Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
SUSTAIN
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
3 other identifiers
interventional
198
3 countries
56
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)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jul 2013
56 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2013
CompletedFirst Submitted
Initial submission to the registry
July 3, 2013
CompletedFirst Posted
Study publicly available on registry
July 10, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2016
CompletedResults Posted
Study results publicly available
January 31, 2020
CompletedJanuary 31, 2020
January 1, 2020
2.7 years
July 3, 2013
December 12, 2019
January 21, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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.
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.
One year
Secondary Outcomes (6)
Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median
One year
Time to First Sickle Cell-related Pain Crisis
Up to one year
Time to Second Sickle Cell-related Pain Crisis
Up to one year
Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median
Up to one year
Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median
One year
- +1 more secondary outcomes
Study Arms (3)
High-dose SelG1 (Selg1 5.0 mg/kg)
EXPERIMENTALIV Infusion, once every 4 weeks through Week 50
Low-dose SelG1 (Selg1 2.5 mg/kg)
EXPERIMENTALIV Infusion, once every 4 weeks through Week 50
Placebo
PLACEBO COMPARATORIV Infusion, once every 4 weeks through Week 50
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (56)
Unknown Facility
Birmingham, Alabama, United States
Unknown Facility
Mobile, Alabama, United States
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Little Rock, Arkansas, United States
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Los Angeles, California, United States
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Oakland, California, United States
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Orange, California, United States
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Sacramento, California, United States
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Aurora, Colorado, United States
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New Haven, Connecticut, United States
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Washington D.C., District of Columbia, United States
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Daytona Beach, Florida, United States
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Jacksonville, Florida, United States
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Miami, Florida, United States
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Orlando, Florida, United States
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Tampa, Florida, United States
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Atlanta, Georgia, United States
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Augusta, Georgia, United States
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Chicago, Illinois, United States
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Peoria, Illinois, United States
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Indianapolis, Indiana, United States
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Louisville, Kentucky, United States
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Baton Rouge, Louisiana, United States
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New Orleans, Louisiana, United States
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Baltimore, Maryland, United States
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Bethesda, Maryland, United States
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Boston, Massachusetts, United States
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Detroit, Michigan, United States
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Jackson, Mississippi, United States
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Kansas City, Missouri, United States
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Las Vegas, Nevada, United States
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New Brunswick, New Jersey, United States
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Newark, New Jersey, United States
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Brooklyn, New York, United States
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New Hyde Park, New York, United States
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The Bronx, New York, United States
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Chapel Hill, North Carolina, United States
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Durham, North Carolina, United States
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Greenville, North Carolina, United States
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Cleveland, Ohio, United States
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Columbus, Ohio, United States
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Oklahoma City, Oklahoma, United States
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Philadelphia, Pennsylvania, United States
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Pittsburgh, Pennsylvania, United States
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Charleston, South Carolina, United States
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Sumter, South Carolina, United States
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Fort Worth, Texas, United States
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Houston, Texas, United States
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Norfolk, Virginia, United States
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Richmond, Virginia, United States
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Salvador, Estado de Bahia, Brazil
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Porto Alegre, Rio Grande do Sul, Brazil
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Campinas, São Paulo, Brazil
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São José Do Rio Preto, São Paulo, Brazil
Unknown Facility
Rio de Janeiro, Brazil
Unknown Facility
São Paulo, Brazil
Unknown Facility
Kingston, Jamaica
Related Publications (3)
Sy SKB, Tanaka C, Grosch K. Population Pharmacokinetics and Pharmacodynamics of Crizanlizumab in Healthy Subjects and Patients with Sickle Cell Disease. Clin Pharmacokinet. 2023 Feb;62(2):249-266. doi: 10.1007/s40262-022-01193-4. Epub 2022 Dec 18.
PMID: 36529836DERIVEDKanter J, Brown RC, Norris C, Nair SM, Kutlar A, Manwani D, Shah N, Tanaka C, Bodla S, Sanchez-Olle G, Albers U, Liles D. Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. Blood Adv. 2023 Mar 28;7(6):943-952. doi: 10.1182/bloodadvances.2022008209.
PMID: 36355805DERIVEDAtaga KI, Kutlar A, Kanter J, Liles D, Cancado R, Friedrisch J, Guthrie TH, Knight-Madden J, Alvarez OA, Gordeuk VR, Gualandro S, Colella MP, Smith WR, Rollins SA, Stocker JW, Rother RP. Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease. N Engl J Med. 2017 Feb 2;376(5):429-439. doi: 10.1056/NEJMoa1611770. Epub 2016 Dec 3.
PMID: 27959701DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
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
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 3, 2013
First Posted
July 10, 2013
Study Start
July 1, 2013
Primary Completion
March 1, 2016
Study Completion
March 1, 2016
Last Updated
January 31, 2020
Results First Posted
January 31, 2020
Record last verified: 2020-01
Data Sharing
- IPD Sharing
- Will share
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.