SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)

NCT03806452 | Completed Phase 2 Results DMC

• 1 other identifier: SIK-FR-17-1
• Study Type: interventional
• Target: 86
• Locations: 5 countries
• Sites: 20

Brief Summary

The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.

Conditions

Sickle Cell Disease

Keywords

albuminuria; hydroxycarbamide

Outcome Measures

Primary Outcomes (1)

• Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value

  The primary endpoint of this study is the proportion of patients in the hydroxycarbamide and placebo groups achieving at least a 30% decrease in ACR baseline value at 6 months after treatment initiation. Patients who do not achieve at least a 30% decrease of the ACR baseline value at month 6 will be considered non-responders.

  6 months

Secondary Outcomes (28)

• Absolute Mean Changes in eGFR Value

  6 months

• Absolute Mean Changes in ACR Value

  6 months

• Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria

  6 months

• Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria

  6 months

• Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria

  6 months

Study Arms (2)

Hydroxycarbamide

EXPERIMENTAL

Hydroxycarbamide will be supplied as 100 mg or 1000 mg film-coated tablets. The posology will be based on the patient's body weight (bw). Hydroxycarbamide will be prescribed at a dose of 15 mg/kg bw/day and will be adminitered for 6 months. Hydroxycarbamide will be administered for 12 months for patients qualified as responders willing to continue to participate in the trial.

Drug: Hydroxycarbamide

Placebo

PLACEBO COMPARATOR

Placebo will be supplied as 100 mg or 1000 mg film-coated tablets. The posology will be based on the patient's body weight (bw). Placebo will be prescribed at a dose of 15 mg/kg bw/day and will be adminitered for 6 months. Hydroxycarbamide will be administered for 12 months for patients qualified as responders willing to continue to participate in the trial.

Drug: Placebo Oral Tablet

Interventions

Hydroxycarbamide DRUG

Hydroxycarbamide tablets of 100 and 1000 mg

Also known as: Siklos

Hydroxycarbamide

Placebo Oral Tablet DRUG

Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed and dated Informed Consent Form (ICF) by a legally competent patient.
• Patients above 18 years.
• Patients with HbSS or HbSβ0 SCD.
• Patients with a value of albuminuria, assessed by ACR, over 3 mg/mmol and inferior to 100 mg/mmol confirmed by 3 positive urine samples taken one day apart.
• Female patients of childbearing potential or postmenopausal female with last period \< 12 months before screening agreeing to use a highly effective form of contraception (oral, injected or implanted hormonal contraception, intrauterine device, diaphragm, condom) during the trial and for 3 months after hydroxycarbamide discontinuation.
• Male patients with partners of childbearing potential agreeing to use a highly effective contraception during the trial and for 3 months after hydroxycarbamide discontinuation. Men with pregnant or lactating women should be advised to use a barrier method of contraception (condom) to prevent the foetus or breastfed infant from exposure to hydroxycarbamide.
• Patients who are covered by insurance scheme according to local regulatory requierements.

You may not qualify if:

• Patients who had severe VOC requiring hospitalisation or ACS within the last 4 weeks preceding screening visit.
• Patients treated with hydroxycarbamide for any reason within the previous 6 months.
• Patients who have had chronic blood transfusion or transfusion in the last 3 months.
• Patients with a history of hypertension (systolic blood pressure ≥ 140 or diastolic blood pressure ≥ 90 mmHg) treated with antihypertensive agent belonging to pharmacological class of RAS inhibitor.
• Patients who have symptoms suggestive of urinary tract infection or patients with gross haematuria.
• Patients with a concomitant primary kidney disease.
• Patients with any systemic condition that could result in a glomerulopathy not related to SCD (e.g. diabetes mellitus, active hepatitis B or C infections, HIV infection, systemic lupus erythematosus, inflammatory arthropathies).
• Patient with a stage 3, 4 or 5 chronic kidney disease (eGFR \< 60 mL/min per 1.73 m2).
• Patients with eGFR ≥ 140 ml/min/1,73m² due to the lack of information regarding the magnitude, direction and significance of the trends in eGFR evolution that could be expected in this population
• Patients requiring long-term treatment with drugs potentially nephrotoxic (see non-exhaustive list).
• Patients requiring long-term treatment with non-steroid anti-inflammatory drugs.
• Patients who have a treatment which can modify the kidney function (see non-exhaustive list) in the last 3 months.
• Patients known to be infected with HIV.
• Female patients who are pregnant or lactating.
• Unreliable patients including non-compliant patients, patients with known alcoholism or drug abuse or with a history of a serious psychiatric disorder as well as patients unwilling to give informed consent or to abide by the requirements of the protocol.

Sponsors & Collaborators

• Theravia: lead

Study Sites (21)

Centre Suisse de Recherches Scientifiques en Côte d'Ivoire (CSRS)

Abidjan, Côte d’Ivoire

Location

CHU d'Angers

Angers, France

Location

Hôpital Saint-André

Bordeaux, France

Location

CHRU Brest

Brest, France

Location

Hôpital Louis Mourier

Colombes, France

Location

Pablo Bartolucci

Créteil, 94017, France

Location

Hopital Edouard Herriot

Lyon, France

Location

Hôpital de la Timone

Marseille, France

Location

Hôpital européen Georges-Pompidou

Paris, France

Location

Hôpital Saint-Antoine

Paris, France

Location

Service de biothérapie, consultation hématologie-drépanocytose hôpital Necker

Paris, France

Location

CHU la Miletrie

Poitiers, France

Location

Hôpital Robert Debré CHU Reims

Reims, France

Location

Hopital Pontchaillou

Rennes, France

Location

CHU Charles Nicolle

Rouen, France

Location

Centre Hospitalier Delafontaine

Saint-Denis, France

Location

Institut Universitaire du Cancer de Toulouse - Oncopole

Toulouse, France

Location

CHU Pointe-à-Pitre/Abymes

Pointe-à-Pitre, Guadeloupe

Location

Centre de Recherche et de Lutte contre la Drépanocytose de Bamako (CRLD)

Bamako, Mali

Location

CHU Martinique

Le Lamentin, Martinique

Location

Service d'Hématologie Clinique, Centre National de Transfusion sanguine, Université Cheikh Anta Diop

Dakar, Senegal

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Albuminuria

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Proteinuria; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Results Point of Contact

• Title: Clinical Operation Manager
• Organization: THERAVIA

laura.thomas-bourgneuf@theravia.com

+330149709583

Study Officials

• Pablo Bartolucci, Pr

  Henri Mondor University Hospital

  PRINCIPAL INVESTIGATOR

• Vincent Audard, Pr

  Henri Mondor University Hospital

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2019
• First Posted: January 16, 2019
• Study Start: May 28, 2019
• Primary Completion: May 30, 2024
• Study Completion: May 30, 2024
• Last Updated: May 14, 2025
• Results First Posted: May 14, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

GU (1); FR (16); MA (1); CÔ (1); SE (1)