Sickle Cell DiseasePhase 2SIKAMICNCT03806452
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
A Phase 2 study testing Hydroxycarbamide in people with Sickle Cell Disease, run by Theravia at 21 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Hydroxycarbamide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 86 participants
- Where
- 21 sitesin 6 countries
- Study dates
- 28 May 2019to 30 May 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 19Show fewer
- 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 ACE inhibitors or ARBs within the 3 months before inclusion regardless of the indication.
- 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.
- Simultaneous participation in other clinical trials on an investigational medicinal product or previous participation within 30 days before inclusion.
- Persons in detention by judicial or administrative decision.
- Patients with chronic conditions that upon investigator judgment may lead to a limited life expectancy
Trial locations and contacts
21 sites in 6 countries.
21 sites in 6 countries
France16 sites
CHU d'Angers
Angers
Hôpital Saint-André
Bordeaux
CHRU Brest
Brest
Hôpital Louis Mourier
Colombes
Pablo Bartolucci
Créteil, 94017
Hopital Edouard Herriot
Lyon
Hôpital de la Timone
Marseille
Hôpital européen Georges-Pompidou
Paris
Hôpital Saint-Antoine
Paris
Service de biothérapie, consultation hématologie-drépanocytose hôpital Necker
Paris
CHU la Miletrie
Poitiers
Hôpital Robert Debré CHU Reims
Reims
Hopital Pontchaillou
Rennes
CHU Charles Nicolle
Rouen
Centre Hospitalier Delafontaine
Saint-Denis
Institut Universitaire du Cancer de Toulouse - Oncopole
Toulouse
Côte d’Ivoire1 site
Centre Suisse de Recherches Scientifiques en Côte d'Ivoire (CSRS)
Abidjan
Mali1 site
Centre de Recherche et de Lutte contre la Drépanocytose de Bamako (CRLD)
Bamako
Senegal1 site
Service d'Hématologie Clinique, Centre National de Transfusion sanguine, Université Cheikh Anta Diop
Dakar
About this study
Official title: Multicentre Randomized Double-blind Placebo-controlled Study to Evaluate the Effect on Albuminuria of 6 Months Treatment With Hydroxycarbamide (Siklos®) or a Placebo in Adults With Sickle Cell Disease:
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
- Being tested
- Hydroxycarbamide, Placebo Oral Tablet
- Keywords
- albuminuria, hydroxycarbamide
- Medical subject headings
- Anemia, Sickle Cell, Albuminuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxycarbamideDrug
Also called Siklos
Hydroxycarbamide tablets of 100 and 1000 mg
Placebo Oral TabletDrug
Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (28)
Absolute Mean Changes in eGFR Value
Measured: 6 months
Absolute Mean Changes in ACR Value
Measured: 6 months
Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria
Measured: 6 months
Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria
Measured: 6 months
Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria
Measured: 6 months
Proportion of Patients With a Shift From Microalbuminuria to Macroalbuminuria
Measured: 6 months
Absolute Mean Changes of Systolic Blood Pressure
Measured: 6 months
Absolute Mean Changes of Body Weight
Measured: 6 months
Absolute Mean Changes of Diastolic Blood Pressure
Measured: 6 months
Absolute Mean Changes of Heart Rate Measure
Measured: 6 months
Absolute Mean Changes in White Blood Cells Count
Measured: 6 months
Absolute Mean Changes in Platelets Count
Measured: 6 months
Absolute Mean Changes in Mean Corpuscular Volume
Measured: 6 months
Absolute Mean Changes in Mean Corpuscular Haemoglobin Concentration
Measured: 6 months
Absolute Mean Changes in Mean Corpuscular Haemoglobin
Measured: 6 months
Absolute Mean Changes in Hemoglobin Count
Measured: 6 months
Absolute Mean Changes in Foetal Hemoglobin Count
Measured: 6 months
Absolute Mean Changes in Free Hemoglobin Count
Measured: 6 months and 12 months for responder patients willing to continue the study after month 6.
Absolute Mean Changes in Dense Red Blood Cells Percentage
Measured: 6 months and 12 months for responder patients willing to continue the study after month 6.
Absolute Mean Changes in Endogenous Erythropoietin Count
Measured: 6 months
Absolute Mean Changes in Ferritin Count
Measured: 6 months
Absolute Mean Changes in Lactate Dehydrogenase
Measured: 6 months
Absolute Mean Changes in Aspartate Aminotransferase
Measured: 6 months
Absolute Mean Changes in Alanine Amino Transferase
Measured: 6 months
Absolute Mean Changes in Blood Urea Nitrogen
Measured: 6 months
Absolute Mean Changes in Conjugated Bilirubin
Measured: 6 months
Absolute Mean Changes in Total Bilirubin
Measured: 6 months
Absolute Mean Changes in Reticulocytes
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2019
- Study start28 May 2019
- Primary completion30 May 2024
- Study completion30 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- Theravia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pablo Bartolucci, Pr, Principal investigator, Henri Mondor University HospitalVincent Audard, Pr, Study chair, Henri Mondor University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03806452
- Sponsor's protocol ID
- SIK-FR-17-1
- Acronym
- SIKAMIC
- Status last verified
- Apr 2025
- First submitted
- 9 Jan 2019
- Last updated
- 14 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentre Randomized Double-blind Placebo-controlled Study to Evaluate the Effect on Albuminuria of 6 Months Treatment With Hydroxycarbamide (Siklos®) or a Placebo in Adults With Sickle Cell Disease:
- ClinicalTrials.gov ID
- NCT03806452
- Acronym / sponsor ID
- SIKAMIC / SIK-FR-17-1
- Conditions
- Sickle Cell Disease
- Keywords
- albuminuria, hydroxycarbamide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- TheraviaIndustry (pharma, biotech, devices)
- Study start
- 28 May 2019 (actual)
- Primary completion
- 30 May 2024 (actual)
- Study completion
- 30 May 2024 (actual)
- First posted
- 16 Jan 2019 (actual)
- Results
- Posted 14 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hydroxycarbamide | Experimental | Drug: HydroxycarbamideHydroxycarbamide 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. |
| Placebo | Placebo comparator | Drug: Placebo Oral TabletPlacebo 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxycarbamideAlso: SiklosHydroxycarbamide tablets of 100 and 1000 mg | Drug | Hydroxycarbamide |
| Placebo Oral TabletPlacebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets | Drug | Placebo |
Primary outcome
Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value
Time frame: 6 months
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.
28 secondary outcomes
Absolute Mean Changes in eGFR Value
Time frame: 6 months
Absolute Mean Changes in ACR Value
Time frame: 6 months
Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria
Time frame: 6 months
Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria
Time frame: 6 months
Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria
Time frame: 6 months
Proportion of Patients With a Shift From Microalbuminuria to Macroalbuminuria
Time frame: 6 months
Absolute Mean Changes of Systolic Blood Pressure
Time frame: 6 months
Absolute Mean Changes of Body Weight
Time frame: 6 months
Absolute Mean Changes of Diastolic