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An Open-label Extension Study of IMR-687 in Adult Patients with Sickle Cell Anemia (Homozygous HbSS or Sickle-ß0 Thalassemia) Who Participated in Study IMR-SCD-102
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19-148-2
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An Open-label Extension Study of IMR-687 in Adult Patients with Sickle Cell Anemia (Homozygous HbSS or Sickle-ß0 Thalassemia) Who Participated in Study IMR-SCD-102
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Dr. Susan Tannenbaum
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Study Title:An Open-label Extension Study of IMR-687 in Adult Patients with Sickle Cell Anemia (Homozygous HbSS or Sickle-B0 Thalassemia) Who Participated in Study IMR-SCD-102 Background: With a neonatal incidence of 294,000 to 330,000 patients worldwide (Piel 2013), SCA is a rare inherited disorder of red blood cells (RBCs) that is both serious and life threatening. The most common manifestations of SCA include vaso-occlusive crisis, chronic and acute severe pain, acute chest syndrome, stroke, priapism, acute anemia (particularly from aplastic crisis and splenic sequestration), increased susceptibility to infection, and progressive damage to major organs including the spleen, brain, kidney, heart, lung, skin, retina, vestibular cochlear systems, and bone. In developed countries where there is prenatal Screening and wide spread access to prophylactic and acute interventions, the median age of death remains in the 40s to 50s though many patients succumb to the disease much earlier (Platt 1994; Lanzkron 2013; Paulukonis 2016). Rationale for the study:Study IMR-SCD-102-EXT is an extension of Study IMR-SCD-102. During the extension, IMR687 100 mg will be administered orally once daily. Since Study IMR-SCD-102 is the first study in patients with SCA, an extension will provide long-term safety and tolerability data for these subjects. In addition, long-term pharamcodynamic (PD) and efficacy data will be collected. Study design: Patients are eligible for this open-label extension study immediately following the End-of-Study visit for Study IMR-SCD-102 (i.e., approximately 30 days after the last dose of study drug in the Phase 2a study). Following screening assessments to determine continued eligibility, patients will be enrolled into this extension study. IMR687 100 mg will be administered orally once daily. Patients will return to the study site for visits at week 1, and months 1, 4, 8, and 12 during the first year. Thereafter, patients will return to the study site every 6 months during years 2, 3, and 4. Telephone-based check-ins may be performed on weeks where there are no scheduled assessments or in the event of lack of patient availability for an in-person visit. Safety and concomitant medications will be monitored throughout the study; PD and clinical outcome measures will be performed at selected site visits. Study sample size:Approximately 70 patients currently participating in Study IMR-SCD-102 are expected to be eligible for this long-term extension. Major study intervention:The dose of IMR-687 to be administered in this extensions study is 100 mg per day as a single oral dose. This is the optimum dose expected being tested in the Phase 2a study and is considered to have positive pharmacodynamic effect, based on preclinical modelling and exposures in the FIH study of IMR-687 in healthy volunteers.Pending emerging data from Study IMR-SCD-102, the dose of IMR-687 may be adjusted in this study as appropriate. Main outcome measures/analyses: Primary objectives: To assess the long-term safety and tolerability of IMR-687 in adult patients with sickle cell anemia (SCA), defined as homozygous sickle hemoglobin (HbSS) or sickle-B0 thalassemia, who were previously enrolled in Study IMR-SCD-102. Exploratory objectives: To assess the pharmacodynamic (PD) effects of IMR-687 in adult patients with SCA who were previously enrolled in Study IMR-SCD-102. To assess the potential efficacy of IMR-687 on SCA-related clinical outcome measures in adult patients with SCA who were previously enrolled in Study IMR-SCD-102.
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Blood - Sickle Cell Disease
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Check with study contact
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Sasia-Marie Jones. Telephone: Not available. Email: sajones@uchc.edu
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Enrolling/recruiting. For current recruitment status, please check with study contact.
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