ß-Thalassemia Subjects with Chronic Anemia (transfusion and non transfusion dependent) MedDRA version: 20.0 Level: PT Classification code 10043391 Term: Thalassaemia beta System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: PTG-300
Product Code: PN-8518A
Pharmaceutical Form: Injection
INN or Proposed INN: Not assigned
CAS Number: Not assigned
Current Sponsor code: PTG-300
Concentration unit: mg/ml milligram
Sponsors
Protagonist Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.NTD and TD ß-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-=65 years) no F.1.3.1 Number of subjects for this age range
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1.To assess the long-term safety and tolerability of PTG-300 in subjects with TD and NTD ß-thalassemia;Secondary Objective: 1.To assess the long-term efficacy of PTG-300 for treating chronic anemia in subjects with ß-thalassemia 2.To assess pharmacokinetic (PK), pharmacodynamic (PD) and immunogenicity with the long-term administration of PTG-300 ;Primary end point(s): NTD: •Proportion of responders, where responders are defined as subjects who achieve an increase in Hgb =1.0 g/dL from pre-treatment baseline without transfusion, confirmed by a successive measurement at least 1 week later •Hgb change from pre-treatment baseline TD: •Subjects who achieve a =20% reduction in the red blood cell (RBC) units transfused over an 8 week period compared to pre-treatment baseline •Change from pre-treatment baseline in the number of units of RBC required ;Timepoint(s) of evaluation of this end point: NTD subjects: every 4 weeks until subject responds or reaches the maximum dose of 80 mg weekly. TD subjects: every 8 weeks until subject responds or reaches the maximum dose of 80 mg weekly. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): NTD: •Proportion of subjects who achieve an increase in Hgb =1.5 g/dL from pre-treatment baseline without transfusion, confirmed by a successive measurement at least 1 week later •Proportion of subjects who achieve a maintenance dose (defined in Study Design and Plan) •Hgb level •Duration of Hgb change of =1.0 g/dL from pre-treatment baseline without transfusion •Duration of Hgb change of =1.5 g/dL from pre-treatment baseline without transfusion •Change from pre-treatment baseline in the following PD parameters: serum iron, ferritin, transferrin saturation (TSAT). TD: •Proportion of subjects who achieve = 33% reduction in the RBC units required over an 8-week period •Proportion of subjects who achieve a maintenance dose (defined in Study Design and Plan) •Duration of response (defined as = 20% reduction in the RBC units required over an 8-week period) •Number of RBC units required •Percent change from pre-treatment baseline in the RBC units required. •Hgb level •Hgb change from pre-treatment baseline •Hgbchange from pre-treatment baseline •Change from baseline in the following PD parameters: serum iron, ferritin, TSAT ;Timepoint(s) of evaluation of this end point: NTD subjects: The first two endpoints are evaluated every 4 weeks until the subject meets the endpoint or reaches the maximum dose of 80 mg weekly. The other endpoints are evaluated at Month 24. TD subjects: The first two endpoints are evaluated every 8 weeks until the subject meets the endpoint or reaches the maximum dose of 80 mg weekly. The other endpoints are evaluated at Month 24. | — |
Countries
Greece, Italy, Lebanon, Malaysia, Thailand, Tunisia, Turkey, United Kingdom, United States
Contacts
Public ContactClinical-Regulatory Info Group
Protagonist Therapeutics, Inc.
Outcome results
None listed