Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency

Related Clinical Trial
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiMZ Subjects ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals COPD Exacerbation Blood and Urine Biomarkers Study Alpha-1 Carrier Genomics Study Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype Study of ARO-AAT in Normal Adult Volunteers Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry Respreeza® Self-administration and Learning Program (AmAREtTI Study) Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Environment Effect on Six-Minute Walk Test Performance Alpha-1 Foundation DNA and Tissue Bank Alpha1-antitrypsin Deficiency Registry Alpha-1 Research Registry Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency Alpha-1 Coded Testing(ACT) Study Long-Term Follow-up Study of ADVM-043 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation” EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency Alpha-1 Foundation Research Registry Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients Evaluate Efficacy and Safety of “Kamada-AAT for Inhalation” in Patients With AATD A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 – Proteinase Inhibitor) Aralast alpha1-proteinase Inhibitor Surveillance Study Pharmacokinetic Study of ARALAST (Human Alpha1- PI) The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

Brief Title

Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency

Official Title

A Stage 1, Prospective, Randomized, Placebo-Controlled, Double- Blind Study to Evaluate the Safety and Efficacy of Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Subjects With A1PI Deficiency and Chronic Obstructive Pulmonary Disease (COPD)

Brief Summary

      The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of
      weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects
      with A1PI deficiency and emphysema/ chronic obstructive pulmonary disease (COPD).
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Rate of Change in Lung Density Based on Group 1 (ARALAST NP) Versus Placebo, Group 3 and Group 4 (GLASSIA) Versus Placebo

Secondary Outcome

 Rate of Change in Lung Density for Each Treatment Group

Condition

Chronic Obstructive Pulmonary Disease

Intervention

ARALAST NP 60 mg/kg

Study Arms / Comparison Groups

 ARALAST NP 60 mg/kg
Description:  60 mg/kg body weight/week

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

7

Start Date

November 2, 2016

Completion Date

September 14, 2018

Primary Completion Date

September 14, 2018

Eligibility Criteria

        Inclusion Criteria:

          1. ≥18 years of age at the time of screening

          2. Endogenous plasma Alpha1-Proteinase Inhibitor (A1PI) level <8 μM at any time during
             the Screening period for treatment-naïve participants, or following 4-weeks minimum
             wash-out from previous augmentation therapy in treatment-experienced participants. The
             screening plasma A1PI level may be repeated if a participant obtains an exclusionary
             value that is suspected to be due to inadequate washout of A1PI).

          3. Participant has documented A1PI genotype of Pi*Z/Z, Pi*Z/Null, Pi*Malton/Z,
             Pi*Null/Null, or other rare genotypes (except PI*MS, PI*MZ, or PI*SZ).

          4. Clinically evident mild-moderate chronic obstructive pulmonary disease (COPD)
             (according to GOLD criteria for diagnosis) at the time of screening.

          5. If the participant is treated with any respiratory medications including inhaled
             bronchodilators, inhaled corticosteroids, or systemic corticosteroids (e.g. prednisone
             ≤ 10 mg/day or its equivalent), the doses of the participant's medications have
             remained stable for at least 28 days prior to screening.

          6. No clinically significant abnormalities (other than emphysema, bronchitis or
             bronchiectasis) detected via a chest computed tomography (CT) or chest X-ray at the
             time of screening.

          7. If female of childbearing potential, participant must have a negative pregnancy test
             at screening and agree to employ adequate birth control measures for the duration of
             the study.

          8. Participant is willing and able to comply with the requirements of the protocol.

        Exclusion Criteria:

          1. Known ongoing or history of clinically significant pulmonary impairment other than
             emphysema/ COPD.

          2. The participant is experiencing lower respiratory infection (LRTI)/acute pulmonary
             exacerbation (APE) at the time of enrollment (signing Informed consent form (ICF)).
             Participant may be rescreened after both clinical resolution of LRTI/APE and having
             also remained stable for at least 4 weeks after the end of LRTI/APE).

          3. Known ongoing or history of cor pulmonale.

          4. Known resting partial pressure of carbon dioxide (PaCO2) levels of > 45 mmHg.

          5. Clinically significant congestive heart failure with New York Heart Association (NYHA)
             Class III/IV symptoms.

          6. The participant has received an organ transplant, has undergone major lung surgery, or
             is currently on a transplant list.

          7. Known history of ongoing malignancy (other than adequately treated basal cell or
             squamous cell carcinoma of the skin or carcinoma in situ of the cervix).

          8. Smoker or participant that has ceased smoking for less than one year prior to
             screening whose levels of cotinine are outside of the normal range of a nonsmoker.

             All participants must agree to refrain from smoking throughout the course of the
             study.

          9. The participant is receiving long-term therapy (> 28 days) of parenteral
             corticosteroids or oral corticosteroids at doses greater than 10 mg/day of prednisone
             or its equivalent).

         10. The participant is receiving long-term round-the-clock oxygen supplementation (other
             than temporary for acute COPD exacerbation, or supplemental oxygen (O2) with
             continuous positive airway pressure [CPAP], or bi-level positive airway pressure
             [BiPAP] during the day).

         11. Participant has contraindications for CT (e.g. body weight and/or body size exceeding
             the weight and gantry size limits specified by the manufacturer of the CT scanner,
             inability to lie flat in the CT scanner, claustrophobia, metal prosthesis or pacemaker
             in the chest wall or upper extremity that would impact lung density assessment).

         12. Participant is unwilling or unable to modify bronchodilator medications for 6 hours
             for short acting β2 agonists, 24 hours for long-acting β2 agonists, and 48 hours for
             long acting anticholinergics prior to the scheduled quantitative CT scan.

         13. Known severe immunoglobulin A (IgA) deficiency (ie, IgA level < 8 mg/dL at screening).

         14. Known history of hypersensitivity following infusions of human blood or blood
             components (eg, human immunoglobulins or human albumin).

         15. Presence of clinically significant laboratory abnormalities at the screening

         16. The participant has a clinically significant medical, psychiatric, or cognitive
             illness, is a recreational drug/alcohol user, or has any other uncontrolled medical
             condition (eg, unstable angina, transient ischemic attack, uncontrolled hypertension)
             that, in the opinion of the investigator, would affect participant's safety or
             compliance or confound the results of the study.

         17. Participant has been exposed to another IP within 28 days prior to enrollment or is
             scheduled to participate in another clinical study involving an IP or investigational
             device during the course of this study.

         18. Participant is a family member or employee of the investigator.

         19. If female, participant is pregnant or nursing at the time of enrollment.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02722304

Organization ID

460503


Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Collaborators

 Baxalta Innovations GmbH, now part of Shire

Study Sponsor

Study Director, Study Director, Shire


Verification Date

October 2019