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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01669421 |
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Date of registration:
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14/08/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
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Scientific title:
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Effect of a Higher Dose of Alpha-1 Antitrypsin Augmentation Therapy on Lung Inflammation in Subjects With Alpha-1 Antitrypsin Deficiency. |
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Date of first enrolment:
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July 2012 |
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Target sample size:
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10 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01669421 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Michael A Campos, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Miami |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Males or Females aged between 18 and 75 years.
- Diagnosis of AATD, based on documentation of "at-risk" genotypes such as Pi ZZ, SZ or
Znull OR documentation of a pre-therapy AAT level < 11 µM.
- Evidence of COPD (emphysema or airflow obstruction) with FEV1 < 80%
- Receiving standard dose of augmentation therapy (with any commercial formulation) for
at least 1 month at the dose of 60 mg/kg/week.
- At least ONE of the following criteria of disease severity:
- 2 or more acute exacerbations or 1 hospitalization due to respiratory symptoms in
the past 12 months. Definition of exacerbations: the use of antibiotics and a
course of steroids to treat a flare of pulmonary symptoms, regardless if the
subject required emergency room care or hospital admission. The diagnosis of the
acute exacerbation will be obtained by direct history obtained from the patient
and confirmed by the PI. Attempts should be made to have documentation from the
patient's treating physicians, although not required for study entry.
- St. George Respiratory Questionnaire (SGRQ) total score = 60.
- Chronic bronchitis: daily or almost daily sputum expectoration at least 3 months
of the year for at least 2 consecutive years. The diagnosis of chronic bronchitis
will be obtained by direct history obtained from the patient and confirmed by the
PI. Attempts should be made to have documentation from the patient's treating
physicians, although not required for study entry.
- Documented FEV1 decline of at least = 60 ml/year for 2 consecutive years while
receiving augmentation therapy
Exclusion Criteria:
- Patients unsuitable to have a bronchoscopy due to poor clinical condition as judged by
the PI. In general we will exclude subjects with hypoxemia, coagulopathy or FEV1 below 40%
predicted.
Note: Subjects with FEV1 values below 40% predicted may be included and reassessed after
optimization of therapy. Final determination to include the patient if deemed suitable for
the procedure will be determined by the PI before first planned bronchoscopy (regardless of
FEV1 value).
- Patients participating in other clinical trials.
- Use of chronic antibiotics or oral steroids
- Continues to smoke
- Inability to sign informed consent
- Pregnancy or willing to become pregnant
- Known IgA deficiency (we will include only patients already receiving augmentation
therapy so it will be unlikely to encounter this exclusion criteria)
Age minimum:
18 Years
Age maximum:
75 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Alpha 1 Antitrypsin Deficiency
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Intervention(s)
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Drug: Alpha-1 Antitrypsin (human)
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Primary Outcome(s)
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Changes in Inflammatory Biomarkers in Bronchoalveolar Lavage Fluid
[Time Frame: Between baseline (week 4), double dose A1PI (week 8) and again standard dose (week 12)]
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Secondary Outcome(s)
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Change in Inflammatory Biomarkers in Serum Samples
[Time Frame: Between baseline (week 4), double dose A1PI (week 8) and again standard dose (week 12)]
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Number of Adverse Events Reported
[Time Frame: From Week 1 to week 12]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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