A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects with β-thalassemia: Population 1 (Transfusion Dependent Thalassemia (TDT) subjects) and Population 2 (Non-Transfusion Dependent Thalassemia (NTDT) subjects).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
122
IMR-687 Safety and Tolerability
Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events
Time frame: Baseline to Week 40
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 12 to Week 24
Proportion of patients with ≥33% hematological improvement (as measured by reduced transfusion burden) from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
Time frame: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions.
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 12 to Week 24 in the absence of transfusions.
Time frame: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 12 to Week 24 in absence of transfusions
Time frame: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 24 to Week 36
Proportion of patients with ≥33% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Time frame: Baseline to Week 36
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50 % Hematological Improvement From Week 12 to Week 24
Proportion of patients with ≥50% hematological improvement from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Herlev Hospital
Herlev, Capital Region, Denmark
Institut Universitaire du Cancer de Toulouse Oncopole
Toulouse, Haute-Garonn, France
Hôpital Edouard Herriot
Lyon, Rhone, France
Hôpital Necker-Enfants Malades
Paris, France
M. Zodelava Hematology Centre
Tbilisi, Borjomi, Georgia
National Center of Surgery
Tbilisi, Georgia
Medinvest - Institute of Hematology and Transfusiology
Tbilisi, Georgia
Aghia Sofia General Children's Hospital
Athens, Attica, Greece
Laiko General Hospital of Athens
Athens, Attica, Greece
Ippokrateio General Hospital of Thessaloniki
Thessaloniki, Central Macedonia, Greece
...and 26 more locations
Time frame: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50% Hematological Improvement From Week 24 to Week 36
Proportion of patients with ≥50% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Time frame: Baseline to Week 36
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 24 to Week 36 in the Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 24 to Week 36 in the absence of transfusions.
Time frame: Baseline to Week 36
NTDT: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 24 to Week 36 in Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 24 to Week 36 in absence of transfusions
Time frame: Baseline to Week 36