Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE)NCT01724580

Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes

A study testing Baricitinib in people with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE) and Juvenile Dermatomyositis (JDM), run by Eli Lilly and Company at 3 sites in 2 countries.

Approved For Marketing

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

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Trial summary

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

Who is it for?: Ages 6 months and older
Conditions the study is for: Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM), Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI)
What is being tested?: Baricitinib
Type of treatment: Pill or oral medicine
How many people: Not given
Where: 3 sitesin 2 countries
Study dates: Start not givento not given
Phase: Expanded access
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 6 months and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Are ≥17.5 months of age (or are ≥6 months of age with Aicardi-Goutières Syndrome [AGS]). Participants younger than 17.5 months of age can be considered for enrollment after discussion with the sponsor.
Have systemic signs and symptoms of inflammation as manifested by the presence of two or more of the following symptoms: rash, fever, musculoskeletal pain, headache, fatigue, weakness, respiratory/breathing symptoms, or ulcers/ischemic lesions.
Have an average daily Diary Score of ≥0.5 (CANDLE Diary; used also for CANDLE-related conditions, AGS Diary) or ≥1.0 (SAVI) or ≥1.0 exclusive of headache and fever symptoms (JDM Diary) assessed over at least 2 weeks prior to entry, if available. Otherwise, participants can complete the diary after study consent is signed during the screening period and meet the inclusion criteria for enrollment into the study.
Are ≥8.5 kilogram (kg) in body weight. Participants weighing less than 8.5 kg can be considered for enrollment after discussion with the sponsor.
Have been previously treated with at least 1 biologic therapy and, in the opinion of the investigator, did not respond or are no longer responding to therapy. If the participant has been diagnosed with CANDLE, Nakajo-Nishimura Syndrome (NNS), SAVI, AGS, or an equivalent syndrome, the need for previous biologic therapy is not required.
Require treatment with oral corticosteroids (≥0.15 milligrams per kilogram per day [mg/kg/d] of prednisone or its equivalent) for control of systemic signs and symptoms of their chronic inflammatory disease for at least 2 weeks prior to study entry, or in the opinion of the investigator, have failed an adequate course of steroids. Treatment with or failure of treatment with steroids is not required for participants with AGS or a confirmed genetic diagnosis of CANDLE or SAVI.
Have had previous documented elevations in acute-phase reactants (for example, high sensitivity C-reactive protein) considered to be the result of the inflammatory disease (participants with CANDLE or CANDLE-related conditions only).
Have the ability to provide informed consent or have legal representative who is willing and able to provide written informed consent, provided that assent is obtained from participants at an age-appropriate level.

Who cannot join

Have received an immunosuppressive biologic agent/monoclonal antibody within 4 half-lives prior to entry, for example, anakinra (4 half-lives=18 hours); etanercept (4 half-lives=18 days); infliximab; adalimumab (4 half-lives=36 days); use of intravenous immune globulin (IVIg) is permitted.
Are pregnant or nursing at the time of entry.
Are females of childbearing potential (women >12 or who have had at least 1 menstrual period regardless of age) who are sexually active and who do not agree to use 2 forms of highly effective methods of birth control or remain abstinent during the study and for at least 28 days following the last dose of investigational product.
Are sexually active males who do not agree to use 2 forms of highly effective birth control with female partners of childbearing potential or remain abstinent during the study and for at least 28 days following the last dose of investigational product.
Have had symptomatic herpes zoster infection within 12 weeks prior to entry or during the screening period.
Have a history of disseminated/complicated herpes zoster (for example, multidermatomal involvement, ophthalmic zoster, central nervous system [CNS] involvement, postherpetic neuralgia).

Show all 21Show fewer

Have evidence of active infection, at the time of entry or during the screening period, that in the opinion of the investigator, would pose an unacceptable risk for participating in the study.
Have a history of active hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
Have documented high titer autoantibodies suggestive clinically of autoimmune diseases other than severe JDM.
Are immunocompromised and, in the opinion of the investigator, are at an unacceptable risk for participating in the study.
Have had a serious systemic or local infection (including an infectious mononucleosis-like illness or herpes zoster) within 12 weeks prior to entry or during the screening period.
Have been exposed to a live vaccine within 12 weeks prior to entry or are expected to need/receive a live vaccine (including herpes zoster vaccination) during the course of the study.
Note: Investigators should review the vaccination status of the participants and follow the local guidelines for vaccination with nonlive vaccines intended to prevent infectious disease prior to entering participants into the study.
Have had household contact with a person with active tuberculosis (TB) and did not receive appropriate and documented prophylaxis for TB.
Have a serious and/or unstable illness that, in the opinion of the investigator, poses an unacceptable risk for the participant's participation in the study.
Have an estimated glomerular filtration rate (eGFR) based on the most recent available serum creatinine of <40 milliliters/minute/1.73 per square meter.
Have or have had a history of lymphoproliferative disease; or signs or symptoms suggestive of possible lymphoproliferative disease, or active primary or recurrent malignant disease; or been in remission from clinically significant malignancy for <5 years.
Note: Participants with resolved cervical dysplasia, or no more than 3 successfully treated basal-cell carcinoma of the skin, may participate in this study.
Have a history of chronic alcohol abuse or intravenous (IV) drug abuse within the 2 years prior to entry.
Are unable or unwilling to make themselves available for the duration of the study and/or are unwilling to follow study restrictions/procedures.
Are currently enrolled in, or discontinued within the last 30 days from a clinical trial involving an investigational product or non-approved use of a drug or device (other than the investigational product used in this study), or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study.

Trial locations and contacts

3 sites in 2 countries.

Country

City, postcode or hospital

3 sites in 2 countries

United States2 sites

Translational Autoinflammatory Disease (TADS)
Bethesda, Maryland, 20892
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

United Kingdom1 site

Great Ormond Street Hospital
London, WC1N 1EH

Other trials in United Kingdom

About this study

Official title: Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis

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

Conditions: Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM), Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI), Aicardi-Goutières Syndrome (AGS)
Being tested: Baricitinib
Medical subject headings: Fever, Dermatomyositis, Bites and Stings

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BaricitinibDrug
Also called LY3009104, INCB 028050
Administered orally

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

First posted12 Nov 2012 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (4)Papers the sponsor has linked to this study.
Other trials for Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE)Studies of the same condition, open and under way.
More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Expanded access

IDs, dates and publications

ClinicalTrials.gov ID: NCT01724580
Sponsor's protocol ID: 14559
Other IDs: I4V-MC-JAGA
Status last verified: Jan 2025
First submitted: 5 Nov 2012
Last updated: 17 Jan 2025
Results posted: No

