A Phase 2 interventional study of SelG1 and Placebo in Sickle Cell Disease, sponsored by Reprixys Pharmaceutical Corporation. Completed at 56 sites in 3 countries. Open to participants aged 16 Years to 65 Years. Per ClinicalTrials.gov, last updated 2020-01-31.
Sponsored by Reprixys Pharmaceutical Corporation · Phase 2, Interventional, and Treatment
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)
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 198 is above the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →This is the only study on the registry with Reprixys Pharmaceutical Corporation as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
IV Infusion, once every 4 weeks through Week 50
Drug: SelG1
IV Infusion, once every 4 weeks through Week 50
Drug: SelG1
IV Infusion, once every 4 weeks through Week 50
Drug: Placebo
Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
Time frame: One year
Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
Time frame: One year
Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median
The annual rate of days hospitalized was calculated as the number of days hospitalized multiplied by 365 divided by the end date minus the date of randomization plus one where the end date is defined as the last dose date plus 14 days (for subjects never dosed, the end date equaled the end of study date, which was the last site contact for these patients).
Time frame: One year
Time to First Sickle Cell-related Pain Crisis
Time to first SCPC is defined as months from randomization to first SCPC. A participant without SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For participants never dosed, the end date is the end of study date.
Time frame: Up to one year
Time to Second Sickle Cell-related Pain Crisis
Time to second SCPC is defined as months from randomization to second SCPC. A patient with less than two SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For patients never dosed, the end date is the end of study date.
Time frame: Up to one year
Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median
Uncomplicated SCPC is defined as an acute episode of pain with no known cause for pain other than a vasoocclusive event; requiring a visit to a medical facility; and requiring treatment with a parenteral or oral narcotic (including opiates), or parenteral NSAIDs; but is NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism.
Time frame: Up to one year
Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median
Acute Chest Syndrome (ACS) is defined on the basis of the finding of a new pulmonary infiltrate involving at least one complete lung segment that was consistent with alveolar consolidation, but excluding atelectasis (as indicated by chest X-ray). At least one of the following additional signs or symptoms needs to be present as well: a participant had to have reported chest pain, a temperature of more than 38.5oC, tachypnea, wheezing or cough.
Time frame: One year
Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire
The BPI instrument was completed by the patients at pre-specified study visits prior to \& during the Treatment \& Follow-Up Evaluation Phases. Patients completed the brief pain inventory long-form, 1-week recall at the indicated pre-specified study visits. The BPI is a standardized self-reported questionnaire developed to provide information on the intensity of pain (the sensory dimension) as well as the degree to which pain interferes with function (the reactive dimension). The BPI also asks questions about pain relief, pain quality, \& the patient's perception of the cause of pain. Since pain can be quite variable over a day, the BPI asks patients to rate their pain at the time of responding to the questionnaire (pain now), \& also at its worst, least, \& average over the previous week. The scorings for pain \& interference have a range from 0 (no pain/no interference) to 10 (worst pain/complete interference). The BPI scoring manual was used to calculate scores for each domain.
Time frame: Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks
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).
| Milestone | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Started | 67 | 66 | 65 |
| Completed | 43 | 45 | 41 |
| Not completed | 24 | 21 | 24 |
| Withdrew: Adverse event | 1 | 1 | 3 |
| Withdrew: Death | 2 | 1 | 2 |
| Withdrew: Lost to follow-up | 4 | 4 | 6 |
| Withdrew: Non-compliance with study | 1 | 3 | 1 |
| Withdrew: Physician decision | 2 | 2 | 2 |
| Withdrew: Withdrawal by subject | 7 | 6 | 6 |
| Withdrew: Lack of efficacy | 0 | 1 | 0 |
| Withdrew: Reasons different from categories above | 7 | 3 | 4 |
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
| SCPC per year | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median | 1.63 (0.0 to 24.3) | 2.01 (0.0 to 24.3) | 2.98 (0.0 to 24.3) |
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
| SCPC per year | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median | 1.63 (0.0 to 24.3) | 2.01 (0.0 to 24.3) | 2.98 (0.0 to 24.3) |
The annual rate of days hospitalized was calculated as the number of days hospitalized multiplied by 365 divided by the end date minus the date of randomization plus one where the end date is defined as the last dose date plus 14 days (for subjects never dosed, the end date equaled the end of study date, which was the last site contact for these patients).
| Days hospitalized per year | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median | 4.00 (0.0 to 130.7) | 6.87 (0.0 to 151.0) | 6.87 (0.0 to 307.4) |
Time to first SCPC is defined as months from randomization to first SCPC. A participant without SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For participants never dosed, the end date is the end of study date.
