A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety and Efficacy of Ubenimex in Patients with Pulmonary Arterial Hypertension (WHO Group I)
- Clements University Hospital
Sonja Bartolome, M.D.
This is a Phase 2, open-label, multicenter, extension study to evaluate long-term safety and efficacy of ubenimex (150 mg TiD) in patients with PaH (WHo Group 1) who complete Study eiG-uBX-001 and meet the eligibility criteria for Study eiG-uBX-002. Study eiG-uBX-001 is a Phase 2, randomized, double-blind, placebo-controlled clinical trial.
Patients may qualify to enter this extension study if they complete study drug treatment and study procedures through Week 24 in Study eiG-uBX-001 and meet the entry criteria for Study eiG-uBX-002. Data gathered at the Week 24 (end-of-Treatment) Visit will form the baseline data for eiG-uBX-002. Treatment in eiG-uBX-002 will continue until the last patient enrolled has received at least 24 weeks of open-label treatment with ubenimex. Clinical assessments will be performed on Week 0e, where [Quote]e[Quote] denotes the extension study eiG-uBX-002 (Week 24 of Study eiG-uBX-001) and on Weeks 4e, 8e, 12e, and 24e, and every 12 weeks thereafter until the end of study. The total duration of treatment for each patient will vary depending on when the patient enrolled and the
recruitment rate in Study eiG-uBX-001. at the end of the Treatment Period in eiG-uBX-002, patients will return 4 weeks after their last dose of ubenimex for a final Follow-up Visit.
endpoints, with the exception of time-to-event analyses, will be changes from eiG-uBX-001 baseline at the end of study compared with placebo.
* Treatment-emergent (Te) aes
* Te Saes
* Te treatment-related aes
* Te treatment-related Saes and deaths
* aes leading to early discontinuation of study treatment
* Te changes in clinical laboratory findings and eCGs
* Change in pulmonary vascular resistance (PVR).
* Change in exercise capacity as determined by the 6MWD.
* Change in Borg dyspnea score.
* Change in WHo/new York Heart association Functional Classification (WHo/nYHa-FC) group.
* Change in disease biomarkers: BnP/nT-proBnP
* PK analysis of trough levels
1. Provide a personally signed and dated informed consent form (ICF) indicating that the patient has been informed of all pertinent aspects of the study.
2. Patients must have completed Study EIG-UBX-001 through Week 24.
3. In the opinion of the Principal Investigator, has been generally compliant with study requirements (including adequate adherence to study drug dosing regimen, defined as having received at least 75% of ubenimex capsules) during Study EIG-UBX-001.
4. Agrees to use a medically acceptable method of contraception (both male and female patients) throughout the entire study period from informed consent through the follow-up visit if the possibility of conception exists. Medically acceptable methods of contraception
include oral contraceptives (starting >=2 months before dosing), diaphragm with vaginal spermicide, intrauterine device, condom and partner using vaginal spermicide, and surgical sterilization (>=6 months after surgery). Women who are surgically sterile or those who are postmenopausal for at least 2 years are not considered to be of childbearing potential.
5. Willing and able to comply with scheduled visits, treatment plans, and laboratory tests and other study procedures.