NCT01724580

Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes

APPROVED FOR MARKETING Last updated 17 January 2025

What this trial tests

trial testing Baricitinib in Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE). Approved for marketing.

Quick facts

Lead sponsor	Eli Lilly and Company
Status	APPROVED FOR MARKETING
Study type	EXPANDED_ACCESS
Sites	3 locations across United Kingdom, United States

Drugs / interventions tested

• Baricitinib (baricitinib) — full drug profile →

Conditions studied

• Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE) — all drugs for Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE) →
• Juvenile Dermatomyositis (JDM) — all drugs for Juvenile Dermatomyositis (JDM) →
• Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI) — all drugs for Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI) →
• Aicardi-Goutières Syndrome (AGS) — all drugs for Aicardi-Goutières Syndrome (AGS) →

Sponsor

Eli Lilly and Company — full company profile →

Who can join

6 Months and older, any sex, with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE) or Juvenile Dermatomyositis (JDM). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

Publications & conference data

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

1. The JAK-STAT pathway: impact on human disease and therapeutic intervention.
   O'Shea JJ, Schwartz DM, Villarino AV, Gadina M, et al · · 2015 · cited 1148× · PMID 25587654 · DOI 10.1146/annurev-med-051113-024537
2. Activated STING in a vascular and pulmonary syndrome.
   Liu Y, Jesus AA, Marrero B, Yang D, et al · · 2014 · cited 1086× · PMID 25029335 · DOI 10.1056/nejmoa1312625
3. Type I/II cytokines, JAKs, and new strategies for treating autoimmune diseases.
   Schwartz DM, Bonelli M, Gadina M, O'Shea JJ. · · 2016 · cited 474× · PMID 26633291 · DOI 10.1038/nrrheum.2015.167
4. JAK1/2 inhibition with baricitinib in the treatment of autoinflammatory interferonopathies.
   Sanchez GAM, Reinhardt A, Ramsey S, Wittkowski H, et al · · 2018 · cited 377× · PMID 29649002 · DOI 10.1172/jci98814
5. 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
6. Additive loss-of-function proteasome subunit mutations in CANDLE/PRAAS patients promote type I IFN production.
   Brehm A, Liu Y, Sheikh A, Marrero B, et al · · 2015 · cited 234× · PMID 26524591 · DOI 10.1172/jci81260
7. 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
8. 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