Clinical trial • Phase II • Rare Disease|Haematology
HUMAN APOTRANSFERRIN for Congenital atransferrinaemia|Hypotransferrinaemia
Phase II trial of HUMAN APOTRANSFERRIN for Congenital atransferrinaemia|Hypotransferrinaemia. open-label, adaptive. 5 participants.
Overview
- Trial Therapeutic Area
- Rare Disease|Haematology
- Trial Disease
- Congenital atransferrinaemia|Hypotransferrinaemia
- Trial Stage
- Phase II
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 15-07-2024
- First CTIS Authorization Date
- 02-08-2024
Trial design
open-label, adaptive Phase II trial across 3 sites in Germany, Spain, Italy.
- Open Label
- Yes
- Adaptive
- Yes
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 5
- Trial Duration For Participant
- 756
Eligibility
Recruits 5 paediatric patients.
- Pregnancy Exclusion
- Fertile women unless they take valid contraceptive measures (ITA only)
- Vulnerable Population
- Children are included as a vulnerable population. Subject information and informed consent forms are available for children (including a child ICF for ages 7-12) and for parents; parental consent/parent information forms are provided. Signed informed consent is required.
Inclusion criteria
- {"criterion_text":"- Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dL"}
- {"criterion_text":"- Signed informed consent"}
Exclusion criteria
- {"criterion_text":"- Known with allergic reactions against human plasma or plasma products"}
- {"criterion_text":"- Having detectable anti-IgA antibodies"}
- {"criterion_text":"- Fertile women unless they take valid contraceptive measures (ITA only)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Increase of hemoglobin (to normal range)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Pharmacokinetics of transferrin","definition_or_measurement_approach":""}
- {"endpoint_text":"- Decrease of serum ferritin (to normal range)","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Increase of hematocrit (to normal range)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Increase of erythrocytes (to normal range)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Decrease of iron overload (liver, heart)","definition_or_measurement_approach":""}
- {"endpoint_text":"- Transferrin levels, transferrin saturation, and serum iron in relation to efficacy","definition_or_measurement_approach":""}
- {"endpoint_text":"- Undetectable NTBI/LPI levels","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 5
- Recruitment Window Months
- 210
- Consent Approach
- Signed informed consent is required. Subject information and informed consent forms exist for adults and children, including a child ICF for ages 7-12 and parental consent/information forms. Documents are available in multiple languages (DE, ES, IT, EN versions present). Parental consent is used for minors; child-specific ICFs are provided.
Geography
- Total Number Of Sites
- 3
- Total Number Of Participants
- 5
Germany
- Earliest CTIS Part Ii Submission Date
- 01-08-2024
- Latest Decision Or Authorization Date
- 12-01-2026
- Processing Time Days
- 529
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Klinikum Aschaffenburg-Alzenau gGmbH
- Department Name
- Klinik für Kinder- und Jugendmedizin
- Principal Investigator Name
- Katja Moser
- Principal Investigator Email
- Katja.Moser@klinikum-ab-alz.de
- Contact Person Name
- Katja Moser
- Contact Person Email
- Katja.Moser@klinikum-ab-alz.de
- Number Of Participants
- 2
Spain
- Earliest CTIS Part Ii Submission Date
- 01-08-2024
- Latest Decision Or Authorization Date
- 20-01-2026
- Processing Time Days
- 537
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Area Materno-Infantil
- Principal Investigator Name
- Cristina Diaz de Heredia Rubio
- Principal Investigator Email
- cristina.diazdeheredia@vallhebron.cat
- Contact Person Name
- Cristina Diaz de Heredia Rubio
- Contact Person Email
- cristina.diazdeheredia@vallhebron.cat
- Number Of Participants
- 2
Italy
- Earliest CTIS Part Ii Submission Date
- 01-08-2024
- Latest Decision Or Authorization Date
- 20-01-2026
- Processing Time Days
- 537
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Fondazione IRCCS San Gerardo Dei Tintori
- Department Name
- SSD Malattie Rare
- Principal Investigator Name
- Raffaella Mariani
- Principal Investigator Email
- r.mariani@asst-monza.it
- Contact Person Name
- Raffaella Mariani
- Contact Person Email
- r.mariani@asst-monza.it
- Number Of Participants
- 1
Sponsor
Primary sponsor
- Full Name
- Prothya Biosolutions Netherlands B.V.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Netherlands
Investigational products
- Investigational Product Name
- Human Apotransferrin (50g/l)
- Active Substance
- HUMAN APOTRANSFERRIN
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous infusion
- Route
- Intravenous infusion
- Orphan Designation
- Yes
- Frequency
- Every 4-8 weeks
- Maximum Dose
- 360 mg/kg (max daily); 30 mg (max total)
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