Dose-Escalation Study of SCD-101 in Sickle Cell Disease
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ClinicalTrials.gov Identifier: NCT02380079 |
Recruitment Status :
Recruiting
First Posted : March 5, 2015
Last Update Posted : February 21, 2021
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Sponsor:
Invenux, LLC
Collaborator:
State University of New York - Downstate Medical Center
Information provided by (Responsible Party):
Invenux, LLC
Tracking Information | |||||||||
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First Submitted Date ICMJE | February 23, 2015 | ||||||||
First Posted Date ICMJE | March 5, 2015 | ||||||||
Last Update Posted Date | February 21, 2021 | ||||||||
Actual Study Start Date ICMJE | February 2015 | ||||||||
Estimated Primary Completion Date | August 2021 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline [ Time Frame: From the time the participant is administered the first dose through the final follow-up (18 weeks) ] | ||||||||
Original Primary Outcome Measures ICMJE |
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline [ Time Frame: From the time the participant is administered the first dose through the final follow-up clinic visit, for a total of 6 weeks ] | ||||||||
Change History | |||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Dose-Escalation Study of SCD-101 in Sickle Cell Disease | ||||||||
Official Title ICMJE | Part A: Phase IB, Single Site, Dose-Escalation of SCD-101 and Part B: Randomized, Double-Blind, Placebo-Controlled Crossover of SCD-101 in Adults With Homozygous Sickle Cell Disease or S/Beta 0 Thalassemia. | ||||||||
Brief Summary | The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease. | ||||||||
Detailed Description | This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities. The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with a 28-day treatment phase and 14-day follow-up phase with five cohorts . Part B is a randomized, placebo-controlled, confirmatory 2x2 crossover cohort with a 28 day washout between periods, and a 28-day follow-up phase. |
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Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 1 | ||||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Crossover Assignment Intervention Model Description: 2x2 crossover Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE | Drug: SCD-101
Administered as gelatin capsules
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
60 | ||||||||
Original Estimated Enrollment ICMJE |
24 | ||||||||
Estimated Study Completion Date ICMJE | November 2021 | ||||||||
Estimated Primary Completion Date | August 2021 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 55 Years (Adult) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number ICMJE | NCT02380079 | ||||||||
Other Study ID Numbers ICMJE | INVX-SCD-101-11 | ||||||||
Has Data Monitoring Committee | No | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Invenux, LLC | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Invenux, LLC | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE | State University of New York - Downstate Medical Center | ||||||||
Investigators ICMJE |
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PRS Account | Invenux, LLC | ||||||||
Verification Date | February 2021 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |