Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruitment ended
Participants planned
5
Countries
3
Sites
3
Congenital atransferrinaemia/ hypotransferrinaemia
To investigate the pharmacokinetics, efficacy and safety of apotransferrin replacement therapy
Key facts
- Sponsor
- Prothya Biosolutions Netherlands B.V.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 12 Nov 2010 → ongoing
- Decision date (initial)
- 2024-08-05
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-515986-33-00
- EudraCT number
- 2009-017409-13
- ClinicalTrials.gov
- NCT01797055
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Dose response, Safety, Efficacy
To investigate the pharmacokinetics, efficacy and safety of apotransferrin replacement therapy
Secondary objectives 1
- To determine the optimal dose regimen
Conditions and MedDRA coding
Congenital atransferrinaemia/ hypotransferrinaemia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10044356 | Transferrin decreased | 100000004848 |
| 23.1 | PT | 10084860 | Hypotransferrinaemia | 100000004851 |
| 21.0 | PT | 10065973 | Iron overload | 100000004861 |
| 20.0 | LLT | 10055212 | Hypochromic microcytic anemia | 10005329 |
| 23.0 | LLT | 10083978 | Congenital atransferrinaemia | 10010331 |
| 21.0 | PT | 10005620 | Blood iron increased | 100000004848 |
Study design 2 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | First phase First 108 weeks
|
2 | None | First phase: Escalating dose every 4-8 weeks | |
| 2 | Long-term Follow-up Long-term follow-up >10 years
|
2 | None | Long-term Follow-up phase: Treatment every 4-8 weeks. Dose dependent on the clinical outcome. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dL
- Signed informed consent
Exclusion criteria 3
- Known with allergic reactions against human plasma or plasma products
- Having detectable anti-IgA antibodies
- Fertile women unless they take valid contraceptive measures (ITA only)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Increase of hemoglobin (to normal range)
- Pharmacokinetics of transferrin
- Decrease of serum ferritin (to normal range)
Secondary endpoints 5
- Increase of hematocrit (to normal range)
- Increase of erythrocytes (to normal range)
- Decrease of iron overload (liver, heart)
- Transferrin levels, transferrin saturation, and serum iron in relation to efficacy
- Undetectable NTBI/LPI levels
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD761491 · Product
- Active substance
- Human Apotransferrin
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 360 mg/kg milligram(s)/kilogram
- Max total dose
- 30 mg milligram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- SANQUIN PLASMA PRODUCTS
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/12/1027
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Prothya Biosolutions Netherlands B.V.
- Sponsor organisation
- Prothya Biosolutions Netherlands B.V.
- Address
- Plesmanlaan 125
- City
- Amsterdam
- Postcode
- 1066 CX
- Country
- Netherlands
Scientific contact point
- Organisation
- Prothya Biosolutions Netherlands B.V.
- Contact name
- Ilona Kleine Budde
Public contact point
- Organisation
- Prothya Biosolutions Netherlands B.V.
- Contact name
- Ilona Kleine Budde
Locations
3 EU/EEA countries · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ongoing, recruitment ended | 2 | 1 |
| Italy | Ongoing, recruitment ended | 1 | 1 |
| Spain | Ongoing, recruiting | 2 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Klinikum Aschaffenburg-Alzenau gGmbH
Klinik für Kinder- und Jugendmedizin, Am Hasenkopf 1, Innenstadt, Aschaffenburg
Fondazione IRCCS San Gerardo Dei Tintori
SSD Malattie Rare, Via Giovanni Battista Pergolesi 33, 20900, Monza
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Germany | 2011-07-12 | 2011-08-29 | 2013-08-29 | ||
| Italy | 2013-06-21 | 2013-07-10 | 2015-07-10 | ||
| Spain | 2010-11-12 | 2010-12-01 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 21 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-515986-33-forpublication | 9 |
| Recruitment arrangements (for publication) | authorized under CTD | 1 |
| Recruitment arrangements (for publication) | authorized under CTD | 1 |
| Recruitment arrangements (for publication) | authorized under CTD | 1 |
| Subject information and informed consent form (for publication) | L1_Datenschutz_parents_extendedFU_DE_pub | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_adults_extendedFU_DE_pub | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_adults_extendedFU_ES_pub | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_adults_extendedFU_IT_pub | 2 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_adults_firstphase_IT_pub | 2 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_adults_FUphase_IT_pub | 1 Italia |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_children7_12y_extendedFU_DE_pub | 2 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_extendedFU_DE_pub | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_extendedFU_ES_pub | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_firstphase_DE_pub | 3 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_firstphase_ES_pub | 2 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_FUphase_DE_pub | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_parents_FUphase_ES_pub | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_DE_2024-515986-33 | 8 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_EN_2024-515986-33 | 8 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_ES_2024-515986-33 | 8 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_IT_2024-515986-33 | 8 |
Application history
12 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-15 | Spain | Acceptable with conditions 2024-08-02
|
2024-08-02 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-12-18 | Spain | Acceptable with conditions 2024-08-02
|
2024-12-18 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-12-20 | Acceptable with conditions 2024-08-02
|
2024-12-20 | |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-01-15 | Acceptable with conditions 2024-08-02
|
2025-01-15 | |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-01-15 | Spain | Acceptable with conditions 2024-08-02
|
2025-01-15 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-5 | 2025-01-15 | Acceptable with conditions 2024-08-02
|
2025-01-15 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-06-16 | Spain | Acceptable 2025-07-28
|
2025-07-29 |
| 8 | NON SUBSTANTIAL MODIFICATION | NSM-6 | 2025-10-02 | Spain | Acceptable 2025-07-28
|
2025-10-02 |
| 9 | NON SUBSTANTIAL MODIFICATION | NSM-7 | 2026-01-12 | Acceptable 2025-07-28
|
2026-01-12 | |
| 10 | NON SUBSTANTIAL MODIFICATION | NSM-8 | 2026-01-20 | Spain | Acceptable 2025-07-28
|
2026-01-20 |
| 11 | NON SUBSTANTIAL MODIFICATION | NSM-9 | 2026-01-20 | Acceptable 2025-07-28
|
2026-01-20 | |
| 12 | SUBSTANTIAL MODIFICATION | SM-2 | 2026-02-27 | Spain | Acceptable 2026-04-10
|
2026-04-13 |