This study has been transitioned to CTIS with ID 2024-513124-41-00 check the CTIS register for the current data. 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
- Infections - pathogen unspecified
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Difference in mean AD and ILD scores (as measured with CT scanning) between t=0
and t=2 years in patients with standard vs higher Ig replacement therapy
dosing.
Secondary outcome
1. The incidence of symptomatic lower pulmonary infections in PAD patients with
high Ig replacement therapy dosing versus standard Ig replacement therapy
dosing.
2. Number of physician diagnosed lower respiratory tract infections in patients
with high vs standard Ig replacement therapy dosing.
3. Number of hospital admissions and duration of hospital admissions for
pulmonary complications (ao exacerbations of bronchiectasis)
4. Outcomes of pulmonary function tests (specifically: Total Lung Capacity
(TLC), Forced Expiratory Volume after 1 second (FEV1), CO diffusion) on t=0 and
t=2 years in all patients
5. Days missed from work / school in patients with high vs standard Ig
replacement therapy dosing.
6. Total therapeutic and preventive costs
Background summary
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. Standard treatment for patients with PAD consists of
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
This study has been transitioned to CTIS with ID 2024-513124-41-00 check the CTIS register for the current data.
To show the protective value and to measure cost effectiveness of higher Ig
dosing on progression of lung disease in PAD.
Study design
Multicenter, randomized controlled study performed by 3 academic centers in The
Netherlands.
Intervention
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
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
Utrecht 3508AB
NL
Lundlaan 6
Utrecht 3508AB
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:
1. Age 8-60 years
2. Diagnosis of Primary Antibody Deficiency / Common Variable Immunodeficiency
Disorder (see Appendix 1).
3. Indication for immunoglobulin replacement therapy and/or treated with
immunoglobulin replacement therapy
4. Current IgG dosing 0.25 - 0.6 gr / kg / 3-4 weeks
5. Receiving treatment and follow-up for PAD by a physician in one of the
participating centers
6. Written informed consent
Exclusion criteria
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
1. Diagnosis of Combined Immunodeficiency (CID) disease at onset of study (see
Appendix 1). Explanation: Combined Immunodeficiency is featured by the
occurrence of more viral infections and reactivations and thus less comparable
to PAD.
2. Severe pulmonary disease, determined by an independent radiologist:
a. Baseline AD score > 7 and/or ILD score > 5, in combination with:
i. Saccular bronchiectasis on CT scan, or;
ii. Clinical diagnosis of severe respiratory insufficiency ( (defined as:
saturations in room air <92%, and/ or oxygen dependency).
b. Baseline pulmonary function (FEV1 and FVC) <70% expected for age and body
weight / length)
3. Active smoker
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| EU-CTR | CTIS2024-513124-41-00 |
| EudraCT | EUCTR2021-005001-26-NL |
| CCMO | NL79088.041.21 |