Evaluating MY008211A for treating Paroxysmal Nocturnal Hemoglobinuria in adults
A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis.
This study is testing a new medication called MY008211A to see if it can help adults with Paroxysmal Nocturnal Hemoglobinuria who are experiencing active hemolysis.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Wuhan Createrna Science and Technology Co., Ltd Industry-sponsored |
| Locations | 1 site (Tianjin, Tianjin) |
| Trial ID | NCT06050226 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the efficacy and safety of MY008211A in adult patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH) who show signs of active hemolysis. Participants must be naive to complement inhibitor therapy and will receive MY008211A tablets as the intervention. The study will take place in China and will follow strict eligibility criteria to ensure the appropriate patient population is selected for evaluation.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of PNH and specific laboratory criteria indicating active hemolysis.
Not a fit: Patients with severe blood cell deficiencies or those who have previously used complement inhibitors may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from PNH.
How similar studies have performed: While this approach is being tested in this specific context, similar studies have shown promise in treating PNH with novel therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male and female participants ≥ 18 years of age, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and flow cytometry with clone size ≥ 10%. * Mean hemoglobin level \<100 g/L. * LDH \> 1.5 x Upper Limit of Normal (ULN) * Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given. Exclusion Criteria: * Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L. * Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest. * History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus. * Known or suspected hereditary complement deficiency * Previous bone marrow or hematopoietic stem cell transplantation. * Previous splenectomy. * A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
Where this trial is running
Tianjin, Tianjin
- Nstitute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College — Tianjin, Tianjin, China (Recruiting)
Study contacts
- Principal investigator: Fengkui Zhang, Ph.D — Blood Disease Hospital, Chinese Academy of Medical Sciences
- Study coordinator: Zhen Fu, Ph.D
- Email: fuzhen@createrna.com
- Phone: 13477072640
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.