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Trial details
An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial
Current status:
Approved
|
Date registered:
18/02/2022
Trial version(s)
Current: 05/07/2021
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2021074838
Protocol number
GBT2104-133
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
05/07/2021
Primary sponsor
Global Blood Therapeutics, Inc.
Primary sponsor: Country of origin
USA
Public title
An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial
Acronym
Scientific title
An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants with Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial
Acronym
Brief summary of the study: English
This multicenter, global, open-label extension study is designed to assess the safety of long-term treatment with inclacumab in participants with sickle cell disease (SCD). The study will be conducted globally and will be available to eligible participants enrolled in a prior sponsored inclacumab clinical study. Participants must have completed participation in their originating clinical study and must meet the entry criteria for this study to be eligible for enrollment. The primary objective of this study is to evaluate the long-term safety of every 12-week dosing of inclacumab in participants with SCD who have completed a prior inclacumab clinical trial.
Brief summary of the study: Arabic
تم تصميم هذه الدراسة الموسعة متعددة المراكز والعالمية والمفتوحة التسمية لتقييم سلامة العلاج طويل الأمد باستخدام inclacumab للمشاركين المصابين بمرض الخلايا المنجلية. سيتم إجراء الدراسة على مستوى العالم وستكون متاحة للمشاركين المؤهلين المسجلين في دراسة inclacumab إكلينيكية سابقة لنفس الراعي. يجب أن يكون المشاركون قد أكملوا المشاركة في دراستهم السريرية الأصلية ويجب أن يستوفوا معايير الدخول لهذه الدراسة ليكونوا مؤهلين للتسجيل. الغرض الرئيسي من هذه الدراسة هو تقييم سالمة وتأثير الجرعات المزمنة طويلة األمد من إنكالكوماب لدى المشاركين الذين شاركوا في دراسة سابقة على عقار إنكالكوماب.
Health conditions/problem studied: Specify
Up to approximately 520 participants who have completed a prior inclacumab clinical trial will be enrolled.
Interventions: Specify
This multicenter, global, open-label extension study is designed to assess the safety of long-term treatment with inclacumab in participants with SCD. The study will be conducted globally and will be available to eligible participants enrolled in a prior Global Blood Therapeutics, Inc. (GBT)-Sponsored inclacumab clinical study (originating study). Participants must have completed participation in their originating clinical study and must meet the entry criteria for this study to be eligible for enrollment. The study will be conducted at up to 150 global clinical sites, and up to approximately 520 participants will be enrolled. Eligible participants will receive inclacumab 30 mg/kg administered intravenously (IV) Q12W if they continue to receive clinical benefit that outweighs risk, as determined by the Investigator, until the participant has access to inclacumab from an alternative source (eg, through commercialization or a managed-access program). All participants will undergo safety and outcome assessments at Baseline (Day 1), Week 6, and every 12 weeks thereafter. Visits to the clinical site for infusion of study drug will occur at Baseline (Day 1) and every 12 weeks (Weeks 12, 24, 36, 48, etc.).
Key inclusion and exclusion criteria: Inclusion criteria
Participants who meet all the following criteria will be eligible for study enrollment: 1. Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study. 2. Participant has completed the originating inclacumab study within 30 calendar days of the Day 1 Visit. Participants who discontinued study drug in the originating study due to a non-study drug-related adverse event (AE), but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with inclacumab. 3. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become of childbearing potential during the study must be willing to have a negative urine pregnancy test to remain in the study. 4. If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 165 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 165 days after the last dose of study drug. 5. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant’s legal representative or legal guardian and the participant’s assent (where applicable) must be obtained based on local requirement.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
90
Key inclusion and exclusion criteria: Exclusion criteria
Participants meeting any of the following exclusion criteria will not be eligible for study enrollment: 1. Female participant who is breastfeeding or pregnant. 2. Participant had an infusion-related reaction (IRR) in the originating inclacumab clinical study. 3. Participant withdrew consent from the originating inclacumab clinical study. 4. Participant was lost to follow-up from the originating inclacumab clinical study. 5. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Safety
Trial scope: Specify scope
Study design: Allocation
Single Arm Study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
Inclacumab
Type of IMP
Immunological
Pharmaceutical class
Inclacumab is a recombinant human monoclonal antibody (huMAb) of the immunoglobulin (Ig)G4 subclass directed against human P-selectin, which is being developed by GBT, for the treatment of SCD. Inclacumab binds to P-selectin, which is a cell adhesion molecule produced by endothelial cells and platelets. Upon activation of these cells (e.g., by thrombin, cytokines, complement components, hypoxia, and heme), P-selectin is translocated to the cell surface where it binds to its primary ligand P-selectin glycoprotein ligand-1 (PSGL-1) and mediates leukocytes recruitment by platelets or endothelial cells. The same mechanism is also responsible for abnormal rolling and adhesion of sickle red blood cells (RBC) to the endothelium, initiating acute vascular occlusion and chronically impairing microvascular blood flow in patients with SCD. Inclacumab binding of P-selectin and prevention of P-selectin binding to its ligands is the putative mechanism by which inclacumab prevents the binding of sickle RBCs or leukocytes to endothelium.
