Apotransferrin in Patients With β-thalassemia | NCT03993613

Studies
Apotransferrin in Patients With β-thalassemia

NCT03993613

Sponsor

Prothya Biosolutions

Status

Terminated

Last Update Posted

Aug 15, 2022Actual

Enrollment

10Actual

Phase

Phase 2

Conditions

β-thalassemia Intermedia

Interventions

human apotransferrin

Countries

Netherlands

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2022

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

no availability of IMP for this study

Expanded Access Info

Start Date

Mar 21, 2019Actual

Primary Completion Date

Mar 31, 2022Actual

Completion Date

Mar 31, 2022Actual

First Submitted Date

May 29, 2019

First Submission Date that Met QC Criteria

Jun 19, 2019

First Posted Date

Jun 20, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 11, 2022

Last Update Posted Date

Aug 15, 2022Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

10Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
human apotransferrin	Experimental	Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.	Biological: human apotransferrin

Interventions

Name	Type	Description	Arm Group Labels	Other Names
human apotransferrin	Biological	Intravenous infusions	human apotransferrin

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Erythropoiesis	Change of haemoglobin level and/or or change of number of RBC units transfused/week	17 weeks

Secondary Outcomes

Measure	Description	Time Frame
Change from baseline in serum iron		17 weeks
Change from baseline in change plasma levels of advanced glycation end products		17 weeks
Change in spleen size

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anaemia in combination with an elevated HbA2 (>2.5%) and a haemoglobin of <6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule.
Age above≥ 17 years.
Adequate renal and hepatic function tests
WHO performance 0, 1 or 2.
Signed informed consent.

Exclusion Criteria:

Known with allergic reactions against human plasma or plasma products.
Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease).
Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias.
Pregnant or lactating females.
Known with IgA deficiency with anti-IgA antibodies

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Bart Biemond, MD, PhD	Academic Medical Centre	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Academic Medical Centre		Amsterdam-Zuidoost	North Holland	1100 DD	Netherlands