Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01895361
• Date of registration: 03/07/2013
• Prospective Registration: No
• Primary sponsor: Reprixys Pharmaceutical Corporation
• Public title: Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises SUSTAIN
• Scientific title: A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind, 12-Month Study to Assess Safety and Efficacy of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Sickle Cell-Related Pain Crises
• Date of first enrolment: July 2013
• Target sample size: 198
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01895361
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Brazil, Jamaica, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Sickle Cell Disease (HbSS, HbSC, HbSß°-thalassemia, or HbSß?-thalassemia)

- If receiving hydroxyurea or erythropoietin, treatment must have been prescribed for at
least 6 months, with the dose stable for at least 3 months

- Between 2 and 10 sickle cell-related pain crises in the past 12 months

Key Exclusion Criteria:

- On a chronic transfusion program or planning on exchange transfusion during the study

- Hemoglobin <4.0 g/dL

- Planned initiation, termination, or dose alteration of hydroxyurea during the study

- Receiving chronic anticoagulation therapy (e.g. warfarin, heparin) other than aspirin



Age minimum: 16 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sickle Cell Disease

Intervention(s)

Drug: SelG1

Drug: Placebo

Primary Outcome(s)

Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median [Time Frame: One year]

Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median [Time Frame: One year]

Secondary Outcome(s)

Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median [Time Frame: One year]

Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median [Time Frame: One year]

Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median [Time Frame: Up to one year]

Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire [Time Frame: Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks]

Time to First Sickle Cell-related Pain Crisis [Time Frame: Up to one year]

Time to Second Sickle Cell-related Pain Crisis [Time Frame: Up to one year]

Secondary ID(s)

R44HL093893

R01FD004805

SelG1-00005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Heart, Lung, and Blood Institute (NHLBI)

Food and Drug Administration (FDA)

Results

• Results available: Yes
• Date Posted: 31/01/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01895361