A Phase I, Single-Centre, Double-Blind, Randomized, Placebo-Controlled Clinical Trial To Determine Urinary Xylose Cut-Off Point For The Diagnosis Of Hypolactasia, After A Single Dose Of Gaxilose in Children Between 5 – 11 Years Of Age.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Venter Pharma S.L.

Participant type

Pediatric, Healthy volunteers

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

22 Jun 2023 → 30 Jun 2023

Decision date (initial)

2023-06-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic

To establish a urinary cut-off point of xylose after a single dose of Gaxilose (LacTEST 0,45g) in healthy subjects between 5 - 11 years of age, for the diagnosis of hypolactasia.

Secondary objectives 1

1. To assess the safety and tolerability of a single oral dose of 0.45g of Gaxilose in subjects between 5 - 11 years of age.

Conditions and MedDRA coding

Hypolactasia

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Either male of female children between 5 or 11 years of age.
2. Participant’s parent(s)/legal guardian(s) are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
3. Subjects in good health with no baseline gastrointestinal condition.
4. Participants that regularly consume lactose containing products and have no signs or symptoms that could be associated with intestinal lactase deficiency (Participants will be inquire about this aspect at screening visit).

Exclusion criteria 14

1. Participant’s parent(s)/legal guardian(s) are not willing or able to comply with either: all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, or other study procedures.
2. Participant has a known or suspected allergy or history of anaphylaxis or other serious adverse reactions to Gaxilose (LacTEST 0,45g) excipients.
3. Participant has a known or suspected allergy or history of anaphylaxis or other serious adverse reactions to any of the ingredients of the on-site meals.
4. Participant has a known family history of the C/T-13910 and G/A-22018 polymorphisms associated with intestinal lactase deficiency.
5. Participant has a known medical history of decreased renal function of any cause (as defined by an estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m2).
6. Participant has a known medical history of either clinical or systemic manifestation of portal hypertension (i.e., ascites, cirrhosis…).
7. Participant has a known medical history of total gastrectomy and/or vagotomy.
8. Participant has a known medical history of myxoedema.
9. Participant has a known medical history of pentosuria and/or galactosaemia.
10. Participant has a known medical history of diabetes mellitus.
11. Participant is currently enrolled or has enrolled in a clinical trial three months prior to inclusion in the current study.
12. Participant has a known history of substance abuse.
13. Participant has taken either acetylsalicylic acid or indomethacin in the 48 hours prior to study enrollment.
14. Any condition or situation precluding or interfering the compliance with the protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. PK analysis for urinary xylose excretion will be based on the following parameters: maximum urinary excretion rate detected at 0-3, 3-4 and 4-5h; its mean time during the corresponding interval (Tmax) and total xylose excreted (Ae0-5). All urine samples will be analyzed at a laboratory located in Hospital Universitario de Getafe

Secondary endpoints 1

1. The incidence of adverse events (AEs), as well as relevant and abnormal changes from baseline in laboratory test results, and changes to vital signs in the treatment arm compared to the control arm.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Venter Pharma S.L.

Sponsor organisation

Venter Pharma S.L.

Address

Calle Almagro 1 1 Dcha

City

Madrid

Postcode

28010

Country

Spain

Scientific contact point

Organisation

Venter Pharma S.L.

Contact name

Carmen Hermida

Public contact point

Organisation

Venter Pharma S.L.

Contact name

Carmen Hermida

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 2 files

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

Application history

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