Interstitial Lymphocytic Lung DiseaseNCT04572620
Rituximab and Abatacept Effectiveness in Differential Treatment of Interstitial Lymphocytic Lung Disease in Children With Primary Immunodeficiencies.
An observational study of Interstitial Lymphocytic Lung Disease, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
Trial summary
The rationale for this retrospective study is to evaluate the efficacy and safety of abatacept and rituximab treatment of ILLD in a cohort of pediatric patients with different forms of PID, who received one of the two therapy regimens predominantly based on the lesions histopathology. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Interstitial Lymphocytic Lung Disease
- What is being tested?
- Rituximab and Abatacept
- Type of treatment
- Injection or infusion
- How many people
- 30 participants
- Where
- 1 siteRussia
- Study dates
- 7 Sep 2020to Sep 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric patients with interstitial lymphocytic lung disease treated in Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia
Who may be able to join
- Histological and/or radiological diagnosis of ILLD.
- Treatment with rituximab or abatacept for at least 12 months.
- No signs of ongoing infectious pulmonary process at the start of the treatment.
- Regular IG substitution with trough IgG levels > 5g/l.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
Russia1 site, 1 recruiting
- Recruiting
Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology
Moscow, 117198
Anna Shcherbina, MD, PhD, +7(495)2876570 ext. 6299, shcher26@hotmail.com
About this study
Official title: Retrospective Chart Review of Children With Primary Immunodeficiencies (PID) Who Received Targeted Therapy of Interstitial Lymphocytic Lung Disease (ILLD) With Abatacept or Rituximab.
The rationale for this retrospective study is to evaluate the efficacy and safety of abatacept and rituximab treatment of ILLD in a cohort of pediatric patients with different forms of PID, who received one of the two therapy regimens predominantly based on the lesions histopathology.
Read the detailed description
Primary immunodeficiencies (PID) represent a heterogeneous group of more than 400 inherited conditions with associated immune dysfunctions. Though severe recurrent/chronic infections are the main cause of mortality and morbidity in PID, immune dysregulation manifesting with oncological and autoimmune or autoinflammatory conditions involving various organs and systems have been the focus of research in the recent years.
The interstitial lymphocytic lung disease (ILLD) is one of the recently characterized non-malignant PID complications. Immune dysregulation in ILLD causes reactive bronchi-associated lymphoid tissue (BALT) hyperplasia that manifests in several pathomorphological forms: follicular bronchiolitis (FB), nodular lymphoid hyperplasia (NLH), and lymphocytic interstitial pneumonia (LIP). Treatment of ILLD patients with various immunosuppressive drugs leads to inconsistent results ranging from partial\transient effect to no effect at all and has been often associated with adverse effects and an increase in infections' rate. Therefore there is a need for targeted therapy of ILLD. In small cohorts of adult PID patients rituximab in combination with azathioprine proved to be effective. Yet, the reports are scarce and there is currently no consensus on ILLD treatment, especially in children.
The study will collect and analyze information on the effectiveness and safety of ILLD monotherapy with rituximab or abatacept, chosen predominantly based on the pathomorphological characteristics of lymphoid infiltration, as well as genetic defects, in a cohort of pediatric patients with PID.
- Conditions
- Interstitial Lymphocytic Lung Disease
- Being tested
- Rituximab, Abatacept
- Keywords
- interstitial lymphocytic lung disease, primary immunodeficiency, children, treatment, rituximab, abatacept
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
375 mg/m2 as 4 weekly consecutive i.v. infusions with subsequent infusions of 375 mg/m2 performed every 3 months for 12 months.
AbataceptDrug
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.
| Study group | Role | What they receive |
|---|---|---|
| group 1(rituximab) | Not described Receives: Drug: Rituximab | |
| group 2 (abatacept) | Not described Receives: Drug: Abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Measured: before the time the first dose of study treatment was administered
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Measured: 3 months of treatment
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Measured: 6 months of treatment
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Measured: 1 year of treatment
Secondary outcomes (2)
Frequency of adverse events
Measured: from the time the first dose of study treatment was administered until 12 mo of treatment
quality of life of patients
The quality of life of patients before and after therapy
Measured: before the time the first dose of study treatment was administered until 12 mo of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2020
- Study start7 Sep 2020
- Primary completionSep 2021 (estimated)
- Study completionSep 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Interstitial Lymphocytic Lung DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04572620
- Sponsor's protocol ID
- NCPHOI-2020-08
- Status last verified
- Sep 2020
- First submitted
- 21 Sep 2020
- Last updated
- 1 Oct 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Chart Review of Children With Primary Immunodeficiencies (PID) Who Received Targeted Therapy of Interstitial Lymphocytic Lung Disease (ILLD) With Abatacept or Rituximab.
