ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 8 of 135 for:    Emphysema: Clinical Trials

Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00263887
Recruitment Status : Completed
First Posted : December 9, 2005
Results First Posted : August 21, 2014
Last Update Posted : August 21, 2014
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Alpha 1-Antitrypsin Deficiency
Interventions Drug: Alpha1-Proteinase Inhibitor (Human)
Drug: Albumin (Human) 20%, United States Pharmacopeia (USP)
Enrollment 77
Recruitment Details Multicenter study with 3 sites in Denmark, Sweden, and the United Kingdom.
Pre-assignment Details  
Arm/Group Title Prolastin (60 mg/kg Body Weight) Placebo
Hide Arm/Group Description [Not Specified] [Not Specified]
Period Title: Overall Study
Started 38 39
Completed 35 32
Not Completed 3 7
Reason Not Completed
Withdrawal by Subject             1             1
Lung transplantation             1             2
Too ill to attend visit             1             0
Adverse Event             0             2
Disease progression             0             1
Lack of Efficacy             0             1
Arm/Group Title Prolastin (60 mg/kg Body Weight) Placebo Total
Hide Arm/Group Description [Not Specified] [Not Specified] Total of all reporting groups
Overall Number of Baseline Participants 38 39 77
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants 39 participants 77 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
33
  86.8%
34
  87.2%
67
  87.0%
>=65 years
5
  13.2%
5
  12.8%
10
  13.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 38 participants 39 participants 77 participants
54.68  (8.29) 55.26  (9.68) 54.97  (9.03)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants 39 participants 77 participants
Female
13
  34.2%
23
  59.0%
36
  46.8%
Male
25
  65.8%
16
  41.0%
41
  53.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 38 participants 39 participants 77 participants
Denmark 18 17 35
United Kingdom 12 14 26
Sweden 8 8 16
Severity of COPD at baseline   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 38 participants 39 participants 77 participants
None 2 2 4
Mild 1 2 3
Moderate 10 8 18
Severe 19 20 39
Very Severe 6 7 13
[1]
Measure Description: Severity of Chronic Obstructive pulmonary Disease (COPD) at baseline according to Global Initiative for Chronic Obstructive Lung Disease (GOLD) classes
1.Primary Outcome
Title The Progression Rate of Emphysema Determined by Change in 15th Percentile of Lung Density Measured by Annual CT Scan of the Whole Lung
Hide Description [Not Specified]
Time Frame 24 or 30 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-To-Treat Population was defined as all subjects in the Intent-To-Treat (ITT) Population (all randomized subjects) who had a valid baseline CT scan and at least one valid post-baseline CT scan measurement.
Arm/Group Title Prolastin (60 mg/kg Body Weight) Placebo
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 35 32
Mean (Standard Deviation)
Unit of Measure: g/L
Baseline 47.980  (19.072) 45.477  (16.949)
Endpoint 45.085  (19.433) 41.354  (16.260)
Change from Baseline -2.895  (4.739) -4.124  (4.147)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Prolastin (60 mg/kg Body Weight), Placebo
Comments The main effect ANCOVA model includes the change from baseline to endpoint as the dependent variable, treatment and center as fixed factors, change in logarithm of total lung volume and baseline measurement as covariates.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.049
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
2.Secondary Outcome
Title Change in Lung Density at Each Visit as Measured by Computed Tomography
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
3.Secondary Outcome
Title The Frequency of Exacerbations as Determined by Patient Diary.
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
4.Secondary Outcome
Title The Deterioration of the Lung Function Will be Assessed by Measurement of the Change in Forced Expiratory Volume at One Second (FEV1) and Transfer Factor of Carbon Monoxide (KCO)
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Duration and Severity of the Exacerbations
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Mortality
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Quality of Life With a Disease Specific Instrument, the St. George's Respiratory Questionnaire
Hide Description [Not Specified]
Time Frame 24 or 30 months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Prolastin (60 mg/kg Body Weight) Placebo
Hide Arm/Group Description [Not Specified] [Not Specified]
All-Cause Mortality
Prolastin (60 mg/kg Body Weight) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Prolastin (60 mg/kg Body Weight) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   9/38 (23.68%)   15/39 (38.46%) 
Cardiac disorders     
Atrial fibrillation  2/38 (5.26%)  0/39 (0.00%) 
Gastrointestinal disorders     
Constipation  1/38 (2.63%)  0/39 (0.00%) 
Gastrooesophageal reflux disease  1/38 (2.63%)  0/39 (0.00%) 
Abdominal pain  0/38 (0.00%)  1/39 (2.56%) 
Intra-abdominal haemorrhage  0/38 (0.00%)  1/39 (2.56%) 
Rectal haemorrhage  0/38 (0.00%)  1/39 (2.56%) 
General disorders     
Nodule  0/38 (0.00%)  1/39 (2.56%) 
Hepatobiliary disorders     
Biliary colic  1/38 (2.63%)  0/39 (0.00%) 
Gallbladder disorder  1/38 (2.63%)  1/39 (2.56%) 
Jaundice cholestatic  0/38 (0.00%)  1/39 (2.56%) 
Infections and infestations     
Pneumonia  3/38 (7.89%)  4/39 (10.26%) 
Malaria  1/38 (2.63%)  0/39 (0.00%) 
Appendicitis  0/38 (0.00%)  1/39 (2.56%) 
Sepsis  0/38 (0.00%)  1/39 (2.56%) 
Subcutaneous abscess  0/38 (0.00%)  1/39 (2.56%) 
Urinary tract infection  0/38 (0.00%)  1/39 (2.56%) 
Injury, poisoning and procedural complications     
Upper limb fracture  1/38 (2.63%)  0/39 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  0/38 (0.00%)  1/39 (2.56%) 
Osteoarthritis  0/38 (0.00%)  1/39 (2.56%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Breast cancer  0/38 (0.00%)  1/39 (2.56%) 
Nervous system disorders     
Transient ischaemic attack  1/38 (2.63%)  0/39 (0.00%) 
Reproductive system and breast disorders     
Menorrhagia  1/38 (2.63%)  0/39 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Pneumothorax  2/38 (5.26%)  0/39 (0.00%) 
Epixtaxis  1/38 (2.63%)  0/39 (0.00%) 
Chronic obstructive pulmonary disease  0/38 (0.00%)  1/39 (2.56%) 
Dyspnoea  0/38 (0.00%)  1/39 (2.56%) 
Pleural effusion  0/38 (0.00%)  1/39 (2.56%) 
Pulmonary embolism  0/38 (0.00%)  2/39 (5.13%) 
Pulmonary oedema  0/38 (0.00%)  1/39 (2.56%) 
Skin and subcutaneous tissue disorders     
Psoriasis  1/38 (2.63%)  0/39 (0.00%) 
Lichen sclerosus  0/38 (0.00%)  1/39 (2.56%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Prolastin (60 mg/kg Body Weight) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   37/38 (97.37%)   38/39 (97.44%) 
Cardiac disorders     
Angina pectoris  2/38 (5.26%)  1/39 (2.56%) 
Eye disorders     
Conjunctivitis  0/38 (0.00%)  2/39 (5.13%) 
Gastrointestinal disorders     
Abdominal pain upper  3/38 (7.89%)  4/39 (10.26%) 
Diarrhoea  3/38 (7.89%)  5/39 (12.82%) 
Mouth ulceration  2/38 (5.26%)  0/39 (0.00%) 
Nausea  2/38 (5.26%)  4/39 (10.26%) 
Abdominal pain  1/38 (2.63%)  4/39 (10.26%) 
Gastritis  1/38 (2.63%)  3/39 (7.69%) 
Vomiting  0/38 (0.00%)  4/39 (10.26%) 
General disorders     
Pyrexia  3/38 (7.89%)  2/39 (5.13%) 
