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Clinical Trials/EUCTR2019-000602-30-SE
EUCTR2019-000602-30-SEActive, not recruitingPhase 1

A Prospective Phase III Multi-center, 2-Year Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% = FEV1 = 80% of predicted; FEV1/SVC = 70%), Followed by a 2-Year Open-Label Extension - Phase III, Efficacy and Safety of Kamada-AAT for Inhalation

Kamada Ltd.0 sites220 target enrollmentStarted: April 24, 2020Last updated:
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Conditions

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
220
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional clinical trial of medicinal product

Eligibility Criteria

Sex
All

Inclusion Criteria

  • •Double-Blind Period
  • •1. Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes.
  • •2. Serum AAT levels = 11 µM at screening.
  • •3. Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC=70%) at screening.
  • •4. 40% = FEV1 = 80% of predicted post-bronchodilator at screening.
  • •5. Patients who are either naïve or washed out of any AAT treatment for at least 8 weeks prior to randomization.
  • •6. Age between 18 to 65 years inclusive at screening.
  • •7. Able to read and sign informed consent and willing to participate in the study.
  • •8. Males or non-pregnant, non-lactating females whose screening pregnancy test is negative, who are using contraceptive methods deemed reliable by the investigator, who are post-menopausal, or are surgically sterilized.
  • •9. Study medication use for at least 20 out of the 28 days of run-in, as recorded in the study nebulization PARI Track data.
  • •10. Demonstrated ability to complete eDiary for at least 20 out of the first 28 days of run-in.
  • •Open-Label Period
  • •1. Patients who completed 104 weeks of DB study treatment and attended the end of treatment visit.
  • •2. Patients who completed the DB period and attended follow-up visits are eligible for the OLE provided that they comply with all other OLE eligibility criteria.
  • •3. Consenting to continue study participation in the OLE phase.
  • •4. Agree to continue using contraceptive methods deemed reliable by the investigator for an additional 2 years, unless post-menopausal or surgically sterilized.
  • •Are the trial subjects under 18? no
  • •Number of subjects for this age range:
  • •F.1.2 Adults (18-64 years) yes
  • •F.1.2.1 Number of subjects for this age range 220
  • •F.1.3 Elderly (>=65 years) no
  • •F.1.3.1 Number of subjects for this age range

Exclusion Criteria

  • •Double-Blind Period
  • •1. Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels<0.05 g/L at screening.
  • •2. History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.
  • •3. Two or more moderate or any severe exacerbation(s) within the year prior to the baseline visit.
  • •4. A moderate exacerbation within 6 weeks prior to the baseline.
  • •5. Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).
  • •6. Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic, immunological, skeletal, or other. Patients might be included after consultation with the treating physician and the sponsor if, in the opinion of the Investigator, their condition will not interfere with the safety, compliance or other aspects of this study.
  • •7. Hospitalization for any cause 6 weeks prior to screening.
  • •8. History of lung or liver transplant.
  • •9. On any thoracic or hepatic surgery waiting list.
  • •10. Any lung surgery within the past two years (including bronchoscopic lung volume reduction).
  • •11. Any smoking within the year prior to screening.
  • •12. Evidence of alcohol abuse or history of alcohol abuse, or use of illegal drugs and/or abuse of legally prescribed drugs in the last 5 years prior to screening.
  • •13. Acute or chronic hepatitis (hepatitis A, hepatitis B, hepatitis C,) or positive human immunodeficiency virus (HIV) serology.
  • •14. Signs of significant abnormalities in serum hematology, serum chemistry, serum inflammatory / immunogenic markers and urinalysis per investigator judgment, taking into considerations the potential effects of the AAT deficiency.
  • •15. Signs of significant abnormalities in ECG per investigator judgment at screening.
  • •16. Presence of psychiatric/ mental disorder or any other medical disorder that might impair the patient’s ability to give informed consent or to comply with the requirements of the study protocol. If, in the opinion of the Investigator, the condition will not interfere with the compliance or other aspects of this study, the patient might be included after consultation with the treating physician and the sponsor.
  • •17. Participation in another clinical trial involving investigational medication or interventional treatment within 30 days and/or last dose 5 half-lives prior to screening visit.
  • •18. Inability to attend scheduled clinic visits and/or comply with study protocol.
  • •19. Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol.
  • •Open-Label Period
  • •1. Any adverse event(s) in the DB period and/or medical condition that, in the opinion of the investigator, might prevent the patient from safely participating in the OLE period of the study, including but not limited to:
  • •a. Occurrence of a life-threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
  • •b. Received lung transplant, entered a waiting list for lung transplantation, or underwent lung surgery.
  • •The investigator should consult the sponsor before inclusion of any patient with a significant condition if the investigator believes that it will not pose an unacceptable risk for the patient.
  • •2. Evidence of alcohol abuse or history of

Investigators

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