Publications

Adang LA, Gavazzi F, D'Aiello R, Isaacs D, Bronner N, Arici ZS, Flores Z, Jan A, Scher C, Sherbini O, Behrens EM, Goldbach-Mansky R, Olson TS, Lambert MP, Sullivan KE, Teachey DT, Witmer C, Vanderver A, Shults J. Hematologic abnormalities in Aicardi Goutieres Syndrome. Mol Genet Metab. 2022 Aug;136(4):324-329. doi: 10.1016/j.ymgme.2022.06.003. Epub 2022 Jun 16.
PubMed 35786528
Kim H, Dill S, O'Brien M, Vian L, Li X, Manukyan M, Jain M, Adeojo LW, George J, Perez M, Grom AA, Sutter M, Feldman BM, Yao L, Millwood M, Brundidge A, Pichard DC, Cowen EW, Shi Y, Lu S, Tsai WL, Gadina M, Rider LG, Colbert RA. Janus kinase (JAK) inhibition with baricitinib in refractory juvenile dermatomyositis. Ann Rheum Dis. 2021 Mar;80(3):406-408. doi: 10.1136/annrheumdis-2020-218690. Epub 2020 Aug 25. No abstract available.
PubMed 32843325
Sanchez GAM, Reinhardt A, Ramsey S, Wittkowski H, Hashkes PJ, Berkun Y, Schalm S, Murias S, Dare JA, Brown D, Stone DL, Gao L, Klausmeier T, Foell D, de Jesus AA, Chapelle DC, Kim H, Dill S, Colbert RA, Failla L, Kost B, O'Brien M, Reynolds JC, Folio LR, Calvo KR, Paul SM, Weir N, Brofferio A, Soldatos A, Biancotto A, Cowen EW, Digiovanna JJ, Gadina M, Lipton AJ, Hadigan C, Holland SM, Fontana J, Alawad AS, Brown RJ, Rother KI, Heller T, Brooks KM, Kumar P, Brooks SR, Waldman M, Singh HK, Nickeleit V, Silk M, Prakash A, Janes JM, Ozen S, Wakim PG, Brogan PA, Macias WL, Goldbach-Mansky R. JAK1/2 inhibition with baricitinib in the treatment of autoinflammatory interferonopathies. J Clin Invest. 2018 Jul 2;128(7):3041-3052. doi: 10.1172/JCI98814. Epub 2018 Jun 11.
PubMed 29649002
Kim H, Brooks KM, Tang CC, Wakim P, Blake M, Brooks SR, Montealegre Sanchez GA, de Jesus AA, Huang Y, Tsai WL, Gadina M, Prakash A, Janes JM, Zhang X, Macias WL, Kumar P, Goldbach-Mansky R. Pharmacokinetics, Pharmacodynamics, and Proposed Dosing of the Oral JAK1 and JAK2 Inhibitor Baricitinib in Pediatric and Young Adult CANDLE and SAVI Patients. Clin Pharmacol Ther. 2018 Aug;104(2):364-373. doi: 10.1002/cpt.936. Epub 2017 Dec 8.
PubMed 29134648

Study at a glance

From the registry record, last updated by the sponsor on 17 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis
ClinicalTrials.gov ID: NCT01724580
Acronym / sponsor ID: 14559
Conditions: Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM), Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI), Aicardi-Goutières Syndrome (AGS)
Study type: Expanded access
Recruitment status: Approved For Marketing
Lead sponsor: Eli Lilly and CompanyIndustry (pharma, biotech, devices)
First posted: 12 Nov 2012 (estimated)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Who may qualify

Ages: 6 months and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (8 listed)

Are ≥17.5 months of age (or are ≥6 months of age with Aicardi-Goutières Syndrome [AGS]). Participants younger than 17.5 months of age can be considered for enrollment after discussion with the sponsor.
Have systemic signs and symptoms of inflammation as manifested by the presence of two or more of the following symptoms: rash, fever, musculoskeletal pain, headache, fatigue, weakness, respiratory/breathing symptoms, or ulcers/ischemic lesions.
Have an average daily Diary Score of ≥0.5 (CANDLE Diary; used also for CANDLE-related conditions, AGS Diary) or ≥1.0 (SAVI) or ≥1.0 exclusive of headache and fever symptoms (JDM Diary) assessed over at least 2 weeks prior to entry, if available. Otherwise, participants can complete the diary after study consent is signed during the screening period and meet the inclusion criteria for enrollment into the study.
Are ≥8.5 kilogram (kg) in body weight. Participants weighing less than 8.5 kg can be considered for enrollment after discussion with the sponsor.
Have been previously treated with at least 1 biologic therapy and, in the opinion of the investigator, did not respond or are no longer responding to therapy. If the participant has been diagnosed with CANDLE, Nakajo-Nishimura Syndrome (NNS), SAVI, AGS, or an equivalent syndrome, the need for previous biologic therapy is not required.

