Clinical trial NCT06561841: A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
Official title: A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Lead sponsor: Haisco Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-08-20
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.


Eligibility criteria: Inclusion Criteria:

1. Male and female participants ≥ 18 years of age;
2. Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes;
3. Have not received complement inhibitor treatment;
4. Blood lactate dehydrogenase(LDH) values \> 1.5 ×upper limit of the normal range (ULN) ;
5. Hemoglobin level \< 100 g/L during the screening period.

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.

Primary outcomes:
measure: Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions
timeFrame: Baseline, 24 weeks
Secondary outcomes:
measure: Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal
timeFrame: Baseline, 24 weeks
measure: Change from baseline in hemoglobin
timeFrame: Baseline, 24 weeks
measure: Change from baseline in reticulocyte count
timeFrame: Baseline, 24 weeks
measure: Change from baseline in LDH
timeFrame: Baseline, 24 weeks
measure: Change from baseline in Indirect bilirubin
timeFrame: Baseline, 24 weeks
measure: Change from baseline in free hemoglobin
timeFrame: Baseline, 24 weeks
measure: Proportion of participants without requiring red blood cells (RBC) transfusions
timeFrame: From week 4 to week 24
measure: Change in the average number of RBC transfused per week
timeFrame: From week 4 to week 24
measure: Change from baseline in PNH RBC clone size
timeFrame: Baseline, 24 weeks
measure: Change from baseline in C3 fragment deposition on PNH RBC
timeFrame: Baseline, 24 weeks
measure: Change from baseline in FACIT-Fatigue score
timeFrame: Baseline, 24 weeks
measure: Incidence and severity of adverse events
timeFrame: 28 weeks
Other outcomes:
Locations:
city: Nanjing
country: China
facility: The First Affiliated Hospital of Nanjing Medical University
state: Jiangsu
Study officials:
References: