A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT01983241Tipo: INTERVENTIONALInicio: 1 de nov de 2013Fin estimado: 1 de ene de 2027

Traducción no disponible, mostrando original

Resumen

This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 70 YearsSexo: ALL

Criterios de inclusión

Have a documented total alpha1-PI serum level \< 11 µM.
Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles.
At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in 1 second (FEV1) ≥ 30% and \< 80% of predicted and FEV1/forced vital capacity (FVC) \< 70% (Global Initiative for Chronic Obstructive Lung Disease \[GOLD\] stage II or III).
Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator's judgment.
Have clinical evidence of pulmonary emphysema per the Investigator's judgment.

Criterios de exclusión

Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening Visit.
Has received alpha1-PI augmentation therapy within one month of the Screening Visit.
Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
History of lung or liver transplant.
Any lung surgery during the past 2 years (excluding lung biopsy).
On the waiting list for lung surgery, including lung transplant.
Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes.
History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase.
Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
Known selective or severe Immunoglobulin A (IgA) deficiency.

Intervenciones

biological

Alpha-1 MP

other

0.9% Sodium Chloride for Injection, USP

Ubicaciones

Grifols Investigative Site

Vicente López, Buenos Aires, Argentina

Grifols Investigative Site

Córdoba, Córdoba Province, Argentina

Patrocinadores

PrincipalGrifols Therapeutics LLC

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