Therapeutic indication
sickle cell disease (SCD)
Therapeutic benefit
Inclacumab is a recombinant huMAb of the IgG4 subclass directed against human P-selectin. The molecule is composed of two heterodimers, each composed of a heavy and a light polypeptide chain. The four polypeptide chains are linked together by disulfide bonds. To avoid antibody-dependent cell-mediated cytotoxicity and to improve structural stability, two single point mutations (L235E, S228P) were introduced into the Fc part of the molecule. The inclacumab drug substance is manufactured by fermentation cell culture using Chinese hamster ovary (CHO) cells followed by purification. The drug substance, drug product, and placebo are manufactured in accordance with Good Manufacturing Practices (GMP). Results from the SUSTAIN trial in patients with SCD showed that treatment with crizanlizumab, a humanized antibody to P-selectin, resulted in a significantly lower rate of sickle cell-related pain crises (i.e., vaso-occlusive crisis (VOC)) than placebo. These data validated P-selectin as a therapeutic target for SCD disease. Inclacumab is currently not approved by any health authority for the treatment of patients with any disease. Inclacumab is being developed to reduce the risk of vaso-occlusive crises in patients with SCD.
Biospecimen retention
None retained
Biospecimen description
Target sample size
520
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
01/10/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
31/12/2028
Recruitment status
Pending
Date of completion
31/12/2028
IPD sharing statement plan
Yes
IPD sharing statement description
Patient's full identity will not be on any of the study documents or sample collected and kept by the sponsor for their studies. Only the partial date of birth will be only collected. Only a unique participant number for the study will link the data or samples to the patients.
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
N.A
N.A
Sources of Monetary or Material Support
Name
Global Blood Therapeutics, Inc.
Secondary Sponsors
Name
N.A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Aziz
Zoghbi
MCT-CRO, Berytech Technology and Health, 5th Floor Damascus Road, Beirut, Lebanon
Lebanon
009613534 213
aziz.zoghbi@mct -cro.com
Director of Country Oversight and Manageme nt MENA, Gulf and Africa
Scientific
Miguel
Abboud
American University of Beirut Medical Center, Cairo Street, Hamra, Beirut, Lebanon
Lebanon
009613534 213
ma56@aub.edu.l b
PI
Scientific
Adlette
Inati
Nini Hospital, el Maarad Street, Triploli, Lebanon
Lebanon
009613228 033
adlette.inati@lau. edu.lb
PI
Scientific
Carolyn
Hoppe
181 Oyster Point Blvd. South San Francisco, CA 94080, USA
United States of America
+1 650 822 8728
choppe@gbt.com
Medical Monitor
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Nini Hospital
Adlette Inati
Professor of Pediatric Hematology and Oncology
Approved
American University of Beirut Medical Center
Miguel Abboud
Professor of Pediatric Hematology and Oncology
Pending
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Nini Hospital
23/06/2021
Nabil Kabbara
nabil.kabbara@hopitalnini.com
+961 (0) 6 431 400 ext 1062
Countries of Recruitment
Name
Health Conditions or Problems Studied
Condition
Code
Keyword
Sickle Cell Disease
Sickle-cell disorders (D57)
SCD
Interventions
Intervention
Description
Keyword
Inclacumab
All participants will receive inclacumab 30 mg/kg administered IV Q12W. All infusions of inclacumab will be administered at the study site.
Treatment
Primary Outcomes
Name
Time points
Measure
Safety
each visit
Incidence of treatment-emergent adverse events (TEAEs).
Safety
each visit
Change from Baseline in laboratory assessments (hematology, chemistry, and coagulation).
Safety
each visit
Vital signs and physical examination
Key Secondary Outcomes
Name
Time points
Measure
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial