NCT02553330

A Study With Ruxolitinib Phosphate Cream Applied Topically to Subjects With Alopecia Areata (AA)

Terminated Phase 2 Results posted Last updated 16 December 2020

What this trial tests

Phase 2 trial testing Placebo Cream in Alopecia Areata in 90 participants. Terminated before completion.

Timeline

18 November 2015

Primary endpoint
3 October 2017

3 October 2017

Quick facts

Lead sponsor	Incyte Corporation
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	90
Start date	18 November 2015
Primary completion	3 October 2017
Estimated completion	3 October 2017
Sites	18 locations across United States

Drugs / interventions tested

• Placebo Cream
• Ruxolitinib Phosphate Cream

Conditions studied

• Alopecia Areata — all drugs for Alopecia Areata →

Sponsor

Incyte Corporation — full company profile →

Who can join

Adults 18 to 70, any sex, with Alopecia Areata. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part A: Percentage of Participants Achieving a ≥ 50% Improvement in SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). Primary · Up to Week 24

Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.

Week 4

Group	Value	95% CI
Part A: Ruxolitinib Cream	0

Week 8

Group	Value	95% CI
Part A: Ruxolitinib Cream	0

Week 12

Group	Value	95% CI
Part A: Ruxolitinib Cream	3

Week 18

Group	Value	95% CI
Part A: Ruxolitinib Cream	4

Week 24

Group	Value	95% CI
Part A: Ruxolitinib Cream	6

Part B : Percentage of Participants Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented) Primary · Week 24

Percentage of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss.

Group	Value	95% CI
Part B : Placebo Cream	5
Part B: Ruxolitinib Cream	5

Part A : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair. Secondary · Up to Week 24

Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

Week 4

Group	Value	95% CI
Part A: Ruxolitinib Cream	0

Week 8

Group	Value	95% CI
Part A: Ruxolitinib Cream	0

Week 12

Group	Value	95% CI
Part A: Ruxolitinib Cream	2

Week 18

Group	Value	95% CI
Part A: Ruxolitinib Cream	3

Week 24

Group	Value	95% CI
Part A: Ruxolitinib Cream	4

Part B : Percentage of Participants With 50% to 100% Scalp Involvement at Baseline Achieving a SALT50 Response in Terminal Hair (Pigmented and Nonpigmented). Secondary · Week 24

Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

Group	Value	95% CI
Part B : Placebo Cream	2
Part B : Ruxolitinib Cream	4

Part A and B : Percentage of Participants Achieving a SALT90 Response in Terminal Hair. Secondary · Weeks 4, 8, 12, 18, and 24.

Number of participants achieving 90% or greater improvement in their SALT score (SALT90) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes.

Week4

Group	Value	95% CI
Part A: Ruxolitinib Cream	0
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	0

Week 8

Group	Value	95% CI
Part A: Ruxolitinib Cream	0
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	0

Week 12

Group	Value	95% CI
Part A: Ruxolitinib Cream	0
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	1

Week 18

Group	Value	95% CI
Part A: Ruxolitinib Cream	1
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	2

Week 24

Group	Value	95% CI
Part A: Ruxolitinib Cream	2
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	2

Part B : Percentage of Subjects Whose AA Lesions Treated Since Baseline Achieved a Physician's Global Assessment of Regrowth (PGARG) Score of at Least 3 Secondary · Baseline to Week 24

Number of subjects achieving a Physician's Global Assessment (PGA) score of 3 or above at week 24 (0, no regrowth; 1, \<25% of regrowth; 2, 25%-49% of regrowth; 3, 50%-74% of regrowth; 4, 75%-99% of re- growth; 5, 100% of regrowth).

Group	Value	95% CI
Part B : Placebo Cream	5
Part B : Ruxolitinib Cream	2

Part B : Percentage of Participants Achieving a SALT50 in Terminal Hair (Pigmented and Nonpigmented). Secondary · Weeks 4, 8, 12, and 18.

Number of participants achieving 50% or greater improvement in their SALT score (SALT50) compared to Baseline. SALT score 0-100 with lower score indicating better health outcomes

Week 4

Group	Value	95% CI
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	0

Week 8

Group	Value	95% CI
Part B : Placebo Cream	0
Part B : Ruxolitinib Cream	2

Week 12

Group	Value	95% CI
Part B : Placebo Cream	1
Part B : Ruxolitinib Cream	3

Week 18

Group	Value	95% CI
Part B : Placebo Cream	4
Part B : Ruxolitinib Cream	5

Part B: Mean Change From Baseline in SALT Score Secondary · Weeks 4, 8, 12, 18, and 24.

Severity of Alopecia Tool Score (SALT) calculation is based on a scoring system. The scalp is divided into the following 4 areas: 1) Vertex: 40% (0.4) of scalp surface area, 2) Right profile of scalp: 18% (0.18) of scalp surface area, 3) Left profile of scalp: 18% (0.18) of scalp surface area, and 4) Posterior aspect of scalp: 24% (0.24) of scalp surface area. The percentage of hair loss in any of these areas is the percentage hair loss multiplied by percent surface area of the scalp in that area. The SALT score is the sum of percentage of hair loss in all the above-mentioned areas, so a lower

Change from Baseline to Week 4

Group	Value	95% CI
Part B : Placebo Cream	1.02	± 1.577
Part B : Ruxolitinib Cream	0.13	± 1.534

Change from Baseline to Week 8

Group	Value	95% CI
Part B : Placebo Cream	2.13	± 2.233
Part B : Ruxolitinib Cream	-1.11	± 2.160

Change from Baseline to Week 12

Group	Value	95% CI
Part B : Placebo Cream	1.23	± 2.956
Part B : Ruxolitinib Cream	-2.55	± 2.873

Change from Baseline to Week 18

Group	Value	95% CI
Part B : Placebo Cream	-0.01	± 3.550
Part B : Ruxolitinib Cream	-4.16	± 3.461

Change from Baseline to Week 24

Group	Value	95% CI
Part B : Placebo Cream	-1.68	± 3.886
Part B : Ruxolitinib Cream	-3.92	± 3.794

Part A and B : Number of Treatment-emergent Adverse Events Secondary · Up to 100 weeks

Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment

Treatment Period

Group	Value	95% CI
Part A: Ruxolitinib Cream	9
Part B : Placebo Cream	28
Part B : Ruxolitinib Cream	23

Extension Period

Group	Value	95% CI
Part A: Ruxolitinib Cream	7
Part B : Placebo Cream	23
Part B : Ruxolitinib Cream	22

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 100 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Placebo Cream

Serious: 0/38 (0%)

Deaths: 0/38

Ruxolitinib Cream

Serious: 4/83 (5%)

Deaths: 0/83

Serious adverse events (6 terms)

Reaction	System	Placebo Cream	Ruxolitinib Cream
Diverticulitis	Infections and infestations	—	—
Generalised anxiety disorder	Psychiatric disorders	—	—
Infected neoplasm	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Lentigo maligna	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Non-cardiac chest pain	General disorders	—	—
Sepsis	Infections and infestations	—	—

Other adverse events (6 terms — click to expand)

Reaction	System	Placebo Cream	Ruxolitinib Cream
Nasopharyngitis	Infections and infestations	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—
Sinusitis	Infections and infestations	—	—
Urinary tract infection	Infections and infestations	—	—
Skin exfoliation	Skin and subcutaneous tissue disorders	—	—
Bronchitis	Infections and infestations	—	—

Most-reported serious reactions: Diverticulitis, Generalised anxiety disorder, Infected neoplasm, Lentigo maligna, Non-cardiac chest pain, Sepsis.

Data from ClinicalTrials.gov NCT02553330 adverse events section.

Sponsor's own description

A phase 2 study to find out if the drug ruxolitinib Phosphate Cream is safe and has beneficial effects in people who have alopecia areata (partial or complete hair loss) when applied to the skin.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. JAK inhibitors in dermatology: The promise of a new drug class.
   Damsky W, King BA. · · 2017 · cited 353× · PMID 28139263 · DOI 10.1016/j.jaad.2016.12.005
2. JAK inhibition as a therapeutic strategy for immune and inflammatory diseases.
   Schwartz DM, Kanno Y, Villarino A, Ward M, et al · · 2017 · cited 308× · PMID 29282366 · DOI 10.1038/nrd.2017.267
3. JAK-inhibitors. New players in the field of immune-mediated diseases, beyond rheumatoid arthritis.
   Fragoulis GE, McInnes IB, Siebert S. · · 2019 · cited 223× · PMID 30806709 · DOI 10.1093/rheumatology/key276
4. Emerging Topical and Systemic JAK Inhibitors in Dermatology.
   Solimani F, Meier K, Ghoreschi K. · · 2019 · cited 199× · PMID 31849996 · DOI 10.3389/fimmu.2019.02847
5. Targeting the Janus Kinase Family in Autoimmune Skin Diseases.
   Howell MD, Kuo FI, Smith PA. · · 2019 · cited 180× · PMID 31649667 · DOI 10.3389/fimmu.2019.02342
6. JAK3 as an Emerging Target for Topical Treatment of Inflammatory Skin Diseases.
   Alves de Medeiros AK, Speeckaert R, Desmet E, Van Gele M, et al · · 2016 · cited 112× · PMID 27711196 · DOI 10.1371/journal.pone.0164080
7. An overview of JAK/STAT pathways and JAK inhibition in alopecia areata.
   Lensing M, Jabbari A. · · 2022 · cited 100× · PMID 36110853 · DOI 10.3389/fimmu.2022.955035
8. Selectivity, efficacy and safety of JAKinibs: new evidence for a still evolving story.
   Bonelli M, Kerschbaumer A, Kastrati K, Ghoreschi K, et al · · 2024 · cited 95× · PMID 37923366 · DOI 10.1136/ard-2023-223850