- ClinicalTrials.gov ID
- NCT04572620
- Acronym / sponsor ID
- NCPHOI-2020-08
- Conditions
- Interstitial Lymphocytic Lung Disease
- Keywords
- interstitial lymphocytic lung disease, primary immunodeficiency, children, treatment, rituximab, abatacept
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 7 Sep 2020 (actual)
- Primary completion
- Sep 2021 (estimated)
- Study completion
- Sep 2021 (estimated)
- First posted
- 1 Oct 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The rationale for this retrospective study is to evaluate the efficacy and safety of abatacept and rituximab treatment of ILLD in a cohort of pediatric patients with different forms of PID, who received one of the two therapy regimens predominantly based on the lesions histopathology.
Detailed description
Primary immunodeficiencies (PID) represent a heterogeneous group of more than 400 inherited conditions with associated immune dysfunctions. Though severe recurrent/chronic infections are the main cause of mortality and morbidity in PID, immune dysregulation manifesting with oncological and autoimmune or autoinflammatory conditions involving various organs and systems have been the focus of research in the recent years.
The interstitial lymphocytic lung disease (ILLD) is one of the recently characterized non-malignant PID complications. Immune dysregulation in ILLD causes reactive bronchi-associated lymphoid tissue (BALT) hyperplasia that manifests in several pathomorphological forms: follicular bronchiolitis (FB), nodular lymphoid hyperplasia (NLH), and lymphocytic interstitial pneumonia (LIP). Treatment of ILLD patients with various immunosuppressive drugs leads to inconsistent results ranging from partial\transient effect to no effect at all and has been often associated with adverse effects and an increase in infections' rate. Therefore there is a need for targeted therapy of ILLD. In small cohorts of adult PID patients rituximab in combination with azathioprine proved to be effective. Yet, the reports are scarce and there is currently no consensus on ILLD treatment, especially in children.
The study will collect and analyze information on the effectiveness and safety of ILLD monotherapy with rituximab or abatacept, chosen predominantly based on the pathomorphological characteristics of lymphoid infiltration, as well as genetic defects, in a cohort of pediatric patients with PID.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| group 1(rituximab) | – | Drug: Rituximab |
| group 2 (abatacept) | – | Drug: Abatacept |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric patients with interstitial lymphocytic lung disease treated in Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia
Key inclusion criteria (4 listed)
- Histological and/or radiological diagnosis of ILLD.
- Treatment with rituximab or abatacept for at least 12 months.
- No signs of ongoing infectious pulmonary process at the start of the treatment.
- Regular IG substitution with trough IgG levels > 5g/l.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rituximab375 mg/m2 as 4 weekly consecutive i.v. infusions with subsequent infusions of 375 mg/m2 performed every 3 months for 12 months. | Drug | group 1(rituximab) |
| Abatacept10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months. | Drug | group 2 (abatacept) |
Primary outcome
Dynamics of severity clinical and radiological symptoms
Time frame: before the time the first dose of study treatment was administered
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Dynamics of severity clinical and radiological symptoms
Time frame: 3 months of treatment
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Dynamics of severity clinical and radiological symptoms
Time frame: 6 months of treatment
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Dynamics of severity clinical and radiological symptoms
Time frame: 1 year of treatment
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
2 secondary outcomes
Frequency of adverse events
Time frame: from the time the first dose of study treatment was administered until 12 mo of treatment
quality of life of patients
Time frame: before the time the first dose of study treatment was administered until 12 mo of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Interstitial Lymphocytic Lung Disease, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anna Shcherbina, MD, PhD (+7(495)2876570 ext. 6299, shcher26@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dynamics of severity clinical and radiological symptoms (time frame: before the time the first dose of study treatment was administered). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2020.