Pharma Focus Europe

Grifols Successfully Enrolls Participants in Phase 3 Study Evaluating Two Dose Regimens of Prolastin®-c for Emphysema Caused by Alpha-1-antitrypsin Deficiency

Friday, July 14, 2023

Grifols, a major producer of plasma-derived medications, has announced that it has successfully enrolled 339 patients in its phase 3 clinical trial called SPARTA (Study of ProlAstin-c Randomized Therapy with Alpha-1 augmentation; NCT01983241). The trial aims to assess whether patients with emphysema and alpha-1 antitrypsin (AAT) deficiency experience a slower progression of lung tissue loss when treated with different doses of Grifols Prolastin®-C on a weekly basis.

Alpha-1 is a genetic disorder that is often underdiagnosed and can lead to chronic obstructive pulmonary disease (COPD), including emphysema, due to low levels of the protective protein alpha-1 antitrypsin (AAT) in the lungs. The current approved dosage for AAT augmentation therapy is 60 mg/kg administered weekly.

SPARTA is the largest randomized, double-blind, placebo-controlled study on AAT augmentation therapy to date. The trial is being conducted across 16 countries and over 50 sites. Its objective is to evaluate the potential of Prolastin®-C to significantly reduce emphysema progression in alpha-1 patients by increasing AAT protein levels through weekly administration of two active dose levels compared to a placebo.

The study will span 156 weeks (approximately three years) and will assess the efficacy and safety of two separate dose regimens of Prolastin®-C (60 and 120 mg/kg/week) compared to a placebo. The primary measure of clinical efficacy will be the rate of pulmonary tissue loss measured using whole lung computed tomography (CT) densitometry.

Sandra Camprubi, Grifols Senior Director of Clinical Operations, expressed the hope that the study will provide clinical evidence of the benefits of the current approved dose and the potential greater impact of doubling the single dose to 120 mg/kg weekly. Topline data from the study is expected to be available in 2026, and based on the results, Grifols will evaluate the next regulatory steps to offer impactful treatment options for alpha-1 patients with emphysema.

Grifols is dedicated to supporting the alpha-1 community and has a strong commitment to innovation. Earlier this year, the company launched the AlphaID™ At Home Genetic Health Risk Service (AlphaID™ At Home), a free direct-to-consumer program in the United States that screens for the genetic risk of alpha-1.

Alpha-1-antitrypsin deficiency, also known as alpha-1, is a rare genetic disease that can lead to chronic obstructive pulmonary disease (COPD), including emphysema. Individuals with alpha-1 have a genetic deficiency of alpha-1 antitrypsin, a plasma protein that protects the lungs from inflammation caused by infection and inhaled irritants like tobacco smoke. Alpha-1 is the primary known genetic risk factor for COPD.

Prolastin®-C is an alpha1-proteinase inhibitor (human) (alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with emphysema caused by severe hereditary deficiency of alpha1-PI (alpha-1-antitrypsin deficiency).

It is important to note that PROLASTIN®-C is contraindicated in patients deficient in immunoglobulin A (IgA) with antibodies against IgA or patients who have a history of severe systemic reactions or anaphylaxis to alpha1-PI products. Hypersensitivity reactions, including anaphylaxis, may occur. Patients should be closely monitored during infusion, and if hypersensitivity symptoms arise, the infusion should be stopped immediately, and appropriate treatment initiated. Since PROLASTIN®-C is derived from human plasma, there is a risk of transmitting infectious agents, such as viruses and theoretical agents like the variant Creutzfeldt-Jakob disease (vCJD) and Creutzfeldt-Jakob disease (CJD). This also includes unknown or emerging viruses and pathogens. The most common drug-related adverse reaction observed in subjects receiving PROLASTIN®-C during clinical trials was upper respiratory tract infection, while the most serious adverse reaction reported was an abdominal and extremity rash in one subject.

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