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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00460096
• Date of registration: 12/04/2007
• Prospective Registration: No
• Primary sponsor: Kamada, Ltd.
• Public title: Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Kamada API
• Scientific 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
• Date of first enrolment: March 2007
• Target sample size: 50
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00460096
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Treatment
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Robert A Sandhaus, M.D.
• Affiliation: National Jewish Health

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Alpha 1-Antitrypsin Deficiency

Intervention(s)

Drug: Kamada-API

Primary Outcome(s)

Efficacy

Secondary Outcome(s)

Safety

Secondary ID(s)

Kamada API-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors