Sickle Cell DiseasePhase 2SUSTAINNCT01895361
Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
A Phase 2 study testing SelG1 in people with Sickle Cell Disease, run by Reprixys Pharmaceutical Corporation at 56 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- SelG1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 198 participants
- Where
- 56 sitesin 3 countries
- Study dates
- Jul 2013to Mar 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
56 sites in 3 countries.
56 sites in 3 countries
United States49 sites
Study site
Atlanta, Georgia
Study site
Augusta, Georgia
Study site
Aurora, Colorado
Study site
Baltimore, Maryland
Study site
Baton Rouge, Louisiana
Study site
Bethesda, Maryland
Study site
Birmingham, Alabama
Study site
Boston, Massachusetts
Study site
Brooklyn, New York
Study site
Chapel Hill, North Carolina
Study site
Charleston, South Carolina
Study site
Chicago, Illinois
Study site
Cleveland, Ohio
Study site
Columbus, Ohio
Study site
Daytona Beach, Florida
Study site
Detroit, Michigan
Study site
Durham, North Carolina
Study site
Fort Worth, Texas
Study site
Greenville, North Carolina
Study site
Houston, Texas
Study site
Indianapolis, Indiana
Study site
Jackson, Mississippi
Study site
Jacksonville, Florida
Study site
Kansas City, Missouri
Study site
Las Vegas, Nevada
Study site
Little Rock, Arkansas
Study site
Los Angeles, California
Study site
Louisville, Kentucky
Study site
Miami, Florida
Study site
Mobile, Alabama
Study site
New Brunswick, New Jersey
Study site
New Haven, Connecticut
Study site
New Hyde Park, New York
Study site
New Orleans, Louisiana
Study site
Newark, New Jersey
Study site
Norfolk, Virginia
Study site
Oakland, California
Study site
Oklahoma City, Oklahoma
Study site
Orange, California
Study site
Orlando, Florida
Study site
Peoria, Illinois
Study site
Philadelphia, Pennsylvania
Study site
Pittsburgh, Pennsylvania
Study site
Richmond, Virginia
Study site
Sacramento, California
Study site
Sumter, South Carolina
Study site
Tampa, Florida
Study site
The Bronx, New York
Study site
Washington D.C., District of Columbia
Brazil6 sites
Study site
Campinas, São Paulo
Study site
Porto Alegre, Rio Grande do Sul
Study site
Rio de Janeiro
Study site
Salvador, Estado de Bahia
Study site
São José Do Rio Preto, São Paulo
Study site
São Paulo
About this study
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
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)
- Conditions
- Sickle Cell Disease
- Being tested
- SelG1, Placebo
- Keywords
- 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
- Medical subject headings
- Anemia, Sickle Cell, Priapism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelG1Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| High-dose SelG1 (Selg1 5.0 mg/kg) | Experimental | IV Infusion, once every 4 weeks through Week 50 Receives: Drug: SelG1 |
| Low-dose SelG1 (Selg1 2.5 mg/kg) | Experimental | IV Infusion, once every 4 weeks through Week 50 Receives: Drug: SelG1 |
| Placebo | Placebo comparator | IV Infusion, once every 4 weeks through Week 50 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: One year
Secondary outcomes (6)
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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2013 (estimated)
- Study startJul 2013
- Primary completionMar 2016
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 198 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reprixys Pharmaceutical Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Food and Drug Administration (FDA)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01895361
- Sponsor's protocol ID
- SelG1-00005
- Other IDs
- R44HL093893, R01FD004805
- Acronym
- SUSTAIN
- Status last verified
- Jan 2020
- First submitted
- 3 Jul 2013
- Last updated
- 31 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
Publications
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.
PubMed 36529836Kanter 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.
PubMed 36355805Ataga 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.
PubMed 27959701
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01895361
- Acronym / sponsor ID
- SUSTAIN / SelG1-00005
- Conditions
- Sickle Cell Disease
- Keywords
- SelG1, P-selectin, monoclonal antibody, sickle cell disease, sickle cell anemia, sickle cell, pain crisis, pain crises
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 198 participants (actual)
- Lead sponsor
- Reprixys Pharmaceutical CorporationIndustry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Food and Drug Administration (FDA)
- Study start
- Jul 2013
- Primary completion
- Mar 2016 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 10 Jul 2013 (estimated)
- Results
- Posted 31 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| High-dose SelG1 (Selg1 5.0 mg/kg) | Experimental | Drug: SelG1IV Infusion, once every 4 weeks through Week 50 |
| Low-dose SelG1 (Selg1 2.5 mg/kg) | Experimental | Drug: SelG1IV Infusion, once every 4 weeks through Week 50 |
| Placebo | Placebo comparator | Drug: PlaceboIV Infusion, once every 4 weeks through Week 50 |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SelG1 | Drug | High-dose SelG1 (Selg1 5.0 mg/kg), Low-dose SelG1 (Selg1 2.5 mg/kg) |
| Placebo | Drug | Placebo |
Primary outcome
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.
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.
6 secondary outcomes
Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median
Time frame: One year
Time to First Sickle Cell-related Pain Crisis
Time frame: Up to one year
Time to Second Sickle Cell-related Pain Crisis
Time frame: Up to one year
Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median
Time frame: Up to one year
Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median
Time frame: One year
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36529836Sy 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.
- Derived from this study: PubMed 36355805Kanter 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.
- Derived from this study: PubMed 27959701Ataga 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.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
56 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SelG1 in people with Sickle Cell Disease, run by Reprixys Pharmaceutical Corporation at 56 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 56 sites in United States, Brazil, Jamaica. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median (time frame: One year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01895361, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2020.