Long-term safety and tolerability of MY008211A tablets for patients with PNH
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study is testing how safe and well-tolerated MY008211A tablets are for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have already benefited from the treatment in earlier trials.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 120 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Wuhan Createrna Science and Technology Co., Ltd Industry-sponsored |
| Locations | 1 site (Tianjin, Tianjin Municipality) |
| Trial ID | NCT06933914 on ClinicalTrials.gov |
What this trial studies
This multicenter, single-arm, open-label study aims to evaluate the long-term safety, tolerability, and efficacy of MY008211A tablets in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It provides continued access to MY008211A for patients who have previously completed Phase 2 or 3 studies and have shown benefit from the treatment. The study will monitor participants for any adverse effects and assess the overall effectiveness of the medication over an extended period.
Who should consider this trial
Good fit: Ideal candidates are patients with PNH who have previously completed MY008211A treatment and demonstrated a benefit.
Not a fit: Patients with a history of recurrent invasive infections or known hereditary complement deficiencies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a safe and effective long-term treatment option for patients with PNH.
How similar studies have performed: Other studies have shown promising results with similar approaches, but this specific long-term evaluation of MY008211A is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Where this trial is running
Tianjin, Tianjin Municipality
- Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences) — Tianjin, Tianjin Municipality, China (Recruiting)
Study contacts
- Study coordinator: Wuhan Createrna Science and Technology Co.,Ltd
- Email: lcyxzx@createrna.com
- Phone: 027-68788900
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.