Chest pain  2/38 (5.26%)  5/39 (12.82%) 
Infusion related reaction  2/38 (5.26%)  0/39 (0.00%) 
Oedema  1/38 (2.63%)  2/39 (5.13%) 
Oedema peripheral  1/38 (2.63%)  5/39 (12.82%) 
Asthenia  0/38 (0.00%)  2/39 (5.13%) 
Fatigue  0/38 (0.00%)  4/39 (10.26%) 
Immune system disorders     
Drug hypersensitivity  2/38 (5.26%)  0/39 (0.00%) 
Hypersensitivity  2/38 (5.26%)  1/39 (2.56%) 
Seasonal allergy  2/38 (5.26%)  1/39 (2.56%) 
Infections and infestations     
Nasopharyngitis  24/38 (63.16%)  22/39 (56.41%) 
Pneumonia  12/38 (31.58%)  9/39 (23.08%) 
Sinusitis  5/38 (13.16%)  5/39 (12.82%) 
Influenza  4/38 (10.53%)  3/39 (7.69%) 
Tooth abscess  3/38 (7.89%)  5/39 (12.82%) 
Bronchitis  2/38 (5.26%)  1/39 (2.56%) 
Localised infection  2/38 (5.26%)  1/39 (2.56%) 
Oral candidiasis  2/38 (5.26%)  2/39 (5.13%) 
Urinary tract infection  2/38 (5.26%)  2/39 (5.13%) 
Gastroenteritis  1/38 (2.63%)  3/39 (7.69%) 
Eye infection  0/38 (0.00%)  2/39 (5.13%) 
Gastroenteritis viral  0/38 (0.00%)  2/39 (5.13%) 
Lower respiratory tract infection  0/38 (0.00%)  2/39 (5.13%) 
Pharyngitis  0/38 (0.00%)  2/39 (5.13%) 
Tooth infection  0/38 (0.00%)  2/39 (5.13%) 
Viral infection  0/38 (0.00%)  2/39 (5.13%) 
Injury, poisoning and procedural complications     
Fall  2/38 (5.26%)  0/39 (0.00%) 
Contusion  1/38 (2.63%)  5/39 (12.82%) 
Arthropod sting  0/38 (0.00%)  2/39 (5.13%) 
Investigations     
Blood potassium decreased  2/38 (5.26%)  1/39 (2.56%) 
Musculoskeletal and connective tissue disorders     
Back pain  5/38 (13.16%)  2/39 (5.13%) 
Arthralgia  3/38 (7.89%)  5/39 (12.82%) 
Monoarthritis  2/38 (5.26%)  0/39 (0.00%) 
Myalgia  2/38 (5.26%)  2/39 (5.13%) 
Pain in extremity  2/38 (5.26%)  6/39 (15.38%) 
Muscle spasms  1/38 (2.63%)  4/39 (10.26%) 
Joint swelling  0/38 (0.00%)  2/39 (5.13%) 
Musculoskeletal pain  0/38 (0.00%)  2/39 (5.13%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Melanocytic naevus  2/38 (5.26%)  1/39 (2.56%) 
Lung neoplasm  1/38 (2.63%)  2/39 (5.13%) 
Nervous system disorders     
Headache  9/38 (23.68%)  10/39 (25.64%) 
Dizziness  1/38 (2.63%)  2/39 (5.13%) 
Syncope  1/38 (2.63%)  2/39 (5.13%) 
Sinus headache  0/38 (0.00%)  2/39 (5.13%) 
Psychiatric disorders     
Insomnia  2/38 (5.26%)  0/39 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Pharyngolaryngeal pain  8/38 (21.05%)  3/39 (7.69%) 
Rhinorrhoea  3/38 (7.89%)  2/39 (5.13%) 
Cough  1/38 (2.63%)  5/39 (12.82%) 
Dysphonia  1/38 (2.63%)  2/39 (5.13%) 
Epistaxis  1/38 (2.63%)  2/39 (5.13%) 
Dyspnoea  0/38 (0.00%)  2/39 (5.13%) 
Skin and subcutaneous tissue disorders     
Rash  3/38 (7.89%)  1/39 (2.56%) 
Vascular disorders     
Hypertension  2/38 (5.26%)  2/39 (5.13%) 
Phlebitis  0/38 (0.00%)  2/39 (5.13%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The investigator must send a draft manuscript of the publication or abstract to the sponsor thirty days in advance of submission in order to obtain approval prior to submission of the final version for publication. This will be reviewed within thirty days and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution, which satisfies both parties.
Results Point of Contact
Name/Title: Henry Li
Organization: Talecris Biotherapeutics
Phone: 1-800-520-2807
Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT00263887     History of Changes
Other Study ID Numbers: 100533
First Submitted: September 12, 2005
First Posted: December 9, 2005
Results First Submitted: September 24, 2009
Results First Posted: August 21, 2014
Last Update Posted: August 21, 2014