Netherton Syndrome

 

Below is a list of our Netherton syndrome studies that are currently enrolling.

A MULTICENTER, RANDOMIZED, VEHICLE-CONTROLLED, DOUBLE-BLIND, PARALLEL COMPARISON, PROOF-OF-CONCEPT STUDY OF QRX003 LOTION IN SUBJECTS WITH NETHERTON SYNDROME

• Population: Patients aged eighteen years or older
• Study Condition: Netherton Syndrome
• Study Design: The purpose of this study is to assess the dose dependent safety, tolerability and potential efficacy of QRX003 lotion in subjects with Netherton Syndrome (NS). This is a multi-center, randomized, vehicle-controlled, double-blind, parallel group comparison study of QRX003 lotion in adult subjects 18 years of age or older with NS. Approximately 18 subjects will be enrolled at approximately 7 sites. Subjects will be randomized (1:1:1) to treatment as follows:
  • 1. QRX003 (dipalmitoyl hydroxyproline) lotion, 2% (Low dose)
  • 2. QRX003 (dipalmitoyl hydroxyproline) lotion, 4% (High dose)
  • 3. Vehicle lotion

  Subjects will apply the assigned test article once daily in the morning to a designated Treatment Area for 12 weeks. Subjects will attend 5 clinic visits for up to 20 weeks.

• Compensation will be provided

 

A study to test whether spesolimab helps people with skin disease called Netherton syndrome

• Population: Patients aged eighteen years and older

• Study Condition: Netherton syndrome (causative SPINK5 mutations)

• Study Design: This is a multi-center, randomized, double-blind, placebo-controlled, phase II/III study to evaluate the efficacy and safety of spesolimab compared to placebo in the treatment of Netherton syndrome. This study will last up to 72 weeks with 16 clinic visits. Eligible patients will be randomized 2:1 to receive either spesolimab i.v. loading doses at Week 0 plus spesolimab subcutaneous doses every 4 weeks, or to receive placebo i.v. loading dose at Week 0 plus placebo subcutaneous doses every 4 weeks. At Week 20, all patients will enter the open label period to receive spesolimab subcutaneous dose every 4 weeks up to Week 52. There will be a safety follow up visit 16 weeks after the last dose.
• Compensation will be provided for time and travel.

 

Please contact Dr. Veronica Emmerich for more information regarding ongoing Netherton syndrome studies. 

 

Email: emmerich@musc.edu

Phone: (843) 876-0110