Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders

Официальное название

Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis

Обзор

Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis

Вмешательства

• Препарат Descartes-08
  In part 1, three different doses will be administered to 3 participants with either disease indication to establish maximum tolerated dose In part 2, the MTD established in Part-1 will be administered as six once-weekly infusions to up to 10 participants per each of four baskets (cSLE, AAV, JDM and JMG) in an outpatient setting.

Первичные конечные точки

• Maximum tolerated dose in Part 1, type and frequency of treatment related SAE's in Part 2 [Срок оценки: Days 22 and 50 for Part1 , Days 22, 50 and Months 3,6,9 and 12 for part 2]
• Maximum Dose Tolerated [Срок оценки: Days 2250 for Part 1, Days 11,50, month 3 for part 2 in addition to months, 4,6,9,12]

Критерии участия

Критерии включения

• At least age 12
• definitive diagnosis of childhood-onset systemic lupus erythematous, juvenile Myasthenie gravis, juvenile dermatomyositis and AAV
• Signs and symptoms of moderate disease
• History of systemic treatment
• Parent/Guardian/Patient must be able to give written informed consent

Критерии исключения

• Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient;
• Abnormal PT/INR or PTT increased > 1.5-fold or patient is on anticoagulation therapy (except in cases of elevated PTT with documented lupus anticoagulant; or in patients who have been on stable doses of anticoagulation therapy for more than 6 months of VTE diagnosis; or in patients on stable doses of anticoagulation therapy for at least 8 weeks of atrial fibrillation diagnosis; these conditions will not be exclusionary unless, in the investigator's opinion, they make participation in the study unsafe);
• ANC < 1000 cells/microliter ;
• Hemoglobin < 8.0 g/dL ;
• Platelets < 50,000/mm3 (NOTE: platelet transfusions are permissible);
• ALT and/or AST with GGT ≥ 3× upper limit of normal
• Creatine Clearance less than 30mL/min /1.73 m2;
• History of primary immunodeficiency, organ, or allogeneic bone marrow transplant;
• Patients must be seronegative for hepatitis B surface antigen;
• Patients must be seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patients must be tested for the presence of viremia by RT-PCR and must be HCV RNA negative;
• History of positive HIV or positive HIV at screening;
• Active tuberculosis or positive QuantiFERON test at screening;
• Any other laboratory abnormality that, in the opinion of the investigator, may jeopardize the subject's ability to participate in the study; 23. Any active significant cardiac or pulmonary disease not related to the primary indication as determined by principal investigator and medical monitor Note: Patients with asthma and COPD controlled with inhaled medications are allowed; 24. Any arterial or venous thromboembolic events in the past 3 months; 25. History of malignancy that required treatment in the past 3 years except for successfully-treated squamous cell and/or basal cell carcinoma of the skin and/or breast or colon cancer that is surgically removed and did not require adjuvant chemotherapy or radiotherapy; 26. Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer); 27. Receipt of a live vaccination within 4 weeks prior to baseline (Day 1) or intent to receive live vaccination during the study (Note: mRNA-based vaccines such as those against SARS-CoV-2 are not considered live; likewise, the Janssen Covid-19 vaccine is not live); 28. History of significant recurrent infections or any active infection that may interfere with the patient's participation in the opinion of the investigator; 29. Any known psychiatric illness that may interfere with the patient's participation in the study in the opinion of the investigator.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 3 центра

• H01- Children's National Hospital — Washington D.C.
• H03- Washington University School of Medicine — St Louis
• Cincinnati Children's Hospital Medical Center — Cincinnati

Идентификаторы

NCT: NCT07089121 · HELIOS-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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