FORMA – a multicentre randomized-controlled trial to evaluate the efficacy and safety of ketoanalogues of essential amino acids in prophylaxis of protein-energy wasting in nephrotic syndrome.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Not possible to specify

Trial duration

4 Jan 2023 → ongoing

Decision date (initial)

2024-12-02

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Agencja Badań Medycznych

External identifiers

EU CT number

2024-517880-22-00

EudraCT number

2022-000529-26

ClinicalTrials.gov

NCT05716880

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Others, Safety

Evaluation of the efficacy, safety and tolerability of the investigational medicinal product (preparation of α-keto analogues of essential amino acids) in a new clinical indication, i.e. in the prophylaxis of muscle loss in patients with moderate to very severe nephrotic syndrome

Secondary objectives 1

1. Assessment of the impact of the studied intervention on the clinical course of glomerular disease, parameters of the metabolic profile, renal function and early biomarkers of vascular endothelial damage potentially related to long-term cardiovascular risk. Assessment of the patients' quality of life and their adherence to the recommendations.

Conditions and MedDRA coding

Protein-energy wasting in nephrotic syndrome

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Nephrotic syndrome defined as a 24-hour proteinuria greater than 3.5 g (or greater than 50 mg/kg), or a urine protein-to-creatinine ratio (uPCR) greater than 3500 mg/g, and a serum albumin concentration less than or equal to 3.5 g/dL.
2. Glomerular filtration rate equal to or higher than 30 mL/min/1.73m2 based on Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI 2021) equation
3. Age minimum 18 years.

Exclusion criteria 8

1. Body mass index (BMI) equal to or greater than 40 kg/m2.
2. Uncontrolled diabetes defined as glycated hemoglobin (HbA1c) level greater than 8% (applicable only to patients with diabetes).
3. Pregnancy.
4. Contraindications to treatment with the preparation of exogenous α-ketoanalogues amino acids.
5. Any disease or condition of the patient that may, in the Investigator's opinion, interfere with the interpretation of the results or with the treatment provided in the clinical trial or otherwise prevent participation in the clinical trial and compliance with the requirements of the study protocol.
6. Addiction to alcohol, psychoactive substances or psychotropic drugs within 1 year prior to the screening visit.
7. Participation in another clinical trial with an unauthorised investigational medicinal product within 6 months preceding the screening visit.
8. Elapsed time less than 5 half-life periods since the last administration an investigational medicinal product in another clinical trial, on the date of randomization of the patient (visit 1).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in lean body mass from baseline within 6 weeks after enrollment in the study (non-inferiority).

Secondary endpoints 8

1. Change in lean body mass from baseline within 6 weeks after enrollment in the study (superiority).
2. Change in handgrip strength value of the dominant handwithin 6 weeks after enrollment in the study.
3. Change from baseline in serum albumin concentration at Week 6.
4. Change from baseline in urine protein-to-creatinine ratio (uPCR) at Week 6.
5. Change from baseline in serum low-density lipoprotein (LDL) concentration at Week 6.
6. Change from baseline in serum triglyceride concentration at Week 6.
7. Change from baseline in serum uric acid concentration at Week 6.
8. Safety assessment including the analysis of adverse events (according to CTCAE and MedDRA), evaluation of the relationship between adverse events and the investigational medicinal product, and analysis of selected laboratory parameters during the treatment period, the mandatory follow-up period, and the observation period.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy

Sponsor organisation

Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy

Address

Ulica Szaserow 128

City

Warsaw

Postcode

04-141

Country

Poland

Scientific contact point

Organisation

Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy

Contact name

Co-ordinating Investigator

Public contact point

Organisation

Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy

Contact name

Wydział Wsparcia i Zarządzania Projektami

Locations

1 EU/EEA country · 8 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 6 files

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

Application history

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