NIH LAUNCHES FIRST EVER THERAPEUTIC CLINICAL TRIAL IN APS TYPE 1

Dr. Lionakis’ Lab and his team of dedicated healthcare providers at the National Institutes of Health in Bethesda, Maryland, have collaborated with the APS Type 1 community since 2012 with the goals of characterizing disease progression and developing new therapeutic approaches to improving the lives of persons with APS Type 1/APECED. Building on their research into the molecular causes of organ damage in APS Type 1/APECED, they are pleased to announce a Phase 2 Trial entitled “Evaluating the efficacy and safety of Ruxolitinib on hair regrowth in patients with APECED-associated alopecia areata.” Alopecia Areata (AA) is a type of hair loss that can be associated with APS Type 1/APECED due to immunological dysfunction. This trial will be the first step to assess if Ruxolitinib can effectively treat AA in patients with APS Type 1/APECED and several other organ-specific autoimmune manifestations (e.g., gastritis, enteropathy, Sjogren’s syndrome and candidiasis) in affected patients. This will be the first ever formal therapeutic clinical trial in APS Type 1/APECED. 

Objective: 

To see if a study drug (Ruxolitinib) can help hair regrowth in people with APECED-associated AA and if it can improve other symptoms caused by the immune system’s attack to the body. The study seeks to establish improvement in alopecia in persons with greater than 50% hair loss that has been persistent for at least 6 months (termed “severe AA”). 

Eligibility

People aged 12 to 65 years with APECED and severe AA. 

Design

Participants will be in this study for up to 10 months. They will have 5 in-person visits and 6 tele-visits, each about 4 weeks apart. One in-person visit may be up to a 10-day stay in the hospital.  The first in-person visit will include screening. Participants will have a physical exam and additional blood tests. Photographs may be taken of their skin. They will answer questions about their quality of life.  Participants will begin taking the study drug during their hospital stay. They will take the pills by mouth twice a day for 8 months with continued monitoring of efficacy and potential side effects and slow increase in the drug dosing dependent on tolerability and efficacy seen.  Enrollment is scheduled to start in January 2023. 

ADDITIONAL INFORMATION 

Name of Protocol  

Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)- Associated Alopecia Areata

Sponsor 

National Institute of Allergy and Infectious Diseases (NIAID) 

Primary Investigator 

Michail S. Lionakis, M.D. 

Protocol Details 

Click here for more details.

Clinical Trials Number 

NCT05398809  

ENROLLMENT & ELIGIBILITY 

For enrollment and eligibility information, please contact: 

Tom DiMaggio, R.N., Senior Research Nurse 
Specialist Study Coordinator NIH/NIAID/LA 
10 Center Drive, Room 11C-207 
Bethesda, MD 20892 
E-mail: thomas.dimaggio@nih.gov  
Phone: 301-443-8341 
Fax: 301-480-3783 

Dr. Joseph Pechacek, MD MS 

Email: joseph.pechacek@nih.gov 

Phone: (301) 761-6858

Taura Webb, CRNP  

Email: taura.webb@nih.gov 
Phone: (301) 496-8985 

Michail S Lionakis, M.D. 
Email: lionakism@mail.nih.gov          
Phone: (301) 443-5089        

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