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The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults. It will also learn about the safety, pharmacokinetic characteristics, and dosing of drug SLN12140.
The study is divided into four phases: screening period, core treatment period, extended dosing period, and follow-up period, and includes two cohorts (Cohorts 1-2), with each cohort enrolling at least 5 treatment-naïve adult PNH subjects for complement inhibitor therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SLN 12140 will be administered subcutaneously . | Experimental | 5 participants will receive SLN12140 100mg QW for 4 weeks, then 300mg QW for 8 weeks, then 200mg QW for 52 weeks. 5 participants will receive SLN12140 200mg QW for 4 weeks, then 600mg Q4W for 60 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SLN12140 | Drug | 5 participants will receive SLN12140 100mg QW for 4 weeks, then 300mg QW for 8 weeks, then 200mg QW for 52 weeks. 5 participants will receive SLN12140 200mg QW for 4 weeks, then 600mg Q4W for 60 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| During the 12-week treatment period, the proportion of participants whose Lactate Dehydrogenase (LDH) decreased by 60% or more from baseline or whose LDH was below the upper limit | To assess efficacy of SLN12140 in participants with PNH | 12weeks after baseline |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage change of LDH from baseline | To assess the efficacy of SLN12140 in participants with PNH | Baseline through Week 64 |
| Proportion of participants achieving hemolysis control (LDH ≤ 1.5×ULN) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Hong Yan Tong, Professor | Contact | 86+13958122357 | hongyantong@aliyun.com | |
| Feng Kui Zhang, Professor | Contact | 86+ | 13821700281 | fkzhang@ihcams.ac.cn |
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It is not decided yet at this moment if we will share IPD with other researchers, or when will IPD will be shared, or with whom will IPD be shared, or by what mechanism will IPD be shared.
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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To assess the efficacy of SLN12140 in participants with PNH
| Baseline through Week 64 |
| Change in hemoglobin (Hb) levels from baseline | To assess the efficacy of SLN12140 in participants with PNH | Baseline through week 64 |
| The proportion of participants whose hemoglobin (Hb) increased by ≥2 g/dL from baseline and who avoided blood transfusion | To assess the efficacy of SLN12140 in participants with PNH | Baseline through Week 64 |
| Proportion of participants who avoided blood transfusion | To assess the efficacy of SLN12140 in participants with PNH | Baseline through Week 64 |
| Incidence(%) of Breakthrough Hemolysis (BTH) | To assess the efficacy of SLN12140 in participants with PNH | Baseline through Week 64 |
| Changes from baseline in intravascular and extravascular hemolysis indicators (including but not limited to reticulocytes, bilirubin, red blood cell count, platelet count, ferritin, etc.) | To assess the efficacy of SLN12140 in participants with PNH | baseline through week 64 |
| Changes in thrombus formation risk markers from baseline (including but not limited to fibrinogen, prothrombin time, activated partial thromboplastin time, thrombin time, fibrin D-dimer, etc.); | To assess the efficacy of SLN12140 in participants with PNH | Baseline through week 64 |
| Change in functional assessment of Functional Assessment of Chronic Illness Therapy (FACIT) | To assess the efficacy of SLN12140 in participants with PNH. FACIT is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function to assess the Impact of SLN12140 on Treatment-Related Outcomes. The minimum value is 0 and maximum value is 52, and higher scores mean a worse outcome. | Baseline through Week 64 |
| Number(%) of participants with Adverse Events (AEs) , Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | To assess the safety and tolerability of SLN12140 in participants with PNH | Baseline through Week 64 |
| Pharmacokinetics (PK)parameters of SLN12140: Area Under The Plasma Concentration-time Curve | To characterize the pharmacokinetics of SLN12140 in participants with PNH | Baseline through week 64 (predose and postdose) |
| Immunegenicity in Paraxysmal Nocturnal Hemoglobinuria | Determine anti-drug antibody titers | Baseline through Week 64 |
| PK: Maximum Plasma Concentration (Cmax) | To characterize the pharmacokinetics of SLN12140 in participants with PNH | Baseline through week 64( predose and postdose) |
| PK: Time To Maximum Concentration (Tmax) | To characterize the pharmacokinetics of SLN12140 in participants with PNH | Baseline through week 64( predose and postdose) |
| Complement Alternative Pathway (AP) Functional Activity | Serum AP functional activity was measured by the Wieslab functional immunoassay method. | Baseline through week 64( predose and post dose) |
| Complement FP | Plasma FP was measured by enzyme-linked immunosorbent assay (ELISA). | Baseline through week 64(predose and postdose) |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |