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Trial details
Trial details
An Open Label Extension Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects
Current status:
Approved
|
Date registered:
08/07/2019
Trial version(s)
History: 03/04/2019
Current: 03/04/2019
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2019070220
Protocol number
PTG300-03
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
19/06/2019
Primary sponsor
Protagonist Therapeutics Inc
Primary sponsor: Country of origin
USA
Public title
An Open Label Extension Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects
Acronym
Scientific title
An Open Label Extension Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects
Acronym
Brief summary of the study: English
This is an open-label, long term extension study for subjects completing study PTG-300-02. After completing the previous study, eligible subjects who choose to continue treatment may enroll in the PTG-300-03 study. The safety evaluation done at the end of study PTG-300-02 will be used to confirm subject eligibility for this study (see Screening in Study Procedure section). No interruption of PTG-300 treatment is expected with the transition between studies.
Brief summary of the study: Arabic
. PTG-300-03 بعد الانتهاء من الدراسة السابقة, قد يتم تسجيل الاشخاص المؤهلين الذين يختارون مواصلة العلاج في دراسة .PTG-300-02 دراسة تمديدية مفتوحة التسمية للاشخاص الذين اكملوا دراسة .لتاكيد اهلية الموضوع لهذه الدراسة .لا يتوقع اي انقطاع للعلاج مع الانتقال بين الدراسات PTG-300-02 سيتم استخدام تقييم السلامة الذي تم في نهاية الدراسة
Health conditions/problem studied: Specify
Chronic anemia due to ineffective erythropoiesis (IE) in subjects with β thalassemia
Interventions: Specify
Subjects rolling over from the PTG-300-02 study, who meet the response criteria defined for this study at the last dose received in study PTG-300-02, will continue to receive the same dose in PTG-300-03. Subjects who did not meet response criteria defined for this study at the last dose received in study PTG-300-02, will start PTG-300-03 at the next higher dose level.
Key inclusion and exclusion criteria: Inclusion criteria
1. NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02. 2. Women of childbearing potential (WOCBP) and men agree to use a highly effective contraceptive measure (base on the Clinical Trial Facilitation Group [CTFG]) during the duration of the study and for 4 weeks after the last dose of study drug in the case of women and 90 days after the last dose of study drug in the case of men. 3. For WOCBP, a negative urine pregnancy test within 24 hours prior to the first dose of study medication in this study. 4. Subjects or legal guardians (in the case of minors) understand the study procedures and agree to participate in the study by giving written informed consent. 5. Subjects, or legal representative (in the case of minors) are willing and able to adhere to the study visit schedule and other protocol requirements. 6. Subjects between 12-<18 years of age understand and provide the assent to participate in the study, according to local guidelines.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
65
Key inclusion and exclusion criteria: Exclusion criteria
1. Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD) 2. Clinically meaningful laboratory abnormalities at Screening. 3. Pregnant or lactating females. 4. Current history of alcohol dependence or illicit drug use. 5. Subject has a concurrent clinically significant, unstable or uncontrolled cardiovascular, pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic or other medical disorder that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the subject by their participation in the study. 6. Subject is mentally or legally incapacitated at the time of Screening visit or has a history of clinically significant psychiatric disorders that would impact the subject’s ability to participate in the trial according to the Investigator. 7. Concurrent participation in any other interventional study.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
N/A: Single arm study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
PTG-300
Type of IMP
Cell therapy
Pharmaceutical class
PTG-300 is a peptidic agent structurally related to natural hepcidin that mimics its inhibitory activity on ferroportin.
Therapeutic indication
β thalassemia
Therapeutic benefit
Administration of PTG-300 may result in iron redistribution in β-thalassemia subjects with potentially beneficial effects on erythropoiesis and consequently improvements in chronic anemia. This improvement in ineffective erythropoiesis may result in a clinical benefit both in NTD and in TD β-thalassemia subjects, by improving the symptomatology of the chronic anemia and the complications of the extramedullary hematopoiesis in the first group and by decreasing the need for transfusions in the latter.
Biospecimen retention
Samples without DNA
Biospecimen description
Blood samples taken throughout the study will be shipped to ICON lab in Ireland for analysis. These samples will be then stored at ICON Lab by Protagonist Therapeutics for up to 10 years.
Target sample size
84
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
05/08/2019
Date of study closure: Type
Anticipated
Date of study closure: Date
27/06/2022
Recruitment status
Pending
Date of completion
25/06/2020
IPD sharing statement plan
No
IPD sharing statement description
Not applicable
Additional data URL
none
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Food and Drug Administration
IND
Sources of Monetary or Material Support
Name
Protagonist Therapeutics.inc
Secondary Sponsors
Name
Not Applicable
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
009611612500
zog_az@mct-cro.com
Regional Manager
Scientific
Ali
Taher
Chronic Care Center, Hazmieh, Lebanon
Lebanon
009613755669
ataher@aub.edu.lb
PI
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Chronic Care Center
Dr. Ali Taher
Hematology/Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Chronic Care Center
01/06/2019
Michele Abi Saad
cccmas@chroniccare.org.lb
05-455101
Countries of Recruitment
Name
Lebanon
Thailand
United Kingdom
United States of America
Turkey
Tunisia
Malaysia
Greece
Italy
Health Conditions or Problems Studied
Condition
Code
Keyword
Thalassemia
Thalassaemia (D56)
Thalassemia
Interventions
Intervention
Description
Keyword
PTG-300
3mg/week
NA
PTG-300
10mg/week
NA
PTG-300
20mg/week
NA
PTG-300
40mg/week
NA
PTG-300
40 mg every 2 weeks
NA
PTG-300
80 mg/week
NA
PTG-300
80 mg every 2 weeks
NA
Primary Outcomes
Name
Time points
Measure
NTD Subjects who achieve an increase in Hgb ≥1.0 g/dL
from pre-treatment baseline without transfusion
Hemoglobin Test
NTD patients: Hgb change
from pre-treatment baseline
Hemoglobin Test
TD Subjects who achieve a ≥20% reduction in the red blood cell (RBC) units transfused over 8-week period compared to pre-treatment baseline
over an 8-week period
RBC units transfused
TD patients: Change in the number of units of RBC required
from pre-treatment baseline
RBC units transfused
Key Secondary Outcomes
Name
Time points
Measure
NTD patients: Proportion of subjects who achieve an increase in Hgb ≥1.5 g/dL
from pre-treatment baseline without transfusion
Hgb test
Proportion of subjects who achieve a maintenance dose
from pre-treatment baseline
Dose
Hgb level
from pre-treatment baseline
Hgb test
NTD Patients: Duration of Hgb change of ≥1.0 g/dL
from pre-treatment baseline without transfusion
Hgb test
NTD Patients: Duration of Hgb change of ≥1.5 g/dL from
from pre-treatment baseline without transfusion
Hgb test
Change in the following PD parameters
from pre-treatment baseline
serum iron, ferritin, transferrin saturation (TSAT)
TD Patients: Proportion of subjects who achieve ≥ 33% reduction in the RBC units required over an 8-week period
over an 8-week period
RBC units transfused
TD Patients: Duration of response defined as ≥ 20% reduction in the RBC units
over an 8-week period
RBC units transfused
TD Patients: Number of RBC units required
from pre-treatment baseline
RBC units transfused
TD Patients: Percent change in the RBC units required
from pre-treatment baseline
RBC units transfused
TD: Hgb change
from pre-treatment baseline
Hgb test
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
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