Key exclusion criteria (21 listed)

Have received an immunosuppressive biologic agent/monoclonal antibody within 4 half-lives prior to entry, for example, anakinra (4 half-lives=18 hours); etanercept (4 half-lives=18 days); infliximab; adalimumab (4 half-lives=36 days); use of intravenous immune globulin (IVIg) is permitted.
Are pregnant or nursing at the time of entry.
Are females of childbearing potential (women >12 or who have had at least 1 menstrual period regardless of age) who are sexually active and who do not agree to use 2 forms of highly effective methods of birth control or remain abstinent during the study and for at least 28 days following the last dose of investigational product.
Are sexually active males who do not agree to use 2 forms of highly effective birth control with female partners of childbearing potential or remain abstinent during the study and for at least 28 days following the last dose of investigational product.
Have had symptomatic herpes zoster infection within 12 weeks prior to entry or during the screening period.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BaricitinibAlso: LY3009104, INCB 028050Administered orally	Drug	–

Primary outcome

Not reported in the source record.

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2025.
Derived from this study: PubMed 35786528Adang LA, Gavazzi F, D'Aiello R, Isaacs D, Bronner N, Arici ZS, Flores Z, Jan A, Scher C, Sherbini O, Behrens EM, Goldbach-Mansky R, Olson TS, Lambert MP, Sullivan KE, Teachey DT, Witmer C, Vanderver A, Shults J. Hematologic abnormalities in Aicardi Goutieres Syndrome. Mol Genet Metab. 2022 Aug;136(4):324-329. doi: 10.1016/j.ymgme.2022.06.003. Epub 2022 Jun 16.
Derived from this study: PubMed 32843325Kim H, Dill S, O'Brien M, Vian L, Li X, Manukyan M, Jain M, Adeojo LW, George J, Perez M, Grom AA, Sutter M, Feldman BM, Yao L, Millwood M, Brundidge A, Pichard DC, Cowen EW, Shi Y, Lu S, Tsai WL, Gadina M, Rider LG, Colbert RA. Janus kinase (JAK) inhibition with baricitinib in refractory juvenile dermatomyositis. Ann Rheum Dis. 2021 Mar;80(3):406-408. doi: 10.1136/annrheumdis-2020-218690. Epub 2020 Aug 25. No abstract available.
Derived from this study: PubMed 29649002Sanchez GAM, Reinhardt A, Ramsey S, Wittkowski H, Hashkes PJ, Berkun Y, Schalm S, Murias S, Dare JA, Brown D, Stone DL, Gao L, Klausmeier T, Foell D, de Jesus AA, Chapelle DC, Kim H, Dill S, Colbert RA, Failla L, Kost B, O'Brien M, Reynolds JC, Folio LR, Calvo KR, Paul SM, Weir N, Brofferio A, Soldatos A, Biancotto A, Cowen EW, Digiovanna JJ, Gadina M, Lipton AJ, Hadigan C, Holland SM, Fontana J, Alawad AS, Brown RJ, Rother KI, Heller T, Brooks KM, Kumar P, Brooks SR, Waldman M, Singh HK, Nickeleit V, Silk M, Prakash A, Janes JM, Ozen S, Wakim PG, Brogan PA, Macias WL, Goldbach-Mansky R. JAK1/2 inhibition with baricitinib in the treatment of autoinflammatory interferonopathies. J Clin Invest. 2018 Jul 2;128(7):3041-3052. doi: 10.1172/JCI98814. Epub 2018 Jun 11.
Derived from this study: PubMed 29134648Kim H, Brooks KM, Tang CC, Wakim P, Blake M, Brooks SR, Montealegre Sanchez GA, de Jesus AA, Huang Y, Tsai WL, Gadina M, Prakash A, Janes JM, Zhang X, Macias WL, Kumar P, Goldbach-Mansky R. Pharmacokinetics, Pharmacodynamics, and Proposed Dosing of the Oral JAK1 and JAK2 Inhibitor Baricitinib in Pediatric and Young Adult CANDLE and SAVI Patients. Clin Pharmacol Ther. 2018 Aug;104(2):364-373. doi: 10.1002/cpt.936. Epub 2017 Dec 8.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) study director

Eli Lilly and Company

3 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 17 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01724580, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2025.