| months | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Time to First Sickle Cell-related Pain Crisis | 4.07 (1.31 to NA) | 2.20 (0.95 to 6.60) | 1.38 (0.39 to 4.90) |
Time to second SCPC is defined as months from randomization to second SCPC. A patient with less than two SCPC before withdrawal or completion of the study is considered censored at the time of the end date. End date is defined as the last dose plus 14 days. For patients never dosed, the end date is the end of study date.
| months | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Time to Second Sickle Cell-related Pain Crisis | 10.32 (4.47 to NA) | 9.20 (3.94 to 12.16) | 5.09 (2.96 to 11.01) |
Uncomplicated SCPC is defined as an acute episode of pain with no known cause for pain other than a vasoocclusive event; requiring a visit to a medical facility; and requiring treatment with a parenteral or oral narcotic (including opiates), or parenteral NSAIDs; but is NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism.
| Uncomplicated SCPC per year | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median | 1.08 (0.0 to 24.3) | 2.00 (0.0 to 24.3) | 2.91 (0.0 to 19.2) |
Acute Chest Syndrome (ACS) is defined on the basis of the finding of a new pulmonary infiltrate involving at least one complete lung segment that was consistent with alveolar consolidation, but excluding atelectasis (as indicated by chest X-ray). At least one of the following additional signs or symptoms needs to be present as well: a participant had to have reported chest pain, a temperature of more than 38.5oC, tachypnea, wheezing or cough.
| accute chest syndrome per year | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median | 0.00 (0.0 to 3.7) | 0.00 (0.0 to 3.9) | 0.00 (0.0 to 24.3) |
The BPI instrument was completed by the patients at pre-specified study visits prior to \& during the Treatment \& Follow-Up Evaluation Phases. Patients completed the brief pain inventory long-form, 1-week recall at the indicated pre-specified study visits. The BPI is a standardized self-reported questionnaire developed to provide information on the intensity of pain (the sensory dimension) as well as the degree to which pain interferes with function (the reactive dimension). The BPI also asks questions about pain relief, pain quality, \& the patient's perception of the cause of pain. Since pain can be quite variable over a day, the BPI asks patients to rate their pain at the time of responding to the questionnaire (pain now), \& also at its worst, least, \& average over the previous week. The scorings for pain \& interference have a range from 0 (no pain/no interference) to 10 (worst pain/complete interference). The BPI scoring manual was used to calculate scores for each domain.
| score on a scale | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo |
|---|---|---|---|
| Pain Severity: Baseline (BL) (n=48,49,55) | 4.363 ± 2.1176 | 4.531 ± 2.0089 | 4.129 ± 2.0076 |
| Pain Severity: CFB to Day 15 (n=38,42,47) | -0.123 ± 1.3419 | 0.073 ± 1.5097 | 0.355 ± 1.7289 |
| Pain Severity: CFB to Week (Wk) 14 (n=32,33,33) | -0.146 ± 1.1520 | -0.068 ± 1.4608 | -0.152 ± 2.0918 |
| Pain Severity: CFB to Week 26 (n=27,31,32) | -0.377 ± 1.2460 | -0.290 ± 1.9865 | -0.563 ± 2.3751 |
| Pain Severity: CFB to Week 38 (n=25,29,29) | -0.267 ± 1.4079 | 0.026 ± 1.3893 | 0.333 ± 1.8430 |
| Pain Severity: CFB to Week 52 (n=18,23,22) | -0.634 ± 1.8501 | 0.130 ± 1.5899 | -0.310 ± 1.9508 |
| Pain Severity: CFB to Wk 58 follow up (n=27,33,30) | -0.145 ± 1.2309 | 0.091 ± 1.3790 | -0.444 ± 1.8626 |
| Pain Interference: BL (n=48,49,55) | 4.643 ± 2.5726 | 4.656 ± 2.6099 | 4.995 ± 2.9470 |
| Pain Interence: CFB to Day 15 (n=38,42,47) | -0.674 ± 2.2868 | -0.099 ± 2.0435 | -0.816 ± 2.3556 |
