NCT03797040

Open-label Phase I Study for PEP or Treatment of HS-ARS PLX-R18 for the Post-Exposure Prevention (PEP) or Treatment of Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS)

Open-label Phase I Study to Evaluate the Safety of PLX-R18 for the Post-Exposure Prevention (PEP) or Treatment of Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS)

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 27 (estimated)
• Sponsor: Pluristem Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The objective of the study is to evaluate the safety of intramuscular (IM) administration of PLX-R18 in subjects exposed to ionizing radiation and who are at risk of developing HS-ARS. Indication:Post-Exposure Prevention (PEP) or treatment of Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS) in subjects suspected to have been exposed to ionizing radiation.

Detailed description

This will be a Phase I, open-label safety study; each subject will receive two administrations of PLX-R18, 4 days apart. Each administration of PLX-R18 will contain 4 million cells/kg (up to a maximal dose of 400 million cells). The first administration should be preferably within 48 hours after suspected exposure and no later than 4 days after suspected exposure. The second administration will be provided 4 days after first administration. All subjects will be hospitalized for at least 24 hours after each administration of PLX-R18,for close monitoring.In order to minimize risks, subjects will be divided in 3 sequential cohorts: Cohort 1: 9 subjects, treated as soon as possible Cohort 2: 18 subjects, treated at least 12 hours following the first dose administered to the 9th subject Cohort 3: 33 subjects, treated at least 12 hours following the first dose administered to the 27th subject After the completion of each Cohort, stopping rules will be assessed. All subjects will receive PLX-R18 in addition to recommended care per physician discretion, based on the REMM guidelines (APPENDIX 2). The study will be comprised of 2 periods: Main study period - Subjects will be followed-up for 12 months and evaluated at the following time points after the first administration: Day 0 (first administration),Day 1, Day 2, Day 3, Day 4 (second administration), Day 5, Day 14 (2 weeks),Day 21 (3 weeks), Day 28 (4 weeks), Day 49 (7 weeks), Day 63 (9 weeks), Day 119(17 weeks), Day 182 (26 weeks), and Day 364 (52 weeks).Long-term survival follow-up -Week 52 to Week 260: During this period, patients will be followed-up for overall survival at: 104 weeks, 156 weeks, 208 weeks and 260 weeks.

Conditions

• Ionizing Radiation Exposure

Interventions

Type	Name	Description
BIOLOGICAL	Cell therapy	PLX-R18: Allogeneic ex vivo expanded placental stromal cells

Timeline

• Start date: 2021-01-01
• Primary completion: 2021-12-01
• Completion: 2021-12-01
• First posted: 2019-01-08
• Last updated: 2020-07-28

Regulatory

• FDA-regulated drug study