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

Alpha 1-Antitrypsin Deficiency Clinical Trial

— Kamada API  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00460096
• Other study ID #: Kamada API-002
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: April 12, 2007
• Last updated: October 16, 2007
• Start date: March 2007
• Est. completion date: September 2007

Study information

• Verified date: October 2007
• Source: Kamada, Ltd.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: September 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Intervention

Drug:
• Kamada-API

Locations

Country	Name	City	State
United States	National Jewish Medical and Research Center	Denver	Colorado
United States	University of Florida School of Medicine	Gainesville	Florida
United States	The University of Texas Health Center at Tyler	Tyler	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Kamada, Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy
Secondary	Safety