Clinical trial to investigate the treatment with apotransferrin of patients with atransferrinemia

Phase 1

Conditions: Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Atransferrinemia
MedDRA version: 20.0Level: LLTClassification code 10055212Term: Hypochromic microcytic anemiaSystem Organ Class: 100000004851
MedDRA version: 21.0Level: PTClassification code 10005620Term: Blood iron increasedSystem Organ Class: 10022891 - Investigations
MedDRA version: 20.0Level: LLTClassification code 10002272Term: AnemiaSystem Organ Class: 100000004851
MedDRA version: 21.0Level: PTClassification code 10065973Term: Iron overloadSystem Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 20.0Level: PTClassification code 10044356Term: Transferrin decreasedSystem Organ Class: 10022891 - Investigations

Registration Number: EUCTR2009-017409-13-DE

Lead Sponsor: Prothya Biosolutions Netherlands B.V.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 6

Inclusion Criteria

1. Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl
2. Informed consent

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Known with allergic reactions against human plasma or plasma products
2. Having detectable anti-IgA antibodies

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate the pharmacokinetics, efficacy and safety of apotransferrin replacement therapy;Secondary Objective: To determine the optimal dose regimen;Primary end point(s): The primary efficacy parameters are the concentration of transferrin, ferritin, Hb and iron in derum as well as the level of transferrin saturation and total binding capacity. PK parameters.;Timepoint(s) of evaluation of this end point: 1. after first phase of the study (108 weeks); 2. after 3 years follow-up; 3. after end of extended follow-up

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Safety and clinical tolerance will be monitored by adverse event surveillance and laboratory measurements.;Timepoint(s) of evaluation of this end point: Monitoring on ongoing basis