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

Phase 2

Completed

• Conditions: Ineffective Erythropoiesis; β-thalassemia; Chronic Anemia
• Interventions: Drug: PTG-300

• Registration Number: NCT03802201
• Lead Sponsor: Protagonist Therapeutics, Inc.

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 Timeline

• Study Start: 2018-12-19
• Study First Submitted: 2019-01-09
• Study First Submitted QC: 2019-01-10
• Study First Posted: 2019-01-14
• Primary Completion: 2020-07-31
• Study Completion: 2020-07-31
• Status Verified: 2021-07
• Disposition First Submitted: 2021-07-09
• Disposition First Submitted QC: 2021-07-13
• Last Update Submitted: 2021-07-13
• Disposition First Posted: 2021-07-16
• Last Update Posted: 2021-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 63

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

Read More

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
PTG-300 Active	PTG-300	Drug: PTG-300 Subcutaneous

Primary Outcome Measures

Name	Time	Method
NTD: Proportion of responders at each dose	4 week period	NTD subjects who achieve an increase in Hgb without transfusion
TD: Proportion of clinical responders at each dose	8 week period	TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period

Trial Locations

Locations (32)

UCSF Benioff Children's Hospital; 🇺🇸 Oakland, California, United States

Hospital of The University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University General Hospital of Patras; 🇬🇷 Patras, Achaia, Greece

Athens General Hospital 'G Gennimatas'; 🇬🇷 Athens, Attiki, Greece

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico; 🇮🇹 Milano, Italy

Hospital Sultanah Aminah; 🇲🇾 Johor Bahru, Malaysia

Hospital Umum Sarawak; 🇲🇾 Kuching, Malaysia

Chulalongkorn University; 🇹🇭 Bangkok, Thailand

Maharaj Nakorn Chiang Mai Chiang Mai University; 🇹🇭 Chiang Mai, Thailand

Khon Kaen University; 🇹🇭 Khon Kaen, Thailand

Naresuan University; 🇹🇭 Phitsanulok, Thailand

Principal Military Hospital; 🇹🇳 Tunis, Tunisia

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi; 🇹🇷 Istanbul, Turkey

Mersin University Medical Facult; 🇹🇷 Mersin, Turkey

Barts Health NHS Trust; 🇬🇧 London, United Kingdom

University Hospital Farhat Hached; 🇹🇳 Sousse, Tunisia

Bone Marrow Transplant Center; 🇹🇳 Tunis, Tunisia

Hippokration Hospital; 🇬🇷 Thessaloníki, Greece

Chronic Care Center; 🇱🇧 Beirut, Lebanon

Hospital Pulau Pinang; 🇲🇾 George Town, Malaysia

Queen Elizabeth Hospital; 🇲🇾 Kota Kinabalu, Malaysia

Acibadem Adana Hospital; 🇹🇷 Adana, Turkey

Hacettepe University Medical Faculty; 🇹🇷 Ankara, Turkey

Aziza Othmana Hospital; 🇹🇳 Tunis, Tunisia

Ege Universitesi Tip Fakultesi; 🇹🇷 Izmir, Turkey

Siriraj Hospital Mahidol University; 🇹🇭 Bangkok, Thailand

Akdeniz Universitesi Hastanesi; 🇹🇷 Antalya, Turkey

Presidio Ospedaliero Antonio Perrino; 🇮🇹 Brindisi, Italy

AOU dell'Università degli Studi della Campania Luigi Vanvitelli; 🇮🇹 Napoli, Italy

Laiko General Hospital of Athens; 🇬🇷 Athens, Attiki, Greece

Hospital Ampang; 🇲🇾 Ampang, Malaysia

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States