PNH - Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
This is a multicenter, randomized, open-label, Phase II clinical study to evaluate the efficacy, safety, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Interventions
BIOLOGICALLP-005 Injection
IV infusion, Q4W
Sponsors
Longbio Pharma
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Male or female, aged 18 to 65 years. * Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes. * Presence of one or more PNH-related signs or symptoms within 3 months prior to screening or a history of transfusion due to PNH. * LDH level ≥2.0×upper limit of the normal range(ULN). * Hemoglobin level \<100 g/L at screening.
Exclusion criteria
* Active or suspected active viral, bacterial, fungal, or parasitic infection within 14 days prior to screening. * History of meningococcal infection. * History of splenectomy or congenital asplenia. * History of systemic autoimmune disease or known/suspected immunodeficiency. * History of hematopoietic stem cell transplantation. * Use of any complement inhibitor within 3 months prior to screening or within 5 drug half-lives (whichever is longer). * Pregnant or breastfeeding women, or women planning to become pregnant during the study or follow-up period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Baseline in serum lactate dehydrogenase (LDH) levels. | Baseline and at Week 12. |
| Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion. | Baseline and at Week 24. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in the number of red blood cell (RBC) transfusions. | Baseline to Week 24 and 48. | — |
| Change from Baseline in hemoglobin levels. | Baseline, Week 12, 24 and 48 | — |
| Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion. | Baseline, Week 12, 24 and 48 | — |
| Proportion of participants with LDH levels normalized. | Baseline, Week 12, 24 and 48 | — |
| Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion. | Baseline and at Week 12 and 48 | — |
| Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) | Baseline and at Week 12, 36, and 48. | FACIT-F is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function. The higher the score, the better the QOL. |
| Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). | Baseline up to approximately 13 months. | — |
| Serum concentrations of LP-005. | Observation from Predose on Day 1 through 337 days post-administration. | — |
| Number of Participants with Treatment-emergent Anti-Drug Antibodies (ADA) and neutralizing antibodies (Nab) Response to LP-005. | Observation from Predose on Day 1 through 337 days post-administration. | — |
| Changes in serum complement hemolytic activity. | Observation from Predose on Day 1 through 337 days post-administration. | — |
| Proportion of participants who are transfusion-free. | Baseline to Week 12, 24 and 48 | — |
Countries
China
Outcome results
None listed