Phase II/III study of Kamada-AAT for inhalation

• Research type

  Research Study

• Full title

  A Phase II/III Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients with Emphysema

• IRAS ID

  29096

• Sponsor organisation

  Kamada Ltd

• Eudract number

  2008-005326-36

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  Alpha-1 Antitrypsin deficiency is an inherited disease characterised by the lack of a protein in the blood called Ó?Alpha-1 Antitrypsin? (AAT). AAT is distributed to the lungs to control neutrophil elastase, which is an enzyme that breaks down elastin, a protein that's essential for proper pulmonary function. In patients with AAT deficiency, neutrophil elastase can accumulate and destroy elastin in the lungs, and as a result the patients can develop emphysema. AAT deficiency is a debilitating disease that's long lasting and can be life-threatening. This study investigates the use of human AAT that will be inhaled by the patient as a new therapy for patients with emphysema secondary to AAT deficiency. Patients with AAT deficiency and clinical evidence of emphysema will be screened for eligibility. If they meet the entry criteria, patients will be randomised equally to either treatment group (??Kamada-AAT for inhalation?) or placebo group. 80 mg of ??Kamada-AAT for inhalation? or placebo will be administered twice daily for a period of 50 weeks starting from baseline, week 0. Patients will be followed for safety and efficacy until the final visit at week 54. Patients will attend the Investigator's clinic for physical examinations, vital sign measurements, clinical laboratory testing, pulmonary function tests, ECG recording and the recording of concomitant medications and adverse events. Patients will be given an electronic diary to record on a daily basis; breathlessness, well-being, sputum volume and colour and concomitant medications. Patients will also be telephoned every two weeks to evaluate overall well-being, adverse events, concomitant medications and electronic diary completion compliance. Patients will be asked to complete the St George??s Respiratory Questionnaire to assess quality of life at the start and end of the study. Safety will be evaluated using the examinations and tests listed above.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  09/H0408/103

• Date of REC Opinion

  2 Nov 2009

• REC opinion

  Favourable Opinion