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