Clinical trial to evaluate effectiveness and cost-effectiveness of pre-emptive genotyping strategy to optimise tacrolimus dosage in a pretransplant chronic kidney disease patients

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Not possible to specify

Trial duration

31 Mar 2025 → ongoing

Decision date (initial)

2025-01-17

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacogenetic

To assess the effectiveness of a pre-emptive pharmacogenetic strategy in reaching tacrolimus target plasma concentrations.

Secondary objectives 3

1. A pharmacoeconomic analysis to assess the feasibility of implementing a preemptive pharmacogenetic strategy in reducing renal transplant rejection and the incidence and severity of adverse events (AE).
2. To evaluate the safety and tolerability of tacrolimus treatment derived from a preemptive pharmacogenetic strategy.
3. To identify new potential predictors of treatment-related AE, such as genetic or clinical factors.

Conditions and MedDRA coding

Kidney transplant

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Participants must be willing and able to provide written informed consent prior the initiation of any study procedures.
2. Subject or their legally authorized representative has voluntarily signed the informed consent document.
3. Participant is on the waiting list for a kidney transplant.
4. Subject is able and willing to take part and be followed-up for the majority of the study duration, and adhere to the procedures specified in this protocol.
5. Subjects must be naïve to any genotyping test of the following genes: CYP3A5.

Exclusion criteria 5

1. Known hypersensitivity/allergy reaction to tacrolimus or any of the excipients.
2. History of renal, heart, and/or liver transplant.
3. History or clinical evidence of any disease and/or existence of any surgical or medical condition, which might interfere in a relevant manner with the absorption, distribution, metabolism, or excretion of the study treatment, except for renal disease.
4. Any condition or situation precluding or interfering the compliance with the protocol.
5. Any condition at medical discretion for which renal transplantation and/or study treatment should not be received.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Tacrolimus concentrations levels at day 4 (+/-1d) will be the effectiveness surrogate outcome. It will be considered therapeutic range levels between 7-10 ng/ml.

Secondary endpoints 8

1. Ratio that divides the differences in costs between both treatments by the difference in effectiveness between both treatments.
2. Number and percentage of patients achieving tacrolimus target plasma concentrations.
3. Number and percentage of patients with transplant rejection. Transplant rejection will be considered if there is histological confirmation and/or the patient initiates any type of therapy aimed at treating rejection (e.g. corticosteroids).
4. Rate of adverse events associated to treatment.
5. Healthcare expenditure related to predefined events of interest: Any costs made as a result of an adverse event.
6. Incidence of discontinuation or treatment modification due to lack of effective related to the drug of inclusion.
7. Identification of new actionable genes/relevant polymorphisms within the predefined population subsets.
8. Novel prognostic and predictive genetic biomarkers of tacrolimus safety and effectiveness will be assessed trough techniques only available at Nation Centre of Oncological Investigations (CNIO) and genome-wide association studies when applicable.

Investigational products

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

Test 7

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz

Sponsor organisation

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz

Address

Paseo De La Castellana 261

City

Madrid

Postcode

28046

Country

Spain

Scientific contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz

Contact name

Borobia Perez, Alberto Manuel

Public contact point

Organisation

Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz

Contact name

Borobia Perez, Alberto Manuel

Locations

1 EU/EEA country · 6 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

Documents 15 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