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Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (TRANSCEND)

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ClinicalTrials.gov Identifier: NCT03802201
Recruitment Status : Completed
First Posted : January 14, 2019
Last Update Posted : August 17, 2020
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE January 9, 2019
First Posted Date  ICMJE January 14, 2019
Last Update Posted Date August 17, 2020
Actual Study Start Date  ICMJE December 19, 2018
Actual Primary Completion Date July 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 8, 2019)
  • NTD: Proportion of responders at each dose [ Time Frame: 4 week period ]
    NTD subjects who achieve an increase in Hgb without transfusion
  • TD: Proportion of clinical responders at each dose [ Time Frame: 8 week period ]
    TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
Original Primary Outcome Measures  ICMJE
 (submitted: January 10, 2019)
  • NTD: Proportion of responders at each dose [ Time Frame: 4 week period ]
    NTD subjects who achieve an increase in Hgb without transfusion
  • NTD: Mean Hgb change from baseline [ Time Frame: 4 week period ]
  • TD: Proportion of clinical responders at each dose [ Time Frame: 8 week period ]
    TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
  • TD: Mean change from baseline in the number of units of RBC required under each dose [ Time Frame: 8 week period ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
Official Title  ICMJE A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia
Brief Summary This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.
Detailed Description This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort and with the potential for individual titration (dose increase or decrease) within each cohort. The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment of chronic anemia in β-thalassemia and to evaluate the appropriate dosing regimen for PTG-300 in the target population
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Intervention Model Description:

Multiple PTG-300 dose levels/regimens are planned to be tested for each subpopulation of β thalassemia (NTD and TD) on separate arms:

  • Cohort 1: 3 mg subcutaneous (SC) weekly
  • Cohort 2: 10 mg SC weekly
  • Cohort 3: 20 mg SC weekly
  • Cohort 4a: 40 mg SC weekly
  • Cohort 4b: 40 mg SC every 2 weeks
  • Cohort 7: 80 mg SC weekly
  • Cohort 8: 40 mg SC twice a week

Additional Cohorts (Cohorts 5,6,9,10, and 11) will include subjects between 12 years of age and less than 18 years of age.

Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • β-thalassemia
  • Ineffective Erythropoiesis
Intervention  ICMJE Drug: PTG-300
Subcutaneous (SC)
Other Name: PTG-300 Active
Study Arms  ICMJE Experimental: PTG-300
PTG-300 Active
Intervention: Drug: PTG-300
Publications * Not Provided

*   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: July 29, 2020)
63
Original Estimated Enrollment  ICMJE
 (submitted: January 10, 2019)
84
Actual Study Completion Date  ICMJE July 31, 2020
Actual Primary Completion Date July 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  1. Male and female subjects aged 18 to 65 years, inclusive (Cohorts 1-4b).
  2. Male and female subjects aged 12-<18 years, with a minimum weight of 30 kg (Cohorts 5 and 6).
  3. Documented diagnosis of β-thalassemia with no other Hgb abnormality.

Inclusion criteria applicable only for NTD β-thalassemia subjects:

  1. Mean Hgb < 10.0 g/dL of two measurements (one performed 7-28 days prior to dosing and the other performed within 7 days prior to dosing).
  2. Requirement of < 6 units RBC transfusion in a 24 week period with the last transfusion at least 8 weeks before screening.

Inclusion criteria applicable only for TD β-thalassemia subjects:

  1. Transfusion requirement of at least 6 units of RBC in the 24 weeks prior to screening with no transfusion free period > 45 days.
  2. Last RBC transfusion 5-10 days prior to dosing.

Main Exclusion Criteria:

  1. Subjects with Sickle Cell disease, Hgb H, Hb Bart's hydrops foetalis or hemoglobin S
  2. Infection requiring hospitalization or IV antimicrobial therapy, or opportunistic infection within 6 months of dosing, any infection requiring antimicrobial therapy within 2 weeks of dosing; history of infection with human immunodeficiency virus (HIV).
  3. 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.
  4. Known primary or secondary immunodeficiency.
  5. History within 6 months of screening of any of the following:

    myocardial infarction, unstable angina, transient ischemic attack, decompensated heart failure requiring hospitalization, congestive heart failure (New York Heart Association Class 3 or 4), uncontrolled arrhythmias, cardiac revascularization, stroke, uncontrolled hypertension (resting systolic blood pressure [BP] > 160mmHg or resting diastolic BP > 100mmHg on more than one occasion) or uncontrolled diabetes (Hgb A1c > 9% or > one episode of severe hypoglycemia).

  6. Pregnant or lactating females.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 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 Greece,   Italy,   Lebanon,   Malaysia,   Thailand,   Tunisia,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03802201
Other Study ID Numbers  ICMJE PTG-300-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Protagonist Therapeutics, Inc.
Study Sponsor  ICMJE Protagonist Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Protagonist Therapeutics, Inc.
Verification Date July 2020

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