RI-001 in Immunosuppressed Respiratory Syncytial Virus (RSV) Infected Patients at Risk of Lower Tract RSV Illness

Study Overview
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Brief Summary
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RSV infections can develop into serious, life threatening conditions among immunocompromised patients. The objective of this study (ADMA 001) is to evaluate the safety and efficacy of RI-001 for the prevention of lower respiratory tract infections in immunocompromised patients identified as being infected with RSV in the upper respiratory tract.

Official Title
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RI-001 in Immunosuppressed Respiratory Syncytial Virus (RSV) Infected Patients at Risk of Lower Tract RSV Illness

Conditions
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Upper Respiratory Tract Infection, Lower Respiratory Tract Infection

Intervention / Treatment
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* Biological: RI-001
* Biological: RI-001
* Biological: RI-001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: An IEC/IRB approved written informed consent signed and dated by the patient or by parent(s) or a legally acceptable representative. The consent form or a specific assent form, where required, will be signed and dated by minors. Documented Bone Marrow Transplant (BMT)/Hematopoietic Stem Cell Transplant (HSCT), Pulmonary/Cardiac Transplant, Pulmonary Transplant or Liver Transplant within the 2 years prior to randomization to the study drug. Male/Female patients age: (Pediatric) ≥2 years and <16 years at the time of informed consent. Male/Female patients age: (Adult) ≥ 16 years and ≤ 65 years at the time of informed consent. Patient must have an URTI as defined by Respiratory Assessment Score (RAS)=1. Patients must be actively taking at least one immunosuppressive agent. Patients must have a positive RSV RT-PCR at the time of the randomization procedures. Female patients must be of non-childbearing potential or have a negative pregnancy test prior to study start and be deemed not at risk of becoming pregnant by adherence to a reliable contraceptive method for the duration of the study. Females of non-childbearing potential are defined as women who have had a hysterectomy, bilateral oophorectomy, tubal ligation or who have been post-menopausal for at least two years, or are considered to be sterile due to recent chemotherapy. Female patients who are not breast-feeding. Patient/legally acceptable representative considered as reliable and capable of adhering to the protocol (e.g. able to understand and complete diaries and questionnaires), visit schedules or treatment regimen according to the judgment of the Investigator. Exclusion Criteria: Documented RSV lower respiratory tract infection (respiratory assessment score is greater than 1) as determined by the site investigators or research staff. Requirement for mechanical ventilation, extracorporeal membrane oxygenation, continuous positive airway pressure or other mechanical respiratory or cardiac support Unstable respiratory status so severe that survival is not expected for longer than 6 months. End organ dysfunction resulting in anticipated survival of less than 6 months. Known to be HIV positive. Administration of any RSV specific products, including palivizumab (Synagis®) in the 3 months prior to randomization procedures. Previous, current, or planned administration of an investigational RSV vaccine. Known hypersensitivity to immunoglobulin. Known Immunoglobulin (IgA) deficiency Known renal impairment requiring any form of dialysis (HD, PD, CRRT). Known hemodynamically significant congenital heart disease. Previous poor compliance with visit schedules. Severe medical, neurological or psychiatric disorders or laboratory values which may have an impact on the safety of the patient. Concurrent participation in other investigational drug product studies; any exception must be approved by the ADMA Biologics Medical Director.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Dose regimen 1 | Biological: RI-001<br>* Dose 1<br>|
| Experimental: 2<br>Dose regimen 2 | Biological: RI-001<br>* Dose 2<br>|
| Placebo Comparator: 3<br>Placebo | Biological: RI-001<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Circulating RI-001 Titer | The primary endpoint of this study was the mean fold titer increase from baseline to Day 18 in circulating serum anti-RSV neutralizing antibody following treatment with RI-001. | Study day 18 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of RSV Progression From Symptomatic Upper Respiratory Tract Infection to Lower Respiratory Tract Infection. |  | Study day 33 | 
| The Number of Patients Achieving at Least a 4-fold Increase in Serum RSV Neutralizing Antibody Titers |  | 18 Days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Transplant, Immunosuppression