NCT04054921

Brief Summary

In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started May 2019

Shorter than P25 for phase_2

Geographic Reach
7 countries

15 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 10, 2019

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

August 8, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 13, 2019

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2020

Completed
Last Updated

July 16, 2021

Status Verified

February 1, 2021

Enrollment Period

1.2 years

First QC Date

August 8, 2019

Last Update Submit

July 12, 2021

Conditions

β-thalassemiaIneffective Erythropoiesis

Keywords

Chronic anemiaβ-thalassemia

Outcome Measures

Primary Outcomes (1)

  • Proportion of subjects with side effects and severity of side effects will be tabulated

    the long-term safety and tolerability of PTG-300 in Beta Thalassemia.

    Over two year Period after receiving PTG-300

Study Arms (1)

Interventions

EXPERIMENTAL

PTG-300

Drug: PTG-300

Interventions

PTG-300DRUG

PTG-300 is a hepcidin Mimetic

Interventions

Eligibility Criteria

Age12 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.

You may not qualify if:

  • Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

Location

Laiko General Hospital of Athens

Athens, 11526, Greece

Location

Athens General Hospital 'G Gennimatas'

Athens, 11527, Greece

Location

University General Hospital of Patras

Pátrai, 26500, Greece

Location

Chronic Care Center

Hazmiyeh, Lebanon

Location

Hospital Ampang

Ampang, 68000, Malaysia

Location

Hospital Umum Sarawak

Kuching, 93586, Malaysia

Location

Siriraj Hospital Mahidol University

Bangkok, 10700, Thailand

Location

Khon Kaen University

Khon Kaen, 40002, Thailand

Location

Chulalongkorn University

Pathum Wan, 10330, Thailand

Location

Naresuan University

Phitsanulok, 65000, Thailand

Location

University Hospital Farhat Hached

Sousse, 4000, Tunisia

Location

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Istanbul, 34093, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi

Izmir, 35100, Turkey (Türkiye)

Location

Barts Health NHS Trust

London, E1 1BB, United Kingdom

Location

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 8, 2019

First Posted

August 13, 2019

Study Start

May 10, 2019

Primary Completion

July 31, 2020

Study Completion

July 31, 2020

Last Updated

July 16, 2021

Record last verified: 2021-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)LE(1)US(2)TU(2)GR(3)MY(2)TH(4)