JSP191 (Briquilimab) in Subjects With LR-MDS

Study Overview
=================
Brief Summary
-----------------
A Phase 1 study in subjects with LR-MDS to assess the safety and tolerability of JSP191 as a second-line therapy.

Detailed Description
-----------------
An open-label, single-arm, dose-escalation study designed to determine the potential safety, efficacy, maximum tolerated dose (MTD) or optimal biologic dose (OBD), and recommended phase 2 dose (RP2D) of JSP191 (briquilimab) monotherapy for LR-MDS subjects with documented cytopenia (red blood cell-transfusion dependent, thrombocytopenia, and/or neutropenia).

Official Title
-----------------
A Phase 1 Open-label, Dose-escalation, Safety, and Tolerability Study of JSP191 as a Second-line Therapy in Subjects With Lower-Risk Myelodysplastic Syndrome (LR-MDS)

Conditions
-----------------
Lower-risk Myelodysplastic Syndrome

Intervention / Treatment
-----------------
* Drug: JSP191


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Age ≥ 18 years MDS with IPSS-R very low, low, or intermediate risk features Symptomatic cytopenias Women of childbearing potential (WOCBP) must agree to use an oral or implanted contraceptive, a double-barrier method of birth control, or an intrauterine device upon enrollment and through 3 months after receiving the last dose of JSP191 Women not of childbearing potential must be post-menopausal (no menstrual period for a minimum of 12 months) or surgically sterilized and have a negative serum pregnancy test upon study entry Male subjects must be surgically sterile or willing to use contraception upon enrollment and through 3 months after receiving the last dose of JSP191 Must be willing and able to provide informed consent Exclusion Criteria: Anemia secondary to iron deficiency, vitamin B12 deficiency, or folate deficiency Prior allogeneic or autologous stem cell transplant Known history of human immunodeficiency virus (HIV) (no laboratory testing is required), or active infection with hepatitis B or hepatitis C Pregnant women or women who are nursing and do not wish to discontinue breastfeeding Any other medical condition that, in the opinion of the Investigator, could pose a significant safety risk to the subject or jeopardize the integrity of the study Subjects who, in the opinion of the Investigator, may not be able to comply with the requirements of the study

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Interventional Model Description: 3 + 3 dose escalation/de-escalation design
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: JSP191<br>This study will explore up to 5 ascending dose levels (Cohorts 1, 2, 3, 4, and 5) and subjects will receive JSP191 on Day 1 on each 8-week cycle for 4 consecutive cycles. | Drug: JSP191<br>* Subjects will receive intravenous JSP191<br>* Other names: JSP191 (Briquilimab);|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of JSP191 | Assessed by the frequency, duration, and severity of adverse events | 32 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
LR-MDS, cytopenia