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Trial record 1 of 1 for:    NCT01895361
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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)

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ClinicalTrials.gov Identifier: NCT01895361
Recruitment Status : Completed
First Posted : July 10, 2013
Last Update Posted : October 2, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Description
Brief Summary:

The purpose of this study is to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea is 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 will also be evaluated, as well as the safety of the drug and how long it stays 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

Study Design

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
Primary Completion Date : March 2016
Study Completion Date : March 2016

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: High-dose SelG1
IV Infusion, once every 4 weeks through Week 50
Drug: SelG1
Experimental: Low-dose SelG1
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


Outcome Measures

Primary Outcome Measures :
  1. Rate of sickle cell-related pain crises [ Time Frame: One year ]

Secondary Outcome Measures :
  1. Rate of sickle cell-related pain crises by concomitant hydroxyurea use [ Time Frame: One year ]
  2. Time to first sickle cell-related pain crisis [ Time Frame: Up to one year ]
  3. Time to second sickle cell-related pain crisis [ Time Frame: Up to one year ]
  4. Number of hospitalization days per year [ Time Frame: Up to one year ]
  5. Absolute change from baseline in hemoglobin [ Time Frame: One year ]
  6. Absolute change from baseline in lactate dehydrogenase [ Time Frame: One year ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, 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
Contacts and Locations

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


  Show 56 Study Locations
Sponsors and Collaborators
Reprixys Pharmaceutical Corporation
National Heart, Lung, and Blood Institute (NHLBI)
Food and Drug Administration
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Reprixys Pharmaceutical Corporation
ClinicalTrials.gov Identifier: NCT01895361     History of Changes
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
Last Update Posted: October 2, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: We are 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 review 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.

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

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors