Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises (SUSTAIN)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01895361 |
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Recruitment Status :
Completed
First Posted : July 10, 2013
Results First Posted : January 31, 2020
Last Update Posted : January 31, 2020
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Sponsor:
Reprixys Pharmaceutical Corporation
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
Reprixys Pharmaceutical Corporation
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| Study Type | Interventional |
|---|---|
| Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
| Condition |
Sickle Cell Disease |
| Interventions |
Drug: SelG1 Drug: Placebo |
| Enrollment | 198 |
Participant Flow
| Recruitment Details | Approx. 174 patients were planned. A total of 198 patients were randomized. 192 patients received at least 1 dose of study drug & were analyzed for safety; 198 patients were analyzed for efficacy, 125 patients contributed data to the analysis of SelG1 PK data, & 176 patients contributed data to the analysis of PD data (% P-selectin inhibition). |
| Pre-assignment Details | The study included a Screening Phase, Treatment Phase, and Follow-up Evaluation Phase. During the Screening Phase, potential study participants were to be fully screened for both inclusion and exclusion criteria before and undergo clinical and laboratory evaluations within 30 days prior to randomization into the study. |
| Arm/Group Title | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
 Arm/Group Description |
IV Infusion, once every 4 weeks through Week 50 SelG1 |
IV Infusion, once every 4 weeks through Week 50 SelG1 |
IV Infusion, once every 4 weeks through Week 50 Placebo |
| Period Title: Overall Study | |||
| Started | 67 | 66 | 65 |
| Completed | 43 | 45 | 41 |
| Not Completed | 24 | 21 | 24 |
| Reason Not Completed | |||
| Adverse Event | 1 | 1 | 3 |
| Death | 2 | 1 | 2 |
| Lost to Follow-up | 4 | 4 | 6 |
| Non-compliance with study | 1 | 3 | 1 |
| Physician Decision | 2 | 2 | 2 |
| Withdrawal by Subject | 7 | 6 | 6 |
| Lack of Efficacy | 0 | 1 | 0 |
| Reasons different from categories above | 7 | 3 | 4 |
Baseline Characteristics
| Arm/Group Title | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo | Total | |
|---|---|---|---|---|---|
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Arm/Group Description |
IV Infusion, once every 4 weeks through Week 50 SelG1 |
IV Infusion, once every 4 weeks through Week 50 SelG1 |
IV Infusion, once every 4 weeks through Week 50 Placebo |
Total of all reporting groups | |
| Overall Number of Baseline Participants | 67 | 66 | 65 | 198 | |
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Baseline Analysis Population Description |
Intent-to-Treat (ITT): The ITT population includes all randomized patients.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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| Number Analyzed | 67 participants | 66 participants | 65 participants | 198 participants | |
| 30.9 (10.89) | 30.1 (9.79) | 29.3 (10.36) | 30.1 (10.33) | ||
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 67 participants | 66 participants | 65 participants | 198 participants | |
| Female |
35 52.2%
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36 54.5%
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38 58.5%
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109 55.1%
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| Male |
32 47.8%
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30 45.5%
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27 41.5%
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89 44.9%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 67 participants | 66 participants | 65 participants | 198 participants | |
| Hispanic or Latino |
20 29.9%
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12 18.2%
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11 16.9%
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43 21.7%
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| Not Hispanic or Latino |
45 67.2%
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52 78.8%
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53 81.5%
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150 75.8%
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| Unknown or Not Reported |
2 3.0%
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2 3.0%
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1 1.5%
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5 2.5%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 67 participants | 66 participants | 65 participants | 198 participants |
| Black or African American | 60 | 62 | 60 | 182 | |
| White | 4 | 2 | 3 | 9 | |
| Other | 3 | 2 | 2 | 7 | |
Outcome Measures
Adverse Events
Limitations and Caveats
The Safety population includes all randomized patients who received at least one dose of study drug.
Although the safety is now coded with MedDRA 21.1, the CSR, when first published 1in 2016, used MedDRA 16.1 CFB = Change from Baseline
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
| Name/Title: | Study Director |
| Organization: | Novartis Pharmaceuticals |
| Phone: | 862-778-8300 |
| EMail: | novartis.email@novartis.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Reprixys Pharmaceutical Corporation |
| ClinicalTrials.gov Identifier: | NCT01895361 |
| Other Study ID Numbers: |
SelG1-00005 R44HL093893 ( U.S. NIH Grant/Contract ) R01FD004805 ( U.S. FDA Grant/Contract ) |
| First Submitted: | July 3, 2013 |
| First Posted: | July 10, 2013 |
| Results First Submitted: | December 12, 2019 |
| Results First Posted: | January 31, 2020 |
| Last Update Posted: | January 31, 2020 |