this Phase 2 study is to evaluate the efficacy and safety of momelotinib (MMB) in combination with luspatercept (LUSPA) in participants with tra…
Idiopathic Pulmonary Fibrosis, a Disease Initiated by Mucociliary Dysfunction
This study seeks to screen first degree family members of people with Idiopathic Pulmonary Fibrosis (IPF) for the earliest signs of lung fibrosis.
PRIME-PPF: PRospective phenotypIng and Multi-omic Endotyping of Progressive Pulmonary Fibrosis
This is a prospective, observational cohort study. Participants with non-idiopatic pulmonary fibrosis, interstitial lung disease (ILD) will be followed for 24 …
A Prospective Multicenter International Randomized Controlled Trial Comparing Surgical and Medical Therapies in the Treatment of Advanced Metabolic Dysfunction Associated Steatohepatitis
Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health con…
A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects With Chronic Pseudomonas Aeruginosa (PsA) Pulmonary Infection
The goal of this Phase 2b clinical trial is to see if nebulized phage (BX004) can treat chronic Pseudomonas aeruginosa (PsA) lung infection in CF subjects. The…
A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
The purpose of this clinical trial is to compare drug combinations to learn which drugs work best to prevent graft-versus-host-disease (GVHD) in people who hav…
MC220806: Phase I Study Evaluating the Efficacy of CSF1R and TAM Receptor or Inhibition in Hematologic Malignancies With Q702, a Small Molecular Inhibitor
This phase I trial tests the safety, side effects, and best dose of Q702 in treating patients with hematologic malignancies. Q702 is in a class of medications …
A Phase I Study to Evaluate the Safety and Maximum Tolerated Dose of Momelotinib Durind and Following Hematopoietic Cell Transplantation for Patients With Myelofibrosis
This is a single-center, open-label, phase I study to determine the safety and tolerability of momelotinib in patients with myelofibrosis during and after hema…
Regional Phenotyping of Cystic Fibrosis Lung Disease and Non-CF Bronchiectasis
The Investigators propose to study pediatric subjects who are diagnosed with cystic fibrosis (CF) and patients with non-CF bronchiectasis, with the goal of dev…
A Single Ascending Dose, Phase 1/2 Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SP-101 Via Nebulizer for the Treatment of Cystic Fibrosis (CF)
This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to…
A Phase 2 Study to Evaluate the Efficacy and Safety of Selinexor Monotherapy in Subjects With JAK Inhibitor-naïve Myelofibrosis and Moderate Thrombocytopenia
The main purpose of this study is to evaluate the efficacy of selinexor in JAKi-naïve participants with myelofibrosis (MF) and with normal platelet counts or w…