SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study

Niigata University Medical & Dental Hospital

Status and phase: Completed; Phase 1

Conditions: Pulmonary Alveolar Proteinosis, Autoimmune

Treatments: Drug: Sargramostim

Study type: Interventional

Funder types: Other

Identifiers

NCT02840708

NTU

Details and patient eligibility

About

Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation.

Study Design: Pharmacokinetic open study

Enrollment: 14 patients

Sex: All

Ages: 20 to 80 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. Subjects judged to be appropriate for the study by the attending physician

2. can provide signed informed consent.

   aPAP patient must meet the following

3. aPAP patient aged over 20 and below 80 years old (as of the date of registration)

4. aPAP severity is mild or moderate. (not severe)

   Healthy volunteer must meet the following

5. Japanese healthy male subject aged over 20 and below 45 years old (as of the date of registration).

6. BMI (Body mass index) is between 18 and 25.

Exclusion criteria

1. WBC of 12,000/mcl or more

2. Fever of 38 degree celsius or more

3. History of malignant disease within recent 5 years (not applied to the treated cases of local basal cell carcinoma)

4. Complication of severe cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc.

5. Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), interstitial pneumonitis, lung fibrosis or bronchiectasis, in which the evaluations of safety of GM-CSF therapy are considered to be difficult.

6. History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.

7. liver dysfunction

8. renal dysfunction

9. Previous experience of severe and unexplained adverse events during aerosol delivery of any kind of medicinal product

10. Positive for HIV-Ab, HBs-Ag, HCV-Ab, STS or TPHA

11. allergic to GM-CSF.

12. addicted to illegal drugs

13. Participation to other clinical trials within 12 weeks before registration.

14. smoking within 5 years

15. cannot follow the procedure defined in this protocol

    aPAP patient must exclude the following

16. Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period

17. Patients who have been treated with whole-lung lavage, repeated segmental-lung lavage, GM-CSF or rituximab within 6 months before the start of the study.

18. taking other inhalation.

    Healthy volunteer must exclude the following

19. taking any medicines (incl. OTC).

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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14 participants in 3 patient groups

125mcg

Active Comparator group

Description:

SK-1401 125mcg single inhalation

Treatment:

Drug: Sargramostim

250mcg

Active Comparator group

Description:

SK-1401 250mcg single inhalation

Treatment:

Drug: Sargramostim

500mcg

Active Comparator group

Description:

SK-1401 500mcg single inhalation

Treatment:

Drug: Sargramostim