Evaluating HSK39297 for treating PNH in patients new to complement inhibitors

A Phase III, Multicenter, Randomized, Open Label, Active-Controlled Study to Evaluate the Efficacy and Safety of HSK39297 Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Naive to Complement Inhibitor Therapy

This study is testing if a new medication called HSK39297 can safely help adults with paroxysmal nocturnal hemoglobinuria (PNH) who haven't tried other treatments yet, compared to the standard drug eculizumab.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 66 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Haisco Pharmaceutical Group Co., Ltd. (Industry-sponsored)
• Drugs / interventions: eculizumab
• Locations: 2 sites (Nanjing, Jiangsu and 1 other locations)
• Trial ID: NCT06799546 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy and safety of HSK39297 tablets in comparison to eculizumab in patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. Participants will be adults aged 18 to 75 with specific blood criteria indicating the severity of their condition. The study will involve monitoring the patients' responses to the treatment over a defined period to determine the effectiveness and safety of HSK39297.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with a confirmed diagnosis of PNH who have not received prior complement inhibitor therapy.

Not a fit: Patients with hereditary complement deficiencies or those who have received prior complement inhibitor treatments will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with PNH, potentially improving their quality of life and health outcomes.

How similar studies have performed: Other studies evaluating similar treatments for PNH have shown promising results, indicating a potential for success with this approach.

Eligibility criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Where this trial is running

Nanjing, Jiangsu and 1 other locations

• The First Affiliated Hospital of Nanjing Medical University — Nanjing, Jiangsu, China (Recruiting)
• The First Affiliated Hospital, College of Medicine, Zhejiang University — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Study coordinator: Fangqiong Li

Conditions PNH