OncoTrials - мониторинг клинических исследований

Описание

This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.

Критерии включения

• 1. Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
• 2. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.

Критерии исключения

• 1. History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
• 2. Known or suspected hereditary complement deficiency.
• 3. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.

Описание

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Критерии включения

• * Confirmed diagnosis of PNH.
• * CS-EVH defined by: Anemia: Hgb ≤ 10.5 g/dL, and absolute reticulocyte count ≥ 100 × 109/L
• * Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.
• * all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1
• * vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae

Критерии исключения

• * Platelet count /< 30000/μL or there is a need for platelet transfusions.
• * ANC /< 500/μL.
• * Clinically significant laboratory abnormalities related to liver function, including:
• * ALT /> 2 × ULN or ALT /> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/mL.
• * Direct bilirubin /> 2 × ULN, unless, in the Investigator`s opinion, is due to hemolysis or Gilbert`s syndrome based on medical history.
• * Current evidence of biliary cholestasis.
• * Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.
• * History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.
• * Known or suspected complement deficiency.
• * Active bacterial or viral infection, a body temperature /> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.

Описание

The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.

Критерии включения

• 1. Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland.
• 2. Patients willing to participate in the study and signed ICF.

Критерии исключения

• 1. Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP.
• 2. Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

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

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