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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:
National Heart, Lung, and Blood Institute (NHLBI)
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
Reprixys Pharmaceutical Corporation

Tracking Information
First Submitted Date  ICMJE July 3, 2013
First Posted Date  ICMJE July 10, 2013
Last Update Posted Date October 2, 2017
Study Start Date  ICMJE July 2013
Actual Primary Completion Date March 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 9, 2013)
Rate of sickle cell-related pain crises [ Time Frame: One year ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01895361 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 9, 2013)
  • Rate of sickle cell-related pain crises by concomitant hydroxyurea use [ 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 ]
  • Number of hospitalization days per year [ Time Frame: Up to one year ]
  • Absolute change from baseline in hemoglobin [ Time Frame: One year ]
  • Absolute change from baseline in lactate dehydrogenase [ Time Frame: One year ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
Official Title  ICMJE 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
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)

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE
  • Drug: SelG1
  • Drug: Placebo
Study Arms  ICMJE
  • Experimental: High-dose SelG1
    IV Infusion, once every 4 weeks through Week 50
    Intervention: Drug: SelG1
  • Experimental: Low-dose SelG1
    IV Infusion, once every 4 weeks through Week 50
    Intervention: Drug: SelG1
  • Placebo Comparator: Placebo
    IV Infusion, once every 4 weeks through Week 50
    Intervention: Drug: Placebo
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 12, 2016)
198
Original Estimated Enrollment  ICMJE
 (submitted: July 9, 2013)
174
Actual Study Completion Date  ICMJE March 2016
Actual Primary Completion Date March 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil,   Jamaica,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01895361
Other Study ID Numbers  ICMJE SelG1-00005
R44HL093893 ( U.S. NIH Grant/Contract )
R01FD004805 ( U.S. FDA Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
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.
Responsible Party Reprixys Pharmaceutical Corporation
Study Sponsor  ICMJE Reprixys Pharmaceutical Corporation
Collaborators  ICMJE
  • National Heart, Lung, and Blood Institute (NHLBI)
  • Food and Drug Administration (FDA)
Investigators  ICMJE Not Provided
PRS Account Reprixys Pharmaceutical Corporation
Verification Date September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP