This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease (HbSS or HbSβ0 genotypes). A total of 168 participants were randomized in a 1:1:1 ratio to receive NUV001 immediate-release (IR), NUV001 gastro-resistant (GR), or placebo, in addition to standard of care, for 90 days over 5 study visits. The primary objective was to assess safety and tolerability based on adverse events, clinical laboratory safety parameters, and vital signs. Exploratory secondary objectives evaluated hematologic, hemolysis, and patient-reported outcomes.
This was a multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study conducted in adult participants with sickle cell disease. Participants were randomized in a 1:1:1 ratio to one of three treatment groups: 1. NUV001 immediate-release (IR) 2. NUV001 gastro-resistant (GR) 3. Matching placebo All treatments were administered orally in addition to standard of care for 90 days. The study was designed to evaluate safety and tolerability, including adverse events, clinical laboratory safety parameters, and vital signs. Exploratory objectives included evaluation of hematologic parameters, markers of hemolysis, and patient-reported outcomes related to pain and quality of life. The study was conducted as a pilot, feasibility, and design-informing clinical study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
168
Daily supplementation with 1000 mg of NUV001 (in two administration orally) immediate release gel capsule formulation for 90 days in total.
Daily supplementation with 1000 mg of NUV001 (in two administration orally) gastro resistant gel capsule formulation for 90 days in total.
Placebo containing starch Powder (1000 mg, daily in two administration orally for 90 days).
Aman Hospital and Research Center
Vadodara, Gujarat, India
Kingsway Hospital
Nagpur, Maharashtra, India
Shivam Hospital
Ahmedabad, India
Sai Krupa Hospital & Research Centre
Ahmedabad, India
Thalassemia & Sickle Cell Society
Hyderabad, India
Index Medical College
Indore, India
NRSMC Hospital
Kolkata, India
Arihant Multispeciality Hospital
Nagpur, India
Shalinitai Meghe Hospital & Research Centre
Nagpur, India
Incidence of adverse events through Day 90.
Subject incidence of treatment-emergent adverse events through the treatment period.
Time frame: Baseline to Day 90
Change from baseline in hematologic and biochemical safety parameters at Day 90.
Change from baseline to Day 90 in complete blood count, blood glucose, calcium, electrolytes, total protein, albumin, alkaline phosphatase, bilirubin, blood urea nitrogen, creatinine, AST, ALT, and estimated glomerular filtration rate (eGFR).
Time frame: Baseline and Day 90
Change from baseline in vital signs at Day 90.
Change from baseline to Day 90 in systolic and diastolic blood pressure, pulse rate, respiration rate, and body temperature.
Time frame: Baseline and Day 90.
Change in percentage of HbF-positive cells.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in HbF content in Red Blood Cells.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in percentage of circulating irreversibly sickled cells.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in hematocrit.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in indirect bilirubin level.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in reticulocyte count.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in serum lactate dehydrogenase level.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in ASCQ-Me Questionnaire (Adult Sickle Cell Quality of Life Measurement Information System)
Questionnaire on acute and/or chronic pain, energy level, usage of pain medications and activity levels.
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in pain intensity score.
Evaluation of pain intensity for each body location (using a numeric pain rating scale from 0, no pain to 10 worst possible pain)
Time frame: Baseline, Day 30, Day 60, Day 90.
Change in pain relief score.
Evaluation and evaluation of pain relief (pain relief scale in percent from 0%, no relief to 100% complete relief)
Time frame: Baseline, Day 30, Day 60, Day 90.
Occurrence of vaso-occlusive crises during the study
Time frame: Day 0 to Day 90.
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