Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
75
Countries
1
Sites
9
Premature patients with bronchopulmonary dysplasia
To evaluate the safety and feasibility of repeated intravenous infusions of UC-MSC 5*106 cells/kg in preterm patients ≤ 28 weeks gestational age and ≤ 1250g weight.
Key facts
- Sponsor
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario Ramon Y Cajal
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Respiratory Tract Diseases [C08]
- Trial duration
- 11 Jan 2024 → ongoing
- Decision date (initial)
- 2024-09-26
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Instituto de Salud Carlos III (ICI19/00092).
External identifiers
- EU CT number
- 2024-516677-79-00
- EudraCT number
- 2022-002653-26
- ClinicalTrials.gov
- NCT06270199
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
To evaluate the safety and feasibility of repeated intravenous infusions of UC-MSC 5*106 cells/kg in preterm patients ≤ 28 weeks gestational age and ≤ 1250g weight.
Secondary objectives 1
- To observe the incidence of severe BPD or death at 36 weeks PD. To observe the incidence of death at 36 and 40 weeks PD and at discharge. To observe the incidence of complications of prematurity. To investigate changes in markers of inflammation, oxidative stress and lung damage. To analyze echocardiographic variations in pulmonary hypertension before and after. Variation of modified respiratory status score and RSS score during therapy and up to 36 weeks EP. Observe the need for supplemental O2 at home discharge and at 18 months and the duration of invasive and noninvasive mechanical ventilation. To evaluate the use of postnatal corticosteroids as treatment/prevention of BPD. To analyze the rate of readmissions in the first year due to respiratory causes. To evaluate the Bayley developmental scale of infants and young children at 24 months of age. Safety, feasibility and explore efficacy.
Conditions and MedDRA coding
Premature patients with bronchopulmonary dysplasia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10006475 | Bronchopulmonary dysplasia | 100000004855 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Recruitment period Randomization to 3 infusions/6 infusions/control will be 1:2:2 and will be done through a computerized platform by competitive randomization until the n is reached:
- Control (n=15)
- Treatment with 3 infusions of 5 MSC 5x106 /kg body weight (n=30)
- Treatment with 6 infusions of 5 MSCs 5x106 /kg body weight (n=30)
|
Randomised Controlled | None | control group: Standard of care experimental group: - Treatment with 3 infusions of 5 MSC 5x106 /kg body weight or ; - Treatment with 6 infusions of 5 MSCs 5x106 /kg body weight |
|
| 2 | Treatment period Randomization to 3 infusions/6 infusions/control will be 1:2:2 and will be done through a computerized platform by competitive randomization until the n is reached:
- Control (n=15)
- Treatment with 3 infusions of 5 MSC 5x106 /kg body weight (n=30)
- Treatment with 6 infusions of 5 MSCs 5x106 /kg body weight (n=30)
For all arms is to discharge.
|
Randomised Controlled | None | experimental group: - Treatment with 3 infusions of 5 MSC 5x106 /kg body weight or - Treatment with 6 infusions of 5 MSCs 5x106 /kg body weight control group: standard of care |
|
| 3 | Follow-up From visit 11 ( 3 months of corrected age) to visit 16 ( 24 months of corrected age)
|
Not Applicable | None | experimental group: - Treatment with 3 infusions of 5 MSC 5x106 /kg body weight (n=30) - Treatment with 6 infusions of 5 MSCs 5x106 /kg body weight (n=30) control group: standard of care |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- Live newborns weighing ≤ 1250 grams and GA ≤ 28 weekswho are on invasive or noninvasive ventilatory support with an FiO2 ≥ 0.3 between days 5 and 14 of life,
Exclusion criteria 1
- - who have other concomitant congenital pathology at the time of inclusion: pulmonary malformations with compromised pulmonary function, active pulmonary hemorrhage, severe pulmonary hypoplasia, renal malformations with systemic compromise, congenital heart disease, polymalformative syndromes, chromosomopathies. - who present refractory hemodynamic instability of any cause at the time of inclusion. - who present severe neurological injury at the time of inclusion (HIV grade III or higher). - Who have required major surgery in the 72 hours prior to inclusion. - Who present necrotizing enterocolitis (NEC) grades ≥II at the time of inclusion, according to the Bell classification. - who are children of a mother with HIV.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Safety assessment: adverse reactions (AR)
