Objective: To show the protective value and to measure cost effectiveness of higher Ig dosing on progression of lung disease in PAD.
ID
Source
Brief title
Condition
- Immunodeficiency syndromes
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Main study parameters/endpoints: 1. Progression of lung disease, determined by
computed tomography (CT) scanning and pulmonary function test
Secondary outcome
2. Number of respiratory infections 3. Days missed from school/work. 4. Cost
savings 5. Quality of life.
Background summary
Rationale: Patients with Primary Antibody Deficiencies (PADs) frequently
encounter chronic lung disease, caused by recurrent airway disease and/or
interstitial lung disease. Chronic lung disease leads to absence from work and
school and significant health costs. Patients with PAD are treated with
immunoglobulin replacement therapy. Optimal dosing to prevent lung disease is
unclear and different dosing regimens, all within prescription label, are used
nationwide. We and others recently showed that higher immunoglobulin (Ig)
trough levels were related to less airway infections and slower progression of
airway disease. These findings need confirmation in a prospective randomized
setting.
*
Study objective
Objective: To show the protective value and to measure cost effectiveness of
higher Ig dosing on progression of lung disease in PAD.
Study design
Study design: Multicenter, prospective randomized controlled trial, performed
by 3 academic centers in The Netherlands.
Intervention (if applicable): Two Ig dosing regimens (both in normal range)
are compared: Control group: Ig dose 0.4-0.6 g/kg/L, vs intervention group:
dose increase of 33% (relative to pre-study dose) will be administered for 2
years. CT scanning and pulmonary function tests will be performed at t=0 and 24
months.
Study burden and risks
Nature and extent of the burden and risks associated with participation,
benefit and group relatedness: The study compares two existing prophylaxis
regimes for pulmonary disease in patients with primary antibody disease.
Administration of medication and measurement of trough levels of medication
follows routine medical guidelines for treatment and follow up. The extra
procedures to which patients are subjected in this protocol are as follows: two
CT scans of the lungs and two pulmonary function tests (t=0 and 24 months; 30
minutes per procedure). Futher, during routine venapunture moments, 5 extra
blood samples will be taken, and 2 extra venapunctures will be taken apart from
the routine samples. Finallly, three times the completion of a questionnaire
set on quality of life and productivity losses (30 minutes per time in total).
The extent of these extra procedures is considered to have low impact. Children
will be part of the study. The study must be conducted in a pediatric
population as well because children are especially at high risk for pulmonary
disease.
Lundlaan 6 Lundlaan 6
Utrecht 3584 EA
NL
Lundlaan 6 Lundlaan 6
Utrecht 3584 EA
NL
Listed location countries
Age
Inclusion criteria
In order to be eligible to participate in this study, a subject must meet all
of the following criteria:
-Age 4-60 years
-Diagnosis of Primary Antibody Deficiency / Common Variable Immunodeficiency
Disorder (see Appendix A).
-Indication for immunoglobulin replacement therapy and/or treated with
immunoglobulin replacement therapy
-Current IgG dosing 0.4 - 0.6 gr / kg / 3-4 weeks
-Receiving treatment and follow up for PAD by one of the physicians in the
participating centers
-Written informed consent
-Normal lung status, or mild to medium severe pulmonary disease (measured by
pulmonary function test and on CT scan, scored by an independent radiologist
based on the following criteria):
* Baseline AD score < 5 and ILD score < 7, or;
* Baseline AD score > 5 and/or ILD score > 7 without clinical diagnosis of
severe respiratory insufficiency (defined as: saturations in room air <92% and
/ or oxygen dependency).
* Baseline pulmonary function (FEV1 and FVC >70% expected for age and body
weight / length)
Exclusion criteria
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
-Age above 60 year
-Diagnosis of Combined Immunodeficiency (CID) disease at onset of study (see
Appendix A). Explanation: Combined Immunodeficiency is featured by the
occurrence of more viral infections and reactivations and thus less comparable
to PAD.
-Severe pulmonary disease, determined by an independent radiologist:
* Baseline AD score > 5 and/or ILD score > 7, in combination with:
o Saccular bronchiectasis on CT scan, or;
o Clinical diagnosis of severe respiratory insufficiency (defined as: (defined
as: saturations in room air <92%, and/ or oxygen dependency).
* Baseline pulmonary function (FEV1 and FVC >70% expected for age and body
weight / length)
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL74793.041.20 |