OncoTrials - мониторинг клинических исследований

Описание

The purpose of this study is to evaluate the efficacy and safety of HSK39297 tablets compared to eculizumab in Patients with PNH who Are Naive to Complement Inhibitor Therapy.

Критерии включения

• 1. Age ≥ 18 and ≤ 75 years, Male and female patients;
• 2. Diagnosis of PNH based on flow cytometry with clone size /> 10% by granulocytes;
• 3. Have not received complement inhibitor treatment;
• 4. Blood LDH values /> 1.5 ×upper limit of the normal range (ULN) ;
• 5. Hemoglobin level /< 10 g/dL at screening.

Критерии исключения

• 1. Hereditary or acquired complement deficiency;
• 2. Active primary or secondary immunodeficiency;
• 3. History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
• 4. History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
• 5. Patients with laboratory evidence of bone marrow failure (reticulocytes /< 100x10/^9/L, or platelets /< 30x10/^9/L or neutrophils /< 0.5x10/^9/L) ;
• 6. Active systemic infection within 2 weeks prior to study drug administration;
• 7. History of serious comorbidities that have been determined to be unsuitable for participation in the study.
• 8. Pregnant or Lactating women.

Описание

This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules. This trial includes an 8-week screening period, a 24-week treatment period, a 2-week dose reduction period, and a 4-week safety follow-up period.

Критерии включения

• 1. Understand the specific process of the experiment, voluntarily participate in this experiment, and sign a written informed consent form.
• 2. Age ≥18 on the day of signing the informed consent, regardless of gender
• 3. It was confirmed to be PNH during screening, and the clone size of red blood cells or granulocytes was detected by flow cytopy />10%
• 4. Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
• 5. The average hemoglobin level of at least two tests in 4 months before screening /< 10 g/dL
• 6. The average hemoglobin level of two tests in the central laboratory during screening /< 10 g/dL
• 7. Inoccution of Neisseris meningitis and Streptococcus pneumoniae vaccine at least 2 weeks before the first administration of HRS-5965; if HRS-5965 treatment must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination.
• 8. Male and female subjects with fertility must agree to adopt efficient contraceptive measures with their partners within 30 days from the signing of the informed consent form to the last administration, and have no family planning and no sperm/egg donation.

Критерии исключения

• 1. In addition to C5 complement inhibitors (ikuzuzumab/covalimamab), those who have participated in clinical trials of any other drug or medical device within 1 month before and are expected to have a residual effect of experimental treatment (judged by researchers), or those who were still in the follow-up period of a clinical trial or the 5 half-life of the experimental drug before screening Inside (whichever is longer)
• 2. Known or suspected hereditary or acquired complement deficiency
• 3. Currently active primary or secondary immunodeficiency
• 4. Those who have a history of splenectomy or plan to perform surgery during the trial
• 5. History of bone marrow/hematopoietic stem cells or solid organ transplantation
• 6. Diagnosed malignant tumors in the past 5 years
• 7. There is laboratory evidence for patients with bone marrow failure during screening
• 8. History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
• 9. There is or is suspected of systemic active bacteria, virus or fungal infection 2 weeks before the first administration of HRS-5965 (according to the researcher`s judgment)
• 10. Fever occurs within 1 week before the first administration of HRS-5965 (body temperature ≥38 ℃)
• 11. Human immunodeficiency virus (HIV) infection
• 12. Hepatitis B surface antigen (HBsAg) and hepatitis C antibody (HCVAb) are positive during screening, or abnormal liver function test during screening
• 13. Use any of the following drugs, unless there is a stable treatment plan before screening: a) erythropoietin (ESA), hypoxic-inducing factor proaminoyl hydroxylase inhibitor (HIF-PHI) or immunosuppressant for at least 8 weeks b) Systemic use of glucocorticoids (≤15 mg/day Prednisone or equivalent doses of glucocorticoids) at least 4 weeks c) Vitamin K antagonists (such as warfarin) have a stable international standardized ratio (INR) at least 4 weeks d) Low molecular weight heparin, oral anticoagulants such as aspirin, rvaroxaban, apifloxaban, etc. at least 4 weeks e) Iron supplements , vitamin B12, folic acid or androgen for at least 4 weeks
• 14. During screening, there are serious concurrent diseases, such as severe kidney disease (such as eGFR/<30 mL/min/1.73 m2, dialysis), advanced heart disease (such as NYHA level IV), severe lung disease such as pulmonary hypertension (WHO level IV) or liver disease (such as active hepatitis), etc. , judged by the researcher that it is not suitable to participate in the researcher
• 15. Any medical condition determined by the researcher that it may affect the patient`s participation in the trial, chronic anemia or unstable thrombosis events that may exist for other causes, and other conditions judged by the researcher to be unsuitable for participation in the trial
• 16. Those who are suspected of being allergic to experimental drugs or any ingredient in experimental drugs
• 17. Screening positive blood pregnancy test and breastfeeding women at the time of the visit

Описание

This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of the study.

Критерии включения

• 1. Patients With PNH who have previously received and completed HSK39297 treatment , and According to the researchers` judgment, the benefits of treatment outweigh the risks and may benefit from continued treatment with HSK39297;
• 2. Understand the study procedures and methods, voluntarily participate in this trial.

Критерии исключения

• 1. Hereditary or acquired complement deficiency;
• 2. Active primary or secondary immunodeficiency;
• 3. History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants;
• 4. History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;
• 5. History of serious comorbidities that have been determined to be unsuitable for participation in the study;
• 6. Pregnant or Lactating women.

Описание

This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.

Критерии включения

• All patients who received Fabhalta.
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Критерии исключения

• Patients receiving Fabhalta for an unapproved indication under the Clinical Trials Act or GCP.

Описание

A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.

Критерии включения

• 1. Diagnosis of PNH confirmed by flow cytometry with clone size /> 10%.
• 2. Have not received complement inhibitor therapy;
• 3. LDH /> 1.5/*ULN at screening.
• 4. Hemoglobin level /< 10 g/dL at screening.

Критерии исключения

• 1. Known or suspected hereditary or acquired complement deficiency;
• 2. Patients with laboratory evidence of bone marrow failure (reticulocytes /<100x109/L; platelets /<30x109/L; neutrophils /<0.5x109/L);
• 3. Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
• 4. History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
• 5. Positive of HIV, HBsAg or HCVAb.

Описание

The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.

Критерии включения

• * Female participant must have a medically confirmed qualifying pregnancy (prospectively or retrospectively identified).
• * Participant informed consent (written or e-consent per local regulations or ethics committee requirements) must be obtained prior to the participant`s enrollment. If the participant is a minor, consent must be obtained from the parent or legal guardian, with assent from the minor (as locally appropriate).
• * Willing to provide contact information for the participant.
• * Willing to authorize HCP(s) to release maternal and infant medical information to the study, upon request, if applicable to current local regulations.
• * Diagnosed with an indication for which Ultomiris is approved, based on HCP or medical records.
• * Exposed to Ultomiris at any point during the defined exposure window based on HCP or medical record documentation. (If exact exposure dates are unknown, the reporter must be able to specify or estimate trimester or timing of exposure /[prior to conception as LMP+14 days, or during breastfeeding/].)
• * Use of Ultomiris per local product information (i.e., United States Prescribing Information /[USPI/] or summary of product characteristics /[SmPC/])

Критерии исключения

• * Participants who are unable to provide consent or assent (as locally appropriate) (e.g., diagnosed with severe psychiatric conditions or severe intellectual disabilities) will be excluded from this study