AIR001-CS06 A PHASE 2, MULTI-CENTER, OPEN-LABEL STUDY TO EVALUATE THE INTERMEDIATE/LONG-TERM SAFETY AND EFFICACY OF AIR001 IN SUBJECTS WITH WHO GROUP 1 PULMONARY ARTERIAL HYPERTENSION
This protocol describes a Phase 2, multi-center, open-label study to determine the intermediate/long-term safety with limited efficacy assessments of inhaled nebulized AIR001.Only subjects who previously completed the 16-week AIR001-CS05 study as planned will be eligible. The EOS visit in AIR001-CS05 will serve as Baseline for the AIR001-CS06 protocol, if performed within 2 weeks of the first dose of AIR001-CS06. Subjects will be allocated to treatment during the Baseline/Day 1 study visit utilizing the Interactive Web Response System (IWRS) to the same dose and regimen as was previously allocated in clinical study AIR001-CS05. Thereafter, subjects will have onsite clinic assessments performed every 24 weeks. Subjects will receive nebulized AIR001 according to the same treatment arm randomized to in AIR001-CS05 (see below).
Treatment Arms (dose loaded in the I-Neb AAD system nebulizer):
and #149; 80 mg AIR001, nebulized 4 times daily
and #149; 46 mg AIR001, nebulized 4 times daily
and #149; 80 mg AIR001, nebulized once daily
1. Evidence of a personally signed and dated informed consent document indicating
that the subject (or a legally acceptable representative) has been informed of all
pertinent aspects of the study prior to initiation of any subject-mandated
2. Is willing and able to comply with scheduled visits, treatment plan, laboratory tests,
and other study procedures.
3. Has completed the 16-week AIR001-CS05study as planned.
4. If on corticosteroids, has been receiving a stable dose of <= 20 mg/day of prednisone
(or equivalent dose, if other corticosteroid) for at least 1 month (30 days) prior to
the AIR001-CS05 study Baseline/Day 1 visit. If receiving treatment for
Connective Tissue Disease (CTD) with any other drugs, doses should remain
stable, if clinically feasible, for the duration of the AIR001-CS06 study.
5. Women of childbearing potential must be using at least one form of medically
acceptable contraception (i.e. either oral, topical, implanted hormonal
contraceptives, or an intrauterine device) or two barrier methods; have a negative
pregnancy test at Screening and Baseline/Day 1 and agree to use reliable methods
of contraception until at least 24-hours after the last dose of study drug. Women
who are surgically sterile (i.e. hysterectomy, bilateral oophorectomy, or tubal
ligation) or those who are post-menopausal for at least 2 years are not considered to
be of childbearing potential. Men who are not sterile (i.e. have not had a
vasectomy) must also agree to use contraception until at least 24-hours after the last
dose of study drug.