Clinical trial NCT01924793: An Open Label Study to Examine the Effects of DAS181 Administered by Dry Powder Inhaler (DPI) or Nebulized Formulation in Immunocompromised Subjects With Parainfluenza (PIV) Infection
Official title: An Open Label Study to Examine the Effects of DAS181 Administered by Dry Powder Inhaler (DPI) or Nebulized Formulation in Immunocompromised Subjects With Parainfluenza (PIV) Infection
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Parainfluenza
Lead sponsor: Ansun Biopharma, Inc.
Sponsor class: INDUSTRY
First posted: 2013-08-19
Enrollment: 23
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This protocol will seek to enroll up to 60 immunocompromised subjects diagnosed with parainfluenza infection.

All subjects will have additional PK and Immunogenicity blood samples collected.

Detailed description: The study will investigate the safety, tolerability, clinical and virologic effect of DAS1814 for the treatment of PIV infections in up to 60 immunocompromised patients with no other treatment options.

Eligible immunocompromised patients positively diagnosed with PIV infection will receive DAS181 by DPI or Nebulized formulation.

DPI: 10 mg DAS181 will be administered for seven (7) consecutive days for up to a total cumulative dose of 70mg.

Nebulized: DAS181 F02 formulation in solution will be administered for seven (7) consecutive days for up to a total cumulative dose of 28.9 mg.

Assessment of viral shedding and viral resistance testing will be performed on collected viral load samples. Blood samples will be obtained to evaluate safety at each scheduled visit.

Routine safety monitoring (including Adverse Event (AE) reporting, clinical laboratory tests, vital signs, electrocardiogram (ECG) and )2 levels) will be conducted in all subjects. A final safety assessment will occur at Day 28 (+/- 2 days) after enrollment/first dose of study drug. Survival, diary cards, and quality of life will be collected at Day 60 and 90. PK will be collected at each visit on all subjects from baseline/visit 2 to visit 11/Day 28. Immunogenicity samples will be collected at selected time points in all subjects.

Eligibility criteria: Inclusion Criteria:

1. Age ≥12 years
2. Able to provide informed consent or child assent with parental consent
3. Immunocompromised, as defined by one of the following:

   * Allogeneic hematopoietic cell transplantation (HCT)
   * Lung or lung-heart transplantation
   * Patients treated with chemotherapy for hematologic malignancies or autologous HCT
4. Confirmed Parainfluenza virus by nasopharyngeal swab or tracheal aspirate for one of the following:

   * Respiratory virus panel
   * DFA
   * Qualitative/quantitative RT-PCR test for parainfluenza virus performed at the local laboratory (a confirmatory PCR test will be done at the central lab but is not required to start the patient on study).
5. Female subjects of child-bearing potential who are capable of conception must be:

   post-menopausal (one year or greater without menses), surgically incapable of childbearing, or practicing two effective methods of birth control. Acceptable methods include intrauterine device, spermicide, barrier, male partner surgical sterilization and hormonal contraception. A female subject must agree to practice two acceptable methods of birth control during the 28 day study period. Abstinence is not an acceptable method of contraception. All reproductive female subjects must have a negative serum pregnancy test during the screening visit.
6. Male subjects must agree to use medically accepted form of contraception during the 28 day study period.

Exclusion Criteria:

1. Psychiatric or cognitive illness or recreational drug/alcohol use that, in the opinion of the principal investigator, would affect patient safety and/or compliance.
2. Any significant finding in the patient's medical history or physical examination that, in the opinion of the investigator, would affect patient safety, ability to use the dry powder inhaler or compliance with the dosing schedule.
3. Subjects currently treated with oral, aerosolized or IV ribavirin
4. Subjects taking any investigational drug used to research or treat PIV

Primary outcomes:
measure: Cumulative incidence and severity of AEs, SAEs, and lab abnormalities as a measure of safety and tolerability.
timeFrame: 2 years
Secondary outcomes:
measure: Proportion of subjects with PIV viral load reduction at Day 28
timeFrame: 2 years
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Blood and Marrow Transplantation
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Cancer Center
state: Kansas
city: Baltimore
country: United States
facility: John Hopkins University School of Medicine
state: Maryland
city: Boston
country: United States
facility: Brigham & Women's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota, School of Medicine
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: Weill Cornell Medical College/New York Presbyterian Hospital
state: New York
city: New York
country: United States
facility: Weill Cornell Medical College-Peds
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Houston
country: United States
facility: The University of Texas MD Anderson Cancer Center
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Seattle
country: United States
facility: Fred Hutchinson Cencer Research Center
state: Washington
Study officials:
affiliation: Ansun Biopharma, Inc.
name: Ron Moss, MD, CEO
role: STUDY_CHAIR
References: