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Trial record 1 of 1 for:    SelG1
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Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises (SUSTAIN)

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
Recruitment Status : Completed
First Posted : July 10, 2013
Results First Posted : January 31, 2020
Last Update Posted : January 31, 2020
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
Reprixys Pharmaceutical Corporation

Brief 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)


Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: SelG1 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 198 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Study Start Date : July 2013
Actual Primary Completion Date : March 2016
Actual Study Completion Date : March 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: High-dose SelG1 (Selg1 5.0 mg/kg)
IV Infusion, once every 4 weeks through Week 50
Drug: SelG1
Experimental: Low-dose SelG1 (Selg1 2.5 mg/kg)
IV Infusion, once every 4 weeks through Week 50
Drug: SelG1
Placebo Comparator: Placebo
IV Infusion, once every 4 weeks through Week 50
Drug: Placebo



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

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


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

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

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

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

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

  6. 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 ]
    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.



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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01895361


Locations
Show Show 56 study locations
Sponsors and Collaborators
Reprixys Pharmaceutical Corporation
National Heart, Lung, and Blood Institute (NHLBI)
Food and Drug Administration (FDA)
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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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 Posted: July 10, 2013    Key Record Dates
Results First Posted: January 31, 2020
Last Update Posted: January 31, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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.

URL: https://www.clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Reprixys Pharmaceutical Corporation:
SelG1
P-selectin
monoclonal antibody
sickle cell disease
sickle cell anemia
sickle cell
pain crisis
pain crises
vasoocclusion
vaso-occlusion
priapism
hepatic sequestration
splenic sequestration
chest syndrome
SCD
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn