Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes (NCT01724580) | Clinical Trial Compass
APPROVED_FOR_MARKETINGNot Applicable
Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes
United States, United Kingdom
Plain-language 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.
Who can participate
Age range6 Months
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 equiv…