Phase 2
N=198
Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT01895361 ↗Enrolled (actual)
198
Serious AEs
28.7%
Results posted
Jan 2020
Primary outcome: Primary: Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median — 1.63; 2.01; 2.98 SCPC per year — p== 0.010
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SelG1 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Reprixys Pharmaceutical Corporation
- Primary completion
- Mar 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median |
1.63; 2.01; 2.98 | = 0.010 sig |
| PRIMARY Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median |
1.63; 2.01; 2.98 | — |
| SECONDARY Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median |
4.00; 6.87; 6.87 | = 0.450 |
| SECONDARY Time to First Sickle Cell-related Pain Crisis |
4.07; 2.20; 1.38 | = 0.001 sig |
| SECONDARY Time to Second Sickle Cell-related Pain Crisis |
10.32; 9.20; 5.09 | = 0.022 sig |
| SECONDARY Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median |
1.08; 2.00; 2.91 | = 0.015 sig |
| SECONDARY Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median |
0.00; 0.00; 0.00 | = 0.780 |
| SECONDARY Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire |
4.363; 4.531; 4.129; -0.123; 0.073; 0.355 | — |
Summary
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream.
Funding Source - FDA Office of Orphan Products Development (OOPD)
Eligibility Criteria
Key Inclusion Criteria
- Sickle Cell Disease (HbSS, HbSC, HbSβ⁰-thalassemia, or HbSβ⁺-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
Data sourced from ClinicalTrials.gov (NCT01895361). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.