Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Alpha-1 15% · 2 trials · 2 indications
AE that occurred at any time from the beginning of IP administration until the final visit of the clinical trial was considered TEAE.
All noxious and unintended responses to a medicinal product or study treatment related to any dose was considered suspected ADRs. The phrase "responses to a medicinal product" means that a causal relationship between a medicinal product or study treatment and an AE is at least a reasonable possibility.
Treatment-emergent ISRs have onset on or after date of the first administration of study treatment up to on or before 28 days after last administration of study treatment. An ISR was counted in the period during which it started.
An adverse event is considered serious if, in the judgment of the investigator or sponsor, it results in death, is life-threatening, requires hospitalization or prolongs an existing hospitalization (admission for 24 hours or more, including emergency room stays), causes significant or persistent disability, results in a congenital anomaly or birth defect, or is an important medical event that may jeopardize the subject or require intervention to prevent these outcomes.
TEAE Leading to Study Withdrawal refers to a TEAE that results in a participant permanently discontinuing or withdrawing from a clinical study because of the adverse event.
The definition of an exacerbation of COPD is an increase in respiratory symptoms (dyspnea, increased cough, and/or production of sputum) over baseline that usually requires medical intervention.
Clinically significant vital sign abnormalities were presented as adverse events, with clinical relevance based on the Investigator's judgment.
FEV1 was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
FVC was performed according to ATS/ERS guidelines. At each visit, testing was performed both pre- and post-bronchodilator (BD) administration.
Out of the normal range laboratory results judged by the Investigator as clinically significant in the context of the participant's medical history were considered adverse events.
Immunogenicity testing was performed using validated assays in a multi-tiered approach.
| Arm | Type | Description |
|---|---|---|
| Treatment Arm 1 (60 mg/kg/week of Liquid Alpha1-PI followed by 90 mg/kg/week of Alpha-1 15%) | EXPERIMENTAL | 8 weeks of IV treatment with 60 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 90 mg/kg/week Alpha-1 15% |
| Treatment Arm 2 (120 mg/kg/week of Liquid Alpha1-PI followed by 180 mg/kg/week of Alpha-1 15%) | EXPERIMENTAL | 8 weeks of IV treatment with 120 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 180 mg/kg/week Alpha-1 15% |
| Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg) | EXPERIMENTAL | Participants will receive Alpha-1 15% 72 mg/kg, single subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1. |
| Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg) | EXPERIMENTAL | Following the week-long treatment period 1, participants in Cohort 1 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1- Proteinase Inhibitor (PI) 60 mg/kg, weekly intravenous (IV) Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. |
| Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg) | EXPERIMENTAL | Following treatment period 1 and single-dose data evaluation period, participants in Cohort 1 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 72 mg/kg, for 8 weekly SC infusions. |
| Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg) | EXPERIMENTAL | Participants will receive Alpha-1 15% 180 mg/kg, single SC infusion in treatment-period 1 (Single-Dose) at Week 1. |
| Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg) | EXPERIMENTAL | Following the week-long treatment period 1, participants in Cohort 2 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation period, Liquid Alpha1-PI 120 mg/kg, weekly IV Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose. |
| Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg) | EXPERIMENTAL | Following treatment period 1 and single-dose data evaluation period, participants in Cohort 2 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 180 mg/kg, for 8 weekly SC infusions. |
| Name | Type | Description |
|---|---|---|
| Alpha-1 15% | DRUG | Alpha1-Proteinase Inhibitor Subcutaneous (Human), 15% |
| Liquid Alpha1-PI | OTHER | Liquid Alpha1-Proteinase Inhibitor (Human) for Intravenous infusion |
| Liquid Alpha1-Proteinase Inhibitor (Human) | BIOLOGICAL | Intravenous infusion |
Inclusion Criteria: * Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (patients with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a ...
Alpha-1 15% is an investigational therapy being developed for Alpha-1 Antitrypsin Deficiency, also known as Alpha1-Antitrypsin Deficiency. It is a small molecule product intended to address this condition, which is characterized by low levels of the SERPINA1 protein. The drug is currently in clinical development and has not been approved by regulatory authorities.
Alpha-1 15% targets SERPINA1, which is the gene that encodes alpha-1 antitrypsin, a protein that protects tissues from enzymes released by immune cells. In Alpha-1 Antitrypsin Deficiency, mutations in SERPINA1 lead to reduced or dysfunctional alpha-1 antitrypsin, contributing to lung and liver damage. Alpha-1 15% is designed to address this deficiency.
Alpha-1 15% is being developed by Grifols, S.A., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety, tolerability, and pharmacokinetics of this investigational therapy for Alpha-1 Antitrypsin Deficiency.
Alpha-1 15% is in Phase 3 clinical development. A Phase 3 study is currently recruiting participants to evaluate the non-inferiority of two different doses of weekly subcutaneous Alpha1-PI 15% compared with standard intravenous Alpha1-PI in individuals with Alpha1-Antitrypsin Deficiency. The drug remains investigational and is not yet approved.
Alpha-1 15% is being studied in two clinical trials. NCT07555483 is a Phase 3, randomized, controlled, non-inferiority study with 40 participants, currently recruiting in the United States, Denmark, Germany, Ireland, Netherlands, Poland, Portugal, Spain, and Sweden. NCT04722887 is a completed Phase 1 study that evaluated safety, tolerability, and pharmacokinetics in 17 participants in the United States.
Yes, Alpha-1 15% is also referred to as Alpha1-PI 15%, which stands for Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15%. This name is used in clinical trial documentation to describe the same investigational product being developed by Grifols for Alpha-1 Antitrypsin Deficiency.