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

ß-thalassemia Clinical Trial

— TRANSCEND  

Official title:

A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03802201
• Other study ID #: PTG-300-02
• Status: Completed
• Phase: Phase 2
• Start date: December 19, 2018
• Est. completion date: July 31, 2020

Study information

• Verified date: July 2021
• Source: Protagonist Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: July 31, 2020
• Est. primary completion date: July 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

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 ß-thalassemia with no other Hgb abnormality.

Inclusion criteria applicable only for NTD ß-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 ß-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.

Related Conditions & MeSH terms

• Anemia
• beta-Thalassemia
• Chronic Anemia
• Ineffective Erythropoiesis
• ß-thalassemia
• Thalassemia

Intervention

Drug:
• PTG-300
Subcutaneous (SC)

Locations

Country	Name	City	State
Greece	Athens General Hospital 'G Gennimatas'	Athens	Attiki
Greece	Laiko General Hospital of Athens	Athens	Attiki
Greece	University General Hospital of Patras	Patras	Achaia
Greece	Hippokration Hospital	Thessaloníki
Italy	Presidio Ospedaliero Antonio Perrino	Brindisi
Italy	Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico	Milano
Italy	AOU dell'Università degli Studi della Campania Luigi Vanvitelli	Napoli
Lebanon	Chronic Care Center	Beirut
Malaysia	Hospital Ampang	Ampang
Malaysia	Hospital Pulau Pinang	George Town
Malaysia	Hospital Sultanah Aminah	Johor Bahru
Malaysia	Queen Elizabeth Hospital	Kota Kinabalu
Malaysia	Hospital Umum Sarawak	Kuching
Thailand	Chulalongkorn University	Bangkok
Thailand	Siriraj Hospital Mahidol University	Bangkok
Thailand	Maharaj Nakorn Chiang Mai Chiang Mai University	Chiang Mai
Thailand	Khon Kaen University	Khon Kaen
Thailand	Naresuan University	Phitsanulok
Tunisia	University Hospital Farhat Hached	Sousse
Tunisia	Aziza Othmana Hospital	Tunis
Tunisia	Bone Marrow Transplant Center	Tunis
Tunisia	Principal Military Hospital	Tunis
Turkey	Acibadem Adana Hospital	Adana
Turkey	Hacettepe University Medical Faculty	Ankara
Turkey	Akdeniz Universitesi Hastanesi	Antalya
Turkey	Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi	Istanbul
Turkey	Ege Universitesi Tip Fakultesi	Izmir
Turkey	Mersin University Medical Facult	Mersin
United Kingdom	Barts Health NHS Trust	London
United Kingdom	Barts Health NHS Trust	London
United States	Boston Children's Hospital	Boston	Massachusetts
United States	UCSF Benioff Children's Hospital	Oakland	California
United States	Hospital of The University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Protagonist Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Greece,  Italy,  Lebanon,  Malaysia,  Thailand,  Tunisia,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	NTD: Proportion of responders at each dose	NTD subjects who achieve an increase in Hgb without transfusion	4 week period
Primary	TD: Proportion of clinical responders at each dose	TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period	8 week period