Overview
Sponsor-declared trial summary
Severe chronic neutropenia (SCN)
Evaluation of the pegG-CSF efficacy in maintaining absolute neutrophil values above 1000/mmc in patients affected with severe chronic neutropenia (SCN) already being treated with G-CSF (once daily) and anti-infective therapy.
Key facts
- Sponsor
- IRCCS Istituto Giannina Gaslini
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15], Diseases [C] - Immune System Diseases [C20]
- Decision date (initial)
- 2025-01-31
- Transition trial
- Yes
- Low-intervention
- Yes
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-519009-36-01
- EudraCT number
- 2005-003096-20
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy
Evaluation of the pegG-CSF efficacy in maintaining absolute neutrophil values above 1000/mmc in patients affected with severe chronic neutropenia (SCN) already being treated with G-CSF (once daily) and anti-infective therapy.
Secondary objectives 8
- Drug compliance
- Dosage per kg of body weight and frequency of the study drug
- Relationship between the frequency of administration and the neutrophil count values
- Incidence and duration of infection in patient treated with peg G-CSF versus patient treated as per standard of care
- pegG-CSF pharmacokinetics
- Liver and kidney tolerability
- Side effects
- Direct and indirect costing
Conditions and MedDRA coding
Severe chronic neutropenia (SCN)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10029354 | Neutropenia | 100000004851 |
| 20.0 | SOC | 10005329 | Blood and lymphatic system disorders | 3 |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-519009-36-00 | Use of pegfilgrastim in severe chronic neutropenia | IRCCS Istituto Giannina Gaslini |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- Patients, none age limits, affected with either congenital or acquired chronic neutropenia defined as absolute neutrophil count < 500/mmc (based on three consecutive tests at least one week apart within a minimum three-months period) already being treated with G-CSF (daily).
Exclusion criteria 7
- Subjects affected with autoimmune neutropenias or idiopathic neutropenia that are not being treated/occasionally treated with growth factor
- Subjects affected with iatrogenic neutropenia anticancer drug-induced
- Subject affected with neutropenia induced by myelodysplastic syndrome and or leukemia with/without clonal cytogenetic abnormalities
- Subject affected with acquired or congenital neutropenia diagnosis
- Subjects affected with hepatic or renal impairment
- Subjects affected with homozygous sickle cell disease or subjects affected with double heterozygosity inheritance of HbS and β-thalassemia
- Pregnancy and nursing woman
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Median of absolute neutrophil count (from second to six months-period of study)
Secondary endpoints 3
- Number, type and duration of the infections (from second to six months-period of study)
- Number of antibiotic courses (from second to six months-period of study)
- Numbero of hospitalizations (from second to six months-period of study)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Neulasta 6 mg solution for injection
PRD379066 · Product
- Active substance
- Pegfilgrastim
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 6 mg/kg milligram(s)/kilogram
- Max total dose
- 324 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- L03AA13 — PEGFILGRASTIM
- Marketing authorisation
- EU/1/02/227/004
- MA holder
- AMGEN EUROPE B.V.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- The use of the investigational medicinal products is evidence-based.
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
IRCCS Istituto Giannina Gaslini
- Sponsor organisation
- IRCCS Istituto Giannina Gaslini
- Address
- Via Gerolamo Gaslini 5
- City
- Genoa
- Postcode
- 16147
- Country
- Italy
Scientific contact point
- Organisation
- IRCCS Istituto Giannina Gaslini
- Contact name
- Francesca Fioredda
Public contact point
- Organisation
- IRCCS Istituto Giannina Gaslini
- Contact name
- Francesca Fioredda
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Authorised, recruitment pending | 20 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 7 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-519009-36-00 | 1 |
| Protocol (for publication) | D1_Protocol 2024-519009-36-00_ALLEGATO 1_ITA | 1 |
| Recruitment arrangements (for publication) | R1_Placeholder | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF child | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents-guardian | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_placeholder | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-01-29 | Italy | Acceptable 2025-01-30
|
2025-01-31 |