Secondary endpoints 1
- Status at week 36 EPM (death/DBP grade 3). Diagnosis and stage of BPD at 36 weeks EPM according to Jensen´s classification (no BPD, grade 1, grade 2 or grade 3). Exitus at 36 and 40 weeks of EPM or at discharge (yes/no). Incidence of comorbidities derived from prematurity. Variations in the levels of biomarkers of inflammation, oxidative stress and lung damage with respect to baseline. Variations in the modified respiratory score and RSS score during therapy and up to week 36 EPM.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Expanded Allogeneic Fetal Umbilical Cord Stem Mesenchymal Cells
PRD11555433 · Product
- Active substance
- Mesenchymal Cells
- Pharmaceutical form
- SUSPENSION FOR IV INFUSION
- Route of administration
- INTRAVENOUS ADMINISTRATION
- Max daily dose
- 5 millilitre(s)/kilogram
- Max total dose
- 5 millilitre(s)/kilogram
- Max treatment duration
- 6 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO RAMON Y CAJAL
- Paediatric formulation
- Yes
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fundacion Para La Investigacion Biomedica Del Hospital Universitario Ramon Y Cajal
- Sponsor organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario Ramon Y Cajal
- Address
- Carretera Del Colmenar Viejo Km 9100, Por El Pardo Por El Pardo
- City
- Madrid
- Postcode
- 28034
- Country
- Spain
Scientific contact point
- Organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario Ramon Y Cajal
- Contact name
- Dr. María Jesús del Cerro
Public contact point
- Organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario Ramon Y Cajal
- Contact name
- Dr. María Jesús del Cerro
Locations
1 EU/EEA country · 9 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruiting | 75 | 9 |
| Rest of world | — | 0 | — |
Investigational sites
University Hospital Virgen Del Rocio S.L.
Neonatology Department, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Universitario La Paz
Neonatology Department, Paseo De La Castellana 261, 28046, Madrid
Hospital Universitario Regional De Malaga
Neonatology Department, Avenida De Carlos De Haya S/N, 29010, Malaga
Hospital Universitario Y Politecnico La Fe
Neonatology Department, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Hospital Universitario Quironsalud Madrid
Neonatology Department, Calle De Diego De Velazquez 1, 28223, Pozuelo De Alarcon
Complexo Hospitalario Universitario A Coruna
Neonatology Department, Lugar Jubias De Arriba 84, 15006, A Coruna
Hospital Clinico San Carlos
Neonatology Department, Calle Del Profesor Martín Lagos S/n, 28040, Madrid
Hospital Universitario Marques De Valdecilla
Neonatology department, Avenida Valdecilla Sn, 39008, Santander
Hospital Universitari Dexeus Grupo Quironsalud
Neonatology department, Calle De Sabino Arana 5-19, 08028, Barcelona
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 |
|---|---|---|---|---|---|
| Spain | 2024-01-11 | 2024-01-11 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 8 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1 Protocol 2024-516677-79-00 V4 28 11 2025 clean | 4 |
| Protocol (for publication) | D1_Protocol_2024-516677-79-00_V3_03 04 24 | 3 |
| Recruitment arrangements (for publication) | Section not applicable | 1 |
| Subject information and informed consent form (for publication) | 7 L1 SIS ICF v3 28 11 2025_clean | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF V2 1_29abril2024 | 2.1 |
| Summary of Product Characteristics (SmPC) (for publication) | Section not applicable | 1 |
| Synopsis of the protocol (for publication) | D1 Protocol synopsis 2024-516677-79-00 V4 28 11 2025 clean | 4 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2024-516677-79-00_V3_03 04 24 | 3 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-20 | Spain | Acceptable with conditions 2024-09-26
|
2024-09-26 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-12-15 | Spain | Acceptable 2026-02-16
|
2026-02-20 |