| Pain Interence: CFB to Wk 14 (n=32,33,33) | -0.213 ± 2.3988 | -0.534 ± 2.7769 | -0.039 ± 3.0412 |
| Pain Interference: CFB to Wk 26 (n=27,32,32) | -0.583 ± 2.2844 | -0.728 ± 2.4220 | -0.821 ± 3.1561 |
| Pain Interference: CFB to Wk 38 (n=25,29,29) | -0.866 ± 2.7720 | -0.119 ± 1.6473 | -0.221 ± 3.1076 |
| Pain Interference: CFB to Wk 52 (n=18,23,22) | -1.014 ± 2.0989 | -0.174 ± 2.2510 | -0.819 ± 2.8490 |
| Pain Interference: CFB to Wk 58 f/up (n=27,33,30) | -0.476 ± 2.3473 | -0.386 ± 2.2249 | -0.802 ± 2.5785 |
Collected over Adverse Event (AE) timeframe: Adverse events were collected from first dose of study treatment until end of study treatment, up to a maximum duration of 58 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| High-dose SelG1 (5.0 mg/kg SelG1) | 2/66 (3%) | 17/66 (25.8%) | 46/66 (69.7%) |
| Low-dose SelG1 (2.5 mg/kg SelG10 | 1/64 (1.6%) | 21/64 (32.8%) | 47/64 (73.4%) |
| Placebo | 2/62 (3.2%) | 17/62 (27.4%) | 42/62 (67.7%) |
| Event | High-dose SelG1 (5.0 mg/kg SelG1) | Low-dose SelG1 (2.5 mg/kg SelG10 | Placebo |
|---|---|---|---|
| PNEUMONIAInfections and infestations | 3/66 | 2/64 | 3/62 |
| URINARY TRACT INFECTIONInfections and infestations | 1/66 | 1/64 | 3/62 |
| INFLUENZAInfections and infestations | 0/66 | 3/64 | 0/62 |
| SICKLE CELL ANAEMIA WITH CRISISCongenital, familial and genetic disorders | 1/66 | 0/64 | 2/62 |
| PYREXIAGeneral disorders | 2/66 | 0/64 | 1/62 |
| LEUKOCYTOSISBlood and lymphatic system disorders | 0/66 | 0/64 | 1/62 |
| RIGHT VENTRICULAR FAILURECardiac disorders | 0/66 | 0/64 | 1/62 |
| DIARRHOEAGastrointestinal disorders | 0/66 | 0/64 | 1/62 |
| NAUSEAGastrointestinal disorders | 0/66 | 0/64 | 1/62 |
| VOMITINGGastrointestinal disorders | 0/66 | 0/64 | 1/62 |
| Event | High-dose SelG1 (5.0 mg/kg SelG1) | Low-dose SelG1 (2.5 mg/kg SelG10 | Placebo |
|---|---|---|---|
| HEADACHENervous system disorders | 11/66 | 14/64 | 10/62 |
| BACK PAINMusculoskeletal and connective tissue disorders | 10/66 | 12/64 | 7/62 |
| NAUSEAGastrointestinal disorders | 12/66 | 11/64 | 7/62 |
| ARTHRALGIAMusculoskeletal and connective tissue disorders | 12/66 | 9/64 | 5/62 |
| PAIN IN EXTREMITYMusculoskeletal and connective tissue disorders | 11/66 | 8/64 | 10/62 |
| URINARY TRACT INFECTIONInfections and infestations | 8/66 | 6/64 | 6/62 |
| MUSCULOSKELETAL PAINMusculoskeletal and connective tissue disorders | 8/66 | 4/64 | 6/62 |
| COUGHRespiratory, thoracic and mediastinal disorders | 4/66 | 4/64 | 7/62 |
| VOMITINGGastrointestinal disorders | 5/66 | 7/64 | 3/62 |
| CHEST PAINGeneral disorders | 1/66 | 7/64 | 1/62 |
Intent-to-Treat (ITT): The ITT population includes all randomized patients.
| Age, Continuous(years) | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo | Total |
|---|---|---|---|---|
| Mean | 30.9 ± 10.89 | 30.1 ± 9.79 | 29.3 ± 10.36 | 30.1 ± 10.33 |
| Sex: Female, Male(Participants) | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo | Total |
|---|---|---|---|---|
| Female | 35 | 36 | 38 | 109 |
| Male | 32 | 30 | 27 | 89 |
| Ethnicity (NIH/OMB)(Participants) | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo | Total |
|---|---|---|---|---|
| Hispanic or Latino | 20 | 12 | 11 | 43 |
| Not Hispanic or Latino | 45 | 52 | 53 | 150 |
| Unknown or Not Reported | 2 | 2 | 1 | 5 |
| Race/Ethnicity, Customized(participants) | High-dose SelG1 (Selg1 5.0 mg/kg) | Low-dose SelG1 (Selg1 2.5 mg/kg) | Placebo | Total |
|---|---|---|---|---|
| Black or African American | 60 | 62 | 60 | 182 |
| White | 4 | 2 | 3 | 9 |
| Other | 3 | 2 | 2 | 7 |
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.
Data sharing details ↗ · www.clinicalstudydatarequest.com
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