Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency

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Brief Title

Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency

Official Title

Randomized Double-Blind Comparison of an Alpha-1 Proteinase Inhibitor (Kamada API) With a Currently Marketed API Product in Individuals With Alpha-1 Antitrypsin Deficiency

Brief Summary

      The primary purpose of this Phase II/III study is to demonstrate that Kamada-API, a new API
      concentrate manufactured by Kamada Ltd., is comparable to a currently marketed API product.
    

Detailed Description

      Alpha-1 Antitrypsin Deficiency, also called Alpha-1-Proteinase Inhibitor (API) deficiency, is
      a genetic disorder characterized by the production of an abnormal amount of AAT protein and
      reduced circulating levels of this protein. Subjects with AAT deficiency are at increased
      risk for developing chronic obstructive pulmonary disease (COPD). It is believed that this is
      the result of the chronic activity of elastase released by cells continually present in the
      lungs in low numbers.

      This study is a randomized, double-blind comparison of Kamada API, an Alpha-1-Proteinase
      Inhibitor with a currently marketed API product.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Efficacy

Secondary Outcome

 Safety

Condition

Alpha 1-Antitrypsin Deficiency

Intervention

Kamada-API


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

Drug

Estimated Enrollment

50

Start Date

March 2007

Completion Date

September 2007


Eligibility Criteria

        Inclusion Criteria:

          1. Signed informed consent.

          2. "At-risk" alleles associated with serum AAT < 11 μM including null alleles and
             deficiency alleles. This must be documented in the subject's history or laboratory
             tests performed at screening.

          3. At least 18 years of age.

          4. Evidence of lung disease related to AAT deficiency, identified by at least one of the
             following:

               -  FEV1<80% predicted (post BD); or

               -  Loss of lung function over a one year period of greater than 35ml in FEV1; or

               -  HRCT evidence of pulmonary emphysema

          5. For actively treated subjects, agreement to not receive any exogenous API product
             (i.e. washout) for five weeks prior to first study infusion.

          6. Use of an effective means of contraception during the 24 weeks of study drug
             administration (this is applicable to both sexes).

          7. Subjects on the BAL, bronchial brushing/biopsy group must be on inhaled
             corticosteroids at a stable dose two weeks prior the first Bronchoscopy and throughout
             the dosing period up the final bronchoscopy.

        Exclusion Criteria:

          1. Laboratory evidence of severe IgA deficiency (from medical history or by IgA testing
             at screening of at least 20% of lower range).

          2. Current smoker or a history of smoking within the past 3 months.

          3. History of allergy to plasma proteins.

          4. Participation in another experimental drug or device trial within the past 30 days.

          5. Evidence of uncontrolled hypertension (systolic ≥180 mm Hg, and/or diastolic ≥ 110 mm
             Hg on 3 consecutive occasions in the supine position)

          6. Pulse ≥ 120/min (prior to the 1st infusion).

          7. Abnormal screening or baseline laboratory measurements that in the opinion of the
             Investigator would affect subject safety.

          8. Pregnancy or lactation.

          9. Current life-threatening malignancy.

         10. Previous organ transplant recipient.

         11. History of infection with HCV, HBV and/or HIV 1 or 2, or (at baseline) infection
             indicated by laboratory measurements obtained at screening.

         12. Acute respiratory tract infection or COPD exacerbation which required antibiotic
             and/or systemic steroid treatment within the past 6 weeks. Patient can be re-evaluated
             for enrollment 6 weeks after an exacerbation.

         13. Any other condition which in the judgment of the investigator may interfere with the
             conduct of the study.

         14. If an adequate home health care agency cannot be established by Centric Health
             Resources due to a potential subject's geographical location.

        Exclusion criteria for subjects entering into the BAL and bronchial biopsy/brushing:

          1. FEV1 < 45% predicted (post-BD).

          2. Inability to undergo bronchoscopy.

          3. Allergy to lidocaine.

          4. Exacerbation of COPD in the previous 6 weeks.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Robert A Sandhaus, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00460096

Organization ID

Kamada API-002



Study Sponsor

Kamada, Ltd.


Study Sponsor

Robert A Sandhaus, M.D., Principal Investigator, National Jewish Health


Verification Date

October 2007