Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises

NCT ID: NCT01895361

Last Updated: 2020-01-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 198 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-07-31
• Study Completion Date: 2016-03-31

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)

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

High-dose SelG1 (Selg1 5.0 mg/kg)

IV Infusion, once every 4 weeks through Week 50

Group Type: EXPERIMENTAL

SelG1

Intervention Type: DRUG

Low-dose SelG1 (Selg1 2.5 mg/kg)

IV Infusion, once every 4 weeks through Week 50

Group Type: EXPERIMENTAL

SelG1

Intervention Type: DRUG

Placebo

IV Infusion, once every 4 weeks through Week 50

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Interventions

SelG1

Intervention Type: DRUG

Placebo

Intervention Type: DRUG

Eligibility Criteria

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

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

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Food and Drug Administration (FDA)

FED

Sponsor Role: collaborator

Reprixys Pharmaceutical Corporation

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Birmingham, Alabama, United States

Mobile, Alabama, United States

Little Rock, Arkansas, United States

Los Angeles, California, United States

Oakland, California, United States

Orange, California, United States

Sacramento, California, United States

Aurora, Colorado, United States

New Haven, Connecticut, United States

Washington D.C., District of Columbia, United States

Daytona Beach, Florida, United States

Jacksonville, Florida, United States

Miami, Florida, United States

Orlando, Florida, United States

Tampa, Florida, United States

Atlanta, Georgia, United States

Augusta, Georgia, United States

Chicago, Illinois, United States

Peoria, Illinois, United States

Indianapolis, Indiana, United States

Louisville, Kentucky, United States

Baton Rouge, Louisiana, United States

New Orleans, Louisiana, United States

Baltimore, Maryland, United States

Bethesda, Maryland, United States

Boston, Massachusetts, United States

Detroit, Michigan, United States

Jackson, Mississippi, United States

Kansas City, Missouri, United States

Las Vegas, Nevada, United States

New Brunswick, New Jersey, United States

Newark, New Jersey, United States

Brooklyn, New York, United States

New Hyde Park, New York, United States

The Bronx, New York, United States

Chapel Hill, North Carolina, United States

Durham, North Carolina, United States

Greenville, North Carolina, United States

Cleveland, Ohio, United States

Columbus, Ohio, United States

Oklahoma City, Oklahoma, United States

Philadelphia, Pennsylvania, United States

Pittsburgh, Pennsylvania, United States

Charleston, South Carolina, United States

Sumter, South Carolina, United States

Fort Worth, Texas, United States

Houston, Texas, United States

Norfolk, Virginia, United States

Richmond, Virginia, United States

Salvador, Estado de Bahia, Brazil

Porto Alegre, Rio Grande do Sul, Brazil

Campinas, São Paulo, Brazil

São José Do Rio Preto, São Paulo, Brazil

Rio de Janeiro, , Brazil

São Paulo, , Brazil

Kingston, , Jamaica

Countries

United States; Brazil; Jamaica

References

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.

Reference Type: DERIVED

PMID: 36529836 (View on PubMed)

Kanter 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.

Reference Type: DERIVED

PMID: 36355805 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 27959701 (View on PubMed)

Other Identifiers

R44HL093893

Identifier Type: NIH

Identifier Source: secondary_id

View Link

R01FD004805

Identifier Type: FDA

Identifier Source: secondary_id

View Link

SelG1-00005

Identifier Type: -

Identifier Source: org_study_id