Study Summary
This trial is testing whether the 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.
Eligible Conditions
- Alopecia Areata
- Autoimmune Polyendocrinopathy-Candidiasis-Ectodermal Dystrophy (APECED)
Treatment Effectiveness
Effectiveness Progress
Study Objectives
1 Primary · 34 Secondary · Reporting Duration: Week 32
32 weeks
Response defined as a 30% improvement from baseline in the Severity of Alopecia Tool (SALT) score at 32 weeks.
End of Study
1. Incidence of SAEs, AEs requiring study drug discontinuation, and other AEs.
Incidence of SAEs, AEs requiring study drug discontinuation, and other AEs.
Variable
5. Time for participants to achieve SALT score </=20.
6. Time for participants to achieve SALT30 and SALT50.
Time for participants to achieve SALT30 and SALT50.
Week 16
2. Achievement of SALT30 at week 16.
Achievement of SALT30 at week 16.
Week 32
10. Improvement of CDLQI score (pediatrics) from baseline at week 32.
11. Improvement of FDLQI score (family members or household contacts) from baseline at week 32.
12. Improvement of SF-36 score (adults) from baseline at week 32.
13. Improvement of PedsQL score (pediatrics) from baseline at week 32.
14. PRO for Scalp Hair Assessment Score of 0 or 1 with a =2-point improvement from baseline among participants with a score of =3 at baseline at week 32.
16. Achievement of ClinRO measure for EB hair loss 0 or 1 with =2-point improvement from baseline (among participants with ClinRO measure for EB hair loss =2 at baseline) at week 32.
17. Achievement of ClinRO measure for EL hair loss 0 or 1 with =2-point improvement from baseline (among participants with ClinRO measure for EL hair loss =2 at baseline) at week 32.
18. Change from baseline in Skindex-16 AA symptoms, emotions, and functioning domain scores at week 32.
19. Change from baseline in histologic and immunologic abnormalities of scalp tissue at week 32.
3. Achievement of SALT50 at week 32.
4. Achievement of SALT score </=20 at week 32.
7. Percentage of hair regrowth at week 32.
8. Improvement of AASIS score from baseline at week 32.
9. Improvement of DLQI score (adults) from baseline at week 32.
Achievement of ClinRO measure for EL hair loss 0 or 1 with =2-point improvement from baseline (among participants with ClinRO measure for EL hair loss =2 at baseline) at week 32.
Achievement of SALT50 at week 32.
Change from baseline in Skindex-16 AA symptoms, emotions, and functioning domain scores at week 32.
Change from baseline in histologic and immunologic abnormalities of scalp tissue at week 32.
Improvement of AA-IGA measure from baseline at week 32.
Improvement of AASIS score from baseline at week 32.
Improvement of CDLQI score (pediatrics) from baseline at week 32.
Improvement of DLQI score (adults) from baseline at week 32.
Improvement of FDLQI score (family members or household contacts) from baseline at week 32.
Improvement of PedsQL score (pediatrics) from baseline at week 32.
Improvement of SF-36 score (adults) from baseline at week 32.
PRO for Scalp Hair Assessment Score of 0 or 1 with a =2-point improvement from baseline among participants with a score of =3 at baseline at week 32.
Percentage of hair regrowth at week 32.
Trial Safety
Safety Progress
This is further along than 68% of similar trials
Side Effects for
Best Available Therapy
23%Pruritus
12%Headache
9%Diarrhoea
9%Upper respiratory tract infection
8%Thrombocytopenia
8%Asthenia
8%Fatigue
7%Dizziness
7%Nausea
7%Night sweats
7%Haematocrit increased
5%Leukocytosis
5%Constipation
5%Influenza
5%Decreased appetite
5%Erythema
5%Weight decreased
4%Arthralgia
4%Hypertension
4%Abdominal pain upper
4%Thrombocytosis
3%Tinnitus
3%Abdominal discomfort
3%Dyspepsia
3%Oedema peripheral
3%Nasopharyngitis
3%Myalgia
3%Pain in extremity
3%Cough
3%Dyspnoea
3%Epistaxis
3%Bronchitis
1%Anaemia
1%Atrial fibrillation
1%Gastrointestinal haemorrhage
1%Rectal haemorrhage
1%Cellulitis
1%Hyponatraemia
1%Bladder transitional cell carcinoma
1%Syncope
1%Extremity necrosis
1%Abdominal pain
1%Pyrexia
1%Flatulence
1%Blood lactate dehydrogenase increased
1%Gamma-glutamyltransferase increased
1%Musculoskeletal pain
1%Hyperuricaemia
1%Osteoarthritis
1%Haematoma
1%Pneumonia
1%Septic shock
1%Neutropenia
1%Meningitis
1%Vertigo
1%Depression
1%Myelofibrosis
1%Respiratory failure
1%Abdominal distension
1%Vomiting
1%Weight increased
1%Muscle spasms
1%Acute myeloid leukaemia
1%Breast cancer
1%Cardiac failure
1%Renal failure
Trial Design
1 Treatment Group
Ruxolitinib/APECED-AA Patients
1 of 1
Experimental Treatment
70 Total Participants · 1 Treatment Group
Primary Treatment: Ruxolitinib · No Placebo Group · Phase 2
Ruxolitinib/APECED-AA Patients
Drug
Experimental Group · 1 Intervention: Ruxolitinib · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ruxolitinib
2018
Completed Phase 3
~910
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week 32
Who is running the clinical trial?
National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,165 Previous Clinical Trials
4,936,951 Total Patients Enrolled
Michail S Lionakis, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
4 Previous Clinical Trials
1,288 Total Patients Enrolled
Eligibility Criteria
Age 12 - 65 · All Participants · 17 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You have not received or have not responded to other treatments for AA.
You have not taken any medication for hair loss in the two months before starting the study.
You are willing to take valacyclovir to prevent the reactivation of herpes virus.
You cannot currently have any hair growth.
You have been experiencing hair loss for more than 6 months.
You have an intrauterine device or hormone-releasing system in place.
Who else is applying?
What state do they live in?
| Virginia | 100.0% |
How old are they?
| < 18 | 100.0% |
What site did they apply to?
| National Institutes of Health Clinical Center | 100.0% |
What portion of applicants met pre-screening criteria?
| Met criteria | 100.0% |
How responsive is this trial?
Typically responds via
| 100.0% | |
Most responsive sites:
- National Institutes of Health Clinical Center: < 48 hours
Average response time
- < 2 Days
Frequently Asked Questions
Is it possible for me to participate in this experiment?
"This trial seeks 70 participants suffering from alopecia areata, between the ages of 12 and 65 to be included." - Anonymous Online Contributor
Unverified Answer
Is enrollment for this research endeavor currently available?
"Data on clinicaltrials.gov verifies that this trial, first posted on December 1st 2022, is actively seeking participants. The information concerning the experiment was recently amended on November 25th of the same year." - Anonymous Online Contributor
Unverified Answer
Is Ruxolitinib a viable therapeutic option with minimal risk?
"Due to its Phase 2 status, there is some evidence of Ruxolitinib's safety but no data attesting to the efficacy. Consequently, a score of two was assigned on our team at Power's scale from 1-3." - Anonymous Online Contributor
Unverified Answer
Does this trial permit individuals under the age of fifty to participate?
"The required age range for this particular experiment is between 12 and 65 years old. Meanwhile, there are 10 trials aimed at minors and 79 focusing on individuals over the retirement age." - Anonymous Online Contributor
Unverified Answer
What is the maximum number of participants this clinical trial can accommodate?
"Affirmative. The clinicaltrials.gov website confirms that this medical trial, which was first published on December 1st 2022, is currently recruiting patients from one location and needs to find 70 participants in total." - Anonymous Online Contributor
Unverified Answer