Clinical trial NCT01895361: Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
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
Lead sponsor: Reprixys Pharmaceutical Corporation
Sponsor class: INDUSTRY
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Food and Drug Administration (FDA)
First posted: 2013-07-10
Enrollment: 198
Sex: ALL
Minimum age (years): 16
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)


Eligibility 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

Primary outcomes:
measure: Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median
timeFrame: One year
measure: Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median
timeFrame: One year
Secondary outcomes:
measure: Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median
timeFrame: One year
measure: Time to First Sickle Cell-related Pain Crisis
timeFrame: Up to one year
measure: Time to Second Sickle Cell-related Pain Crisis
timeFrame: Up to one year
measure: Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median
timeFrame: Up to one year
measure: Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median
timeFrame: One year
measure: Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire
timeFrame: Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Mobile
country: United States
state: Alabama
city: Little Rock
country: United States
state: Arkansas
city: Los Angeles
country: United States
state: California
city: Oakland
country: United States
state: California
city: Orange
country: United States
state: California
city: Sacramento
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: New Haven
country: United States
state: Connecticut
city: Washington
country: United States
state: District of Columbia
city: Daytona Beach
country: United States
state: Florida
city: Jacksonville
country: United States
state: Florida
city: Miami
country: United States
state: Florida
city: Orlando
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Augusta
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Peoria
country: United States
state: Illinois
city: Indianapolis
country: United States
state: Indiana
city: Louisville
country: United States
state: Kentucky
city: Baton Rouge
country: United States
state: Louisiana
city: New Orleans
country: United States
state: Louisiana
city: Baltimore
country: United States
state: Maryland
city: Bethesda
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Detroit
country: United States
state: Michigan
city: Jackson
country: United States
state: Mississippi
city: Kansas City
country: United States
state: Missouri
city: Las Vegas
country: United States
state: Nevada
city: New Brunswick
country: United States
state: New Jersey
city: Newark
country: United States
state: New Jersey
city: Bronx
country: United States
state: New York
city: Brooklyn
country: United States
state: New York
city: New Hyde Park
country: United States
state: New York
city: Chapel Hill
country: United States
state: North Carolina
city: Durham
country: United States
state: North Carolina
city: Greenville
country: United States
state: North Carolina
city: Cleveland
country: United States
state: Ohio
city: Columbus
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Charleston
country: United States
state: South Carolina
city: Sumter
country: United States
state: South Carolina
city: Fort Worth
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: Norfolk
country: United States
state: Virginia
city: Richmond
country: United States
state: Virginia
city: Salvador
country: Brazil
state: Bahia
city: Porto Alegre
country: Brazil
state: Rio Grande Do Sul
city: Campinas
country: Brazil
state: São Paulo
city: São José do Rio Preto
country: Brazil
state: São Paulo
city: Rio de Janeiro
country: Brazil
city: São Paulo
country: Brazil
city: Kingston
country: Jamaica
Study officials:
References:
citation: Ataga 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.