MedPath

Viral Inhibition in Children for Treatment of RSV

Phase 2
Completed
Conditions
RESPIRATORY SYNCYTIAL VIRUS INFECTIONS
Interventions
Drug: Placebo
Registration Number
NCT02654171
Lead Sponsor
Shanghai Ark Biopharmaceutical Co., Ltd.
Brief Summary

VICTOR is a randomized, double-blind, placebo-controlled, multicenter, 2-part study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antiviral effect of single and multiple dosing of AK0529 in infants hospitalized with RSV infection.

Detailed Description

Globally, Respiratory Syncytial Virus (RSV) is recognized as the leading cause of respiratory tract infections in infants and young children and a major cause of hospitalization due to severe respiratory infection. Despite four decades of effort, there are still no effective methods to control RSV infection. Treatment of RSV has been limited to supportive measures. There is an urgent need for safe and effective drugs to treat and prevent RSV disease.

AK0529 is an investigational antiviral agent that targets the RSV fusion protein on the surface of the viral envelope and exerts antiviral activity against RSV by inhibiting viral entry into host cells and preventing fusion protein induced cell-cell fusion. AK0529 was generally well tolerated in healthy volunteers.

This study is designed as a randomized, double-blind, placebo-controlled, multicenter, phase 2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antiviral effect of single and multiple dosing of AK0529 in infants hospitalized with RSV infection. It will consist of two parts, Part 1 and Part 2. Each part will consist of three phases, a pre-treatment phase, a treatment phase and post-treatment follow-up phase.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
72
Inclusion Criteria
  • Male or female patients of any race or ethnicity with an age adjusted for any prematurity of ≥1 month and ≤24 months.
  • Diagnosis of RSV infection by virological means, which may include rapid diagnostic point-of-care testing, within 96 hours preceding screening for Part 1 and 72 hours for Part 2.
  • Patient must weigh >3 kg at screening and be within the 10th and 90th percentiles (inclusive) for the patient's age, based on the local child growth standards, i.e. the Australian Paediatric Endocrine Group Growth Charts.
  • The parent / legal guardian of the patient must have provided written informed consent for the patient to participate.
  • For patients aged <12 months, an occipito-frontal head circumference within the normal range for age and gender.
Exclusion Criteria
  • The patient has taken, is currently taking or requires any restricted medications.
  • Patient is known to be HIV-positive (or the mother, if the potential patient is a child aged <6 months).
  • Participation in an investigational drug or device study within 30 days prior to the date of screening.
  • Requires vasopressors or inotropic support at the time of enrolment.
  • Concurrent gastrointestinal conditions that could, in the opinion of the investigator, prejudice absorption of the Investigational Medicinal Product (e.g. protracted vomiting, malabsorption syndrome, a history of necrotising enterocolitis with consequent short gut syndrome).
  • Bronchopulmonary dysplasia or chronic lung disease requiring assisted ventilation at the time of enrolment.
  • Diminished ventilatory reserve at risk for hypercapnia (e.g. pulmonary hypoplasia, sequestration syndromes, cystadenomatoid malformation, a history of surgery for diaphragmatic hernia).
  • Left to right shunt meriting corrective therapy.
  • Renal failure including renal anomalies likely to be associated with renal insufficiency (e.g. clinical conditions of renal dysplasia, polycystic renal disease, renal agenesis).
  • Clinical evidence of hepatic decompensation (e.g. hepatic disorder with associated coagulopathy or associated encephalopathy).
  • Cerebral palsy with microcephaly, chronic or persistent feeding difficulties or seizures.
  • Symptomatic because of inborn errors of metabolism (e.g. mitochondrial disorders, disorders of carbohydrate metabolism, glycogen storage disorders).
  • Congenital or acquired immunodeficiency (e.g. congenital agammaglobulinaemia, common variable immunodeficiency, immunosuppressive therapy other than glucocorticoid or montelukast therapy forming part of care directed by the treating physician).
  • For Part 2 of this study, children with a history of having received palivizumab or any other monoclonal agent directed against RSV in the preceding 120 days. This exclusion criterion does not apply to Part 1.
  • Evidence of active or uncontrolled respiratory, cardiac, hepatic, central nervous system or renal disease unrelated to RSV infection at baseline or any other medical condition that in the opinion of the investigator renders the patient unsuitable for enrolment.
  • A history of epilepsy or seizures including febrile seizures.
  • Allergy to test medication or constituents.
  • Weight less than 10th percentile or greater than 90th percentile for age and gender adjusted for any prematurity.
  • The patient's parent or legally acceptable representative is an employee of the investigator or the study center, with direct involvement in the proposed study or other studies under the direction of that investigator of the study center, or any family members of the employees or the investigator.
  • Failure to satisfy the investigator of fitness to participate for any other reason.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
AK0529AK0529Subjects will receive single or multiple doses of AK0529 at different dose levels within different cohorts.
PlaceboPlaceboPatients who are randomized to the control arm within each cohort will receive the corresponding placebo to AK0529.
Primary Outcome Measures
NameTimeMethod
Incidence of Adverse Events during the studyDay -4 to single dose Day 7 (Part 1) and Day -3 to multiple dose Day 14 (Part 2)
Subject withdrawals due to Adverse EventsDay -4 to single dose Day 7 (Part 1) and Day -3 to multiple dose Day 14 (Part 2)
Secondary Outcome Measures
NameTimeMethod
Area under curve change of viral loadFrom baseline to Day 5

The antiviral effects in infants hospitalized with RSV are to be determined by measuring the RSV viral load area under the curve in nasal, pharyngeal and tracheal washes / aspirates from baseline to last administration of study medication (Day 5).

Maximum plasma concentration of AK0529 (Cmax)Pharmacokinetic samples will be taken predose, 2, 4, 6, 12, 18, 24 hours postdose on Day 1, 48 hours postdose (Part 1 and Part 2) and Day 5 (Part 2 )

For patients without intravenous cannulation, samples will be collected at pre-dose, 2, 6 and 12 hours post-dose on Day 1.

Apparent total body clearance (CL/F)Pharmacokinetic samples will be taken predose, 2, 4, 6, 12, 18, 24 hours postdose on Day 1, 48 hours postdose (Part 1 and Part 2) and Day 5 (Part 2 )

For patients without intravenous cannulation, samples will be collected at pre-dose, 2, 6 and 12 hours post-dose on Day 1.

Incidence of F-protein genotypesSpecimen will be collected predose and 24 hours postdose on Day 1 (Part 1) and on Day 1-5 (Part 2).

The incidence of F-protein genotypes associated with reduced sensitivity to IMP will be evaluated.

Change of symptom scoreFrom baseline to Day 1 (Part 1) and up to Day 5 after multiple drug administration (Part 2).

To evaluate the change of RSV related symptom score in AK0529 arms compared with the change in placebo arm after treatment. The total score is reported with a range from 0 to 12. A decreasing value of total score represents clinical improvement. Subscales are not applicable in this symptom score.

Plasma concentration of AK0529 at 12 hours postdose (C12)Pharmacokinetic samples will be taken predose, 2, 4, 6, 12, 18, 24 hours postdose on Day 1, 48 hours postdose (Part 1 and Part 2) and Day 5 (Part 2 )

For patients without intravenous cannulation, samples will be collected at pre-dose, 2, 6 and 12 hours post-dose on Day 1.

Apparent central compartment volume of distribution (Vc/F)Pharmacokinetic samples will be taken predose, 2, 4, 6, 12, 18, 24 hours postdose on Day 1, 48 hours postdose (Part 1 and Part 2) and Day 5 (Part 2 )

For patients without intravenous cannulation, samples will be collected at pre-dose, 2, 6 and 12 hours post-dose on Day 1.

Area under the plasma concentration-time curve from time 0 to infinity (AUC)Pharmacokinetic samples will be taken predose, 2, 4, 6, 12, 18, 24 hours postdose on Day 1, 48 hours postdose (Part 1 and Part 2) and Day 5 (Part 2 )

For patients without intravenous cannulation, samples will be collected at pre-dose, 2, 6 and 12 hours post-dose on Day 1.

Trial Locations

Locations (28)

Gold Coast University Hospital

🇦🇺

Gold Coast, Queensland, Australia

Soroka University Medical Center

🇮🇱

Beer-Sheva, Israel

Schneider Children's Medical Center

🇮🇱

Petach Tikvah, Israel

Ruth Rappaport Children's Hospital

🇮🇱

Haifa, Israel

Rafik Hariri University Hospital

🇱🇧

Beirut, Lebanon

Hospital Sibu

🇲🇾

Sibu, Sarawak, Malaysia

Hospital Selayang

🇲🇾

Batu Caves, Selangor, Malaysia

Hospital Tengku Ampuan Rahimah

🇲🇾

Klang, Selangor, Malaysia

Szpital im. Świętej Jadwigi Śląskiej w Trzebnicy

🇵🇱

Trzebnica, Trzebnica County, Poland

Linkou Chang Gung Memorial Hospital

🇨🇳

Taoyuan, Taiwan

Taipei Municipal Wanfang Hospital

🇨🇳

Taipei, Taiwan

Cukurova University Balcali Hospital

🇹🇷

Adana, Adana Province, Turkey

Eskisehir Osmangazi University Faculty of Medicine

🇹🇷

Eskişehir, Eskişehir Province, Turkey

Marmara University Faculty of Medicine

🇹🇷

İstanbul, Turkey

American University of Beirut Medical Center

🇱🇧

Beirut, Lebanon

Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem w Poznaniu

🇵🇱

Poznań, Greater Poland, Poland

Lady Cilento Children's Hospital

🇦🇺

South Brisbane, Queensland, Australia

Makassed General Hospital

🇱🇧

Beirut, Lebanon

Nowy Szpital w Świeciu

🇵🇱

Świecie, Kujawy-Pomerania, Poland

Kaohsiung Chang Gung Memorial Hospital

🇨🇳

Kaohsiung, Taiwan

Prince of Wales Hospital

🇭🇰

Sha Tin, Hong Kong

Saint George Hospital University Medical Center

🇱🇧

Beirut, Lebanon

University Malaya Medical Center

🇲🇾

Kuala Lumpur, Malaysia

Hospital Tuanku Jaafar

🇲🇾

Seremban, Negeri Sembilan, Malaysia

Mackay Memorial Hospital

🇨🇳

Taipei, Taiwan

Ege University Medical Faculty

🇹🇷

İzmir, İzmir Province, Turkey

Women's and Children's Hospital

🇦🇺

Adelaide, South Australia, Australia

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

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