NCT03288324

Open-label Study of Tofacitinib for Moderate to Severe Skin Involvement in Young Adults With Lupus

Completed Phase 1, PHASE2 Results posted Last updated 3 March 2026

What this trial tests

Phase 1, PHASE2 trial testing Tofacitinib in Cutaneous Lupus in 13 participants. Completed in 1 December 2023.

Timeline

23 August 2017

Primary endpoint
1 November 2022

1 December 2023

Quick facts

Lead sponsor	Children's Hospital Medical Center, Cincinnati
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	13
Start date	23 August 2017
Primary completion	1 November 2022
Estimated completion	1 December 2023
Sites	2 locations across United States

Drugs / interventions tested

• Tofacitinib (tofacitinib) — full drug profile →

Conditions studied

• Cutaneous Lupus — all drugs for Cutaneous Lupus →
• Systemic Lupus Erythematosus — all drugs for Systemic Lupus Erythematosus →

Sponsor

Children's Hospital Medical Center, Cincinnati

Who can join

Adults 18 to 45, any sex, with Cutaneous Lupus or Systemic Lupus Erythematosus. 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.

Oral Clearance (CL/F) (Cohort 1 Only) Primary · Day 5

Apparent total clearance of the drug from plasma after oral administration

Group	Value	95% CI
Cohort 1	28.4	± 23.5

Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score Secondary · weeks 4, 8 and 24 compared to baseline.

Proportion of subjects who achieve a skin response per the validated CLASI The Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) activity score consists of two scores. The first summarizes the activity of the disease while the second is a measure of the damage done by the disease. The Activity Score range is 0-70 with the maximum score (70) indicating the worst outcome. The Damage Score range is 0-80 with the maximum score (80) indicating the worst outcome.

Baseline

Group	Value	95% CI
Cohort 1 & Cohort 2	16.55	± 8.03

Week 4

Group	Value	95% CI
Cohort 1 & Cohort 2	10.91	± 8.4

Week 8

Group	Value	95% CI
Cohort 1 & Cohort 2	8.64	± 7.8

Week 24

Group	Value	95% CI
Cohort 1 & Cohort 2	8.82	± 7.9

AUCt (Cohort 1 Only) Secondary · Day 5

Area under the plasma concentration-time curve linear scale Median Concentration (ng/mL) per nominal time 0-8 hours.

Group	Value	95% CI
Cohort 1	176	± 23.5

Cmax (Cohort 1 Only) Secondary · Day 5

Maximum (or peak) plasma concentration of Tofacitinib

Group	Value	95% CI
Cohort 1	51.7	± 21.6

Tmax (Cohort 1 Only) Secondary · Day 5

Time to reach maximum (peak) plasma concentration following administration of Tofacitinib

Group	Value	95% CI
Cohort 1	1	.5 – 2

Vz/F (Cohort 1 Only) Secondary · Day 5

Apparent volume of distribution during terminal phase after non-intravenous administration

Group	Value	95% CI
Cohort 1	85.2	± 25.6

Half-life of Tofacitinib (Cohort 1 Only) Secondary · Day 5

half-life of Tofacitinib

Group	Value	95% CI
Cohort 1	2.1	± 0.321

Safety of Tofacitinib: Total Number of Adverse Events is Reported (Cohorts 1 and 2) Secondary · 76 weeks

Rate and severity of adverse events and lab abnormalities experienced by participants in both cohorts of the research study.

Group	Value	95% CI
Cohort 1 and 2	73

Steroid Dose Comparison at 72 Weeks Secondary · 72 weeks

Assess steroid sparing properties of Tofacitinib by comparing doses to baseline and rate of steroid discontinuation

Group	Value	95% CI
Cohort 1 & Cohort 2	7.5	± 3.54

Change in SLE Disease Activity Index (SLEDAI) Score Secondary · 72 weeks

Measure Tofacitinib impact on disease activity The SLEDAI (version 2k) was completed and its MC domain (range:0-6) and extra-MC (range: 0-99) scores calculated. SLEDAI summary scores can be interpreted as following: 1-5 mild disease activity, 6-10 moderate disease activity, and 11 and more severe disease activity27, range: 0-105), with higher scores indicating higher activity

Baseline

Group	Value	95% CI
Cohort 1 & Cohort 2	7.1	± 1.96

Week 24

Group	Value	95% CI
Cohort 1 & Cohort 2	4	± 1.6

Week 72

Group	Value	95% CI
Cohort 1 & Cohort 2	4.4	± 2.41

Change in SKINDEX Score Secondary · Baseline, week 24 and week 72

The Skindex-29 is a validated quality-of-life instrument designed to assess the impact of skin disease on patients. Each of the 29 items is rated on a 5-point Likert scale, from 1 ("Never") to 5 ("All the time"), with higher values indicating greater impairment of quality of life. Responses are averaged within domains or across all items, and the resulting mean scores are transformed to a 0-100. On this scale, 0 represents the lowest possible impact and 100 represents the highest possible impact.

Baseline

Group	Value	95% CI
Cohort 1 & Cohort 2	37.6	± 24.16

Week 24

Group	Value	95% CI
Cohort 1 & Cohort 2	24.3	± 28.67

Week 72

Group	Value	95% CI
Cohort 1 & Cohort 2	25.5	± 30.71

Change in Patients Global Assessment Score Secondary · Baseline, week 24 and week 76

Quality-of-life measure for patients The Global Assessment is a rating scale used to capture the patient's overall perception of disease activity. Patients rate their condition on a 0-10 scale, where 0 indicates no disease activity and 10 represents the maximum level of disease activity. Higher scores therefore reflect worse overall disease impact.

Baseline

Group	Value	95% CI
Cohort 1 & Cohort 2	2.7	± 1.47

week 24

Group	Value	95% CI
Cohort 1 & Cohort 2	1.3	± 1.2

week 72

Group	Value	95% CI
Cohort 1 & Cohort 2	1.5	± 1.54

Adverse events — posted to ClinicalTrials.gov

Time frame: 76 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Cohort 1 and 2

Serious: 1/11 (9%)

Deaths: 0/11

Serious adverse events (3 terms)

Reaction	System	Cohort 1 and 2
Pelvic inflammatory disease	Infections and infestations	—
Appendicitis	Infections and infestations	—
Migraine with aura	Nervous system disorders	—

Other adverse events (42 terms — click to expand)

Reaction	System	Cohort 1 and 2
Nausea	Gastrointestinal disorders	—
Upper respiratory tract infection	Infections and infestations	—
Diarrhoea	Gastrointestinal disorders	—
Fungal infection	Infections and infestations	—
Headache	Nervous system disorders	—
Nasopharyngitis	Infections and infestations	—
Onychomycosis	Infections and infestations	—
Skin papilloma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
Systemic lupus erythematosus	Musculoskeletal and connective tissue disorders	—
Urinary tract infection	Infections and infestations	—
Vomiting	Gastrointestinal disorders	—
Abdominal tenderness	Gastrointestinal disorders	—
Acne	Skin and subcutaneous tissue disorders	—
Arthralgia	Musculoskeletal and connective tissue disorders	—
Back pain	Musculoskeletal and connective tissue disorders	—
Bronchitis	Infections and infestations	—
Burns second degree	Injury, poisoning and procedural complications	—
Chlamydial infection	Infections and infestations	—
Conjunctival haemorrhage	Eye disorders	—
Depression	Psychiatric disorders	—
Dysuria	Renal and urinary disorders	—
Ear infection	Infections and infestations	—
Folliculitis	Infections and infestations	—
Gastroenteritis	Infections and infestations	—
Gastrointestinal viral infection	Infections and infestations	—
Hypokalaemia	Metabolism and nutrition disorders	—
Iron deficiency anaemia	Blood and lymphatic system disorders	—
Ligament sprain	Injury, poisoning and procedural complications	—
Medial tibial stress syndrome	Musculoskeletal and connective tissue disorders	—
Menorrhagia	Reproductive system and breast disorders	—
Otitis externa	Infections and infestations	—
Pelvic inflammatory disease	Infections and infestations	—
Rash	Skin and subcutaneous tissue disorders	—
Sinusitis	Infections and infestations	—
Staphylococcal infection	Infections and infestations	—
Streptococcal infection	Infections and infestations	—
Suicidal ideation	Psychiatric disorders	—
Syncope	Nervous system disorders	—
Tinnitus	Ear and labyrinth disorders	—
Urticaria	Skin and subcutaneous tissue disorders	—

Most-reported serious reactions: Pelvic inflammatory disease, Appendicitis, Migraine with aura.

Data from ClinicalTrials.gov NCT03288324 adverse events section.

Sponsor's own description

This 76-week, 3-part Phase 1b/2 study is intended to evaluate the pharmacological properties (pharmacokinetics and pharmacodynamics), safety, tolerability and preliminary effectiveness of TOFA administrated to young adults (18-45 years) with moderately to severely active SLE-CL. Subjects will be studied at the Cincinnati Children's Hospital Medical Center (CCHMC) and in Cleveland at MetroHealth Medical Center.

Publications & conference data

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

1. Lipid metabolism in sickness and in health: Emerging regulators of lipotoxicity.
   Yoon H, Shaw JL, Haigis MC, Greka A. · · 2021 · cited 350× · PMID 34547235 · DOI 10.1016/j.molcel.2021.08.027
2. Janus kinase-targeting therapies in rheumatology: a mechanisms-based approach.
   Tanaka Y, Luo Y, O'Shea JJ, Nakayamada S. · · 2022 · cited 329× · PMID 34987201 · DOI 10.1038/s41584-021-00726-8
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. JAK/STAT pathway: Extracellular signals, diseases, immunity, and therapeutic regimens.
   Hu Q, Bian Q, Rong D, Wang L, et al · · 2023 · cited 190× · PMID 36911202 · DOI 10.3389/fbioe.2023.1110765
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. New Horizons in the Genetic Etiology of Systemic Lupus Erythematosus and Lupus-Like Disease: Monogenic Lupus and Beyond.
   Demirkaya E, Sahin S, Romano M, Zhou Q, et al · · 2020 · cited 98× · PMID 32151092 · DOI 10.3390/jcm9030712
7. 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
8. JAK-STAT signaling in human disease: From genetic syndromes to clinical inhibition.
   Luo Y, Alexander M, Gadina M, O'Shea JJ, et al · · 2021 · cited 92× · PMID 34625141 · DOI 10.1016/j.jaci.2021.08.004