Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Expanded Access
Eligible Ages 6 Months and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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.

Exclusion Criteria:

  • - 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).
  • - 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 Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT01724580
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Eli Lilly and Company
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

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)
Principal Investigator Affiliation Eli Lilly and Company
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Approved for marketing
Countries United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

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)

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Bethesda, Maryland

Status

Address

Translational Autoinflammatory Disease (TADS)

Bethesda, Maryland, 20892

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Status

Address

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

International Sites

Great Ormond Street Hospital, London, United Kingdom

Status

Address

Great Ormond Street Hospital

London, , WC1N 1EH