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The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.
This is an open-label, single-dose, dose-escalation study to assess the safety, tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC injection in subjects with PNH or evidence of circulating PNH cells.
Eligible subjects with PNH will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated or if revaccination is required).
On study day 1, the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as a SC injection, and be followed with additional study evaluations over the next 59 days.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TT30 (ALXN1102 Formulation) | Experimental | IV: 0.1, 0.3, and 1.0 mg/kg |
|
| TT30 (ALXN1103 Formulation) | Experimental | IV: 3.0, 6.0, and 10.0 mg/kg SC: 1.0 and 3.0 mg/kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN1102 | Drug | Single Ascending Dose IV |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Assess the safety and tolerability of a single dose of TT30. | Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development. | 60 days |
| Measure | Description | Time Frame |
|---|---|---|
| Characterize PK, PD and immunogenicity of a single dose of TT30. | Immunogenicity will be will be assessed using standard measures for these parameters. PK and PD will be assessed by using standard measures, including reticulocyte count and lactate dehydrogenase (LDH) levels. | 60 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bill Lundberg, MD | Alexion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USC/Norris Comprehensive Cancer Center | Los Angeles | California | 90033 | United States | ||
| Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze |
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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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| ALXN1103 |
| Drug |
Single Ascending Dose IV or SC |
|
| U Nemocnice 1 |
| Prague 2 |
| 128 08 |
| Czechia |
| Federico II University of Naples | Via Pansini 5 | Naples | 80131 | Italy |
| University Clinical Centre | Dębinki | Gdańsk | 80-952 | Poland |
| King's College Hospital NHS Foundation Trust | Denmark Hill | London | SE5 9RS UK | United Kingdom |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |