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CompletedNCT00499837Updated Jun 9, 2016

Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients

A Phase 2 interventional study of Aerosolized, human, plasma-derived Alpha-1 Antitrypsin in Cystic Fibrosis, sponsored by Kamada, Ltd.. Completed at 1 site in Israel. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2016-06-09.

Sponsored by Kamada, Ltd. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
21
Allocation
Randomized
Ages
5 Years and older
Sex
All
01

Study summary

Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways.

The purpose of this study is to collect data about the resolution of the chronic inflammatory state in addition to assure the safety of the therapy in CF patients.

Read the detailed description

In CF patients the unregulated inflammatory response overwhelms the normal protease (elastase)/antiprotease (AAT) balance, leading to the accumulation of elastase in the lung, destruction of the lung architecture, severe pulmonary dysfunction, and ultimately death.

Administration of AAT is to address the elastase/antiprotease imbalance in order to prevent destruction of the lung tissue and reduce the inflammatory dysregulation that causes pulmonary dysfunction.

02

Conditions studied

  • Cystic Fibrosis
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 21 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Kamada, Ltd. is the lead sponsor of 10 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Diagnosis of CF by clinical symptoms and positive sweat test or disease inducing mutation.
  • Age >5 yrs
  • Proven ability to perform reproducible PFTs
  • FEV1 >25% predicted
  • Steady disease state for 3 months and no decrease in lung function exceeding 10% during that period
  • Colonization
  • Stable concomitant therapy >2 weeks prior to visit 1 and during the study
  • Non-tobacco user of any kind
  • Ability for sputum induction
  • Written informed consent

Exclusion criteria

Exclusion Criteria:

  • Severe CF with an FEV1 of \<25% predicted
  • History of lung transplant
  • Active allergic bronchopulmonary aspergillosis (ABPA)
  • Treatment with additional antibiotics (beyond standard CF treatment) for a period of 14 days before study entry (routine antibiotics permitted)
  • Treatment with additional oral and/or IV steroids (beyond standard CF treatment) for a period of 14 days before study entry (screening day)
  • Known hypersensitivity to plasma products
  • IgA deficiency
  • Uncontrolled hypertension
  • Lung surgery in the previous two years
  • Being on any thoracic surgery waiting list
  • Severe concomitant disease
  • Hospitalization within 1 month before study entry, not due to an airway disease
  • Severe liver cirrhosis with ascites
  • Hypersplenism
  • Grade III/IV oesophageal varices
  • Active pulmonary exacerbation within the 4 weeks prior to screening
  • History of significant hemoptysis within the previous year
  • Use of tobacco products or recreational drugs
  • Pregnancy or breastfeeding
  • Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would interfere with patient treatment, assessment, or compliance with the protocol.
  • Being a female of child-bearing age without adequate contraception
  • Participation in research study within 1 month
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    80 mg/kg AAT inhaled

    80 mg/kg AAT inhaled

    Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

  • Placebo comparator
    Placebo inhaled

    Placebo inhaled

    Drug: Aerosolized, human, plasma-derived Alpha-1 Antitrypsin

Interventions

  • DrugAerosolized, human, plasma-derived Alpha-1 Antitrypsin
06

What researchers measure

Primary outcomes

  1. Safety and airway inflammation

    Time frame: days 1,7,14,21,28,35,42,49,56,63

Secondary outcomes

  1. Sputum microbiology, pulmonary function and serum CRP

    Time frame: days 1,7,14,21,28,35,42,49,56,63

07

Study locations

1 site
  • Hadassah Hebrew University, Medical Center
    Jerusalem, 91240, Israel
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 9, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00499837
Lead sponsor
Kamada, Ltd.
Responsible party
Sponsor
First posted
Jul 12, 2007
Start date
Sep 2007
Primary completion
Jul 2008
Completion
Jul 2008
Last update
Jun 9, 2016

Study contacts

Eitan Kerem, MD
principal investigator · Hadassah Hebrew University, Medical Center, Mt. Scopus, Jerusalem

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2016. You cannot join it, but the record below documents what was studied.

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