Apotransferrin in Atransferrinemia

Congenital Atransferrinemia Clinical Trial

Official title:

Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01797055
• Other study ID #: MD2009.04
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: December 2010
• Est. completion date: January 2027

Study information

• Verified date: April 2024
• Source: Prothya Biosolutions
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to investigate the pharmacokinetics, efficacy and safety of Apotransferrin replacement therapy in atransferrinemia patients.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 5
• Est. completion date: January 2027
• Est. primary completion date: March 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl

• Informed consent

Exclusion Criteria:

• Known with allergic reactions against human plasma or plasma products

• Having detectable anti-immunoglobulin A antibodies

Related Conditions & MeSH terms

• Congenital Atransferrinemia

Intervention

Drug:
• Human apotransferrin
intravenous infusion

Locations

Country	Name	City	State
Germany	Klinikum Aschaffenburg	Aschaffenburg
Italy	AO San Gerardo	Monza
Spain	Vall d'Hebron Hospital	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Prothya Biosolutions

Countries where clinical trial is conducted

Germany,  Italy,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	transferrin levels	Effective transferrin levels	10 years
Other	adverse drug reactions	number of adverse drug reactions	10 years
Primary	Hemoglobulin	Hemoglobulin within normal range	10 year
Primary	pharmacokinetics of transferrin	serum transferrin levels measured on several time points before and after infusion	first infusion and year 3
Primary	iron overload in organs	Elimination of iron overload (liver, heart)	10 years
Secondary	Iron overload	Serum Ferritin within normal values	10 years
Secondary	Hematocrit	Hematocrit within normal values	10 years
Secondary	Erythrocytes	Erythrocytes within normal values	10 years