Parenteral Retinol Level and Tolerability Trial

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Aspire Pharma Limited

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

13 Feb 2026 → ongoing

Decision date (initial)

2025-04-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Aspire Pharma Limited

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

Objectives - Primary:
• To assess Retinol serum concentration at Days 0 and 28, in preterm infants, with GA between 22 weeks + 0 day and 29 weeks + 6 days.
• To evaluate the safety and tolerability of RetinolX from Day 0 to week 36 Post-menstrual age (PMA).

Secondary objectives 4

1. To assess the relative increase of retinol serum concentration to 60% at Day 28.
2. To analyze Retinol serum concentration at Day 7 and Day 14.
3. Need for respiratory support and/or oxygen at 28 days postnatal age.
4. Need for respiratory support and/or oxygen at 36 weeks + 0 day PMA

Conditions and MedDRA coding

Bronchopulmonary Dysplasia

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

Austrian Agency For Health And Food Safety, Swedish Medical Products Agency

EMA paediatric investigation plan (PIP)

EMEA-002790-PIP01-20

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Very preterm infants with a GA <30 weeks (N=12): • 7 extremely preterm infants ≤27 weeks + 6 days GA and • 5 very preterm infants ≥28 weeks + 0 day GA and ≤29 weeks + 6 days GA to cover data from both sub-populations of preterm infants <30 weeks
2. Enrollment between 24 hours (h) and 72 h of birth. Note: All genders and ethnicities are eligible for the study.
3. Informed consent must be obtained from the parents of the infants prior to enrollment in the study.

Exclusion criteria 5

1. Major congenital anomalies
2. Intraventricular Hemorrhage (IVH) grade 2-4
3. Infants at a high mortality risk, as judged by the hospital principal investigator (PI), for example • Terminal illness as evidenced by severe acidosis (pH < 7.0 for > 2 h) or persistent bradycardia (heart rate < 100 beats per minute) associated with hypoxia for > 2 h • Congenital nonbacterial infection with overt signs at birth
4. Infants who are to receive vitamin A in a parenteral fat emulsion in doses exceeding recommendations for multivitamin preparations.
5. Infants born preterm due to maternal exposure to modifiable risk factors such as smoking, alcohol consumption, substance abuse, or other known teratogenic exposures.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Primary Efficacy: Relative increase in serum retinol levels at Day 28 compared to baseline
2. Safety: Incidence of Treatment Emergent Adverse Events (TEAEs) and Adverse Event of Specific Interest (AESI) throughout the study.
3. Safety: Occurrence of clinically significant changes in laboratory data, physical examination results, and vital signs values throughout the study.

Secondary endpoints 4

1. Secondary efficacy: Percentage of subjects with relative increase of 60% in serum retinol levels between Day 0 and Day 28.
2. Secondary efficacy: Relative increase in serum retinol levels at Day 7 and Day 14 compared to baseline.
3. Secondary efficacy: Percentage of subjects with need for respiratory support and/or oxygen at 28 days postnatal age.
4. Secondary efficacy: Percentage of subjects with need for respiratory support and/or oxygen at 36 weeks + 0 day PMA.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Aspire Pharma Limited

Sponsor organisation

Aspire Pharma Limited

Address

4 Rotherbrook Court, Bedford Road Bedford Road

City

Petersfield

Postcode

GU32 3QG

Country

United Kingdom

Scientific contact point

Organisation

Aspire Pharma Limited

Contact name

Senior Regulatory Clinical Manager

Public contact point

Organisation

Aspire Pharma Limited

Contact name

Senior Regulatory Clinical Manager

Third parties 5

Locations

2 EU/EEA countries · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 2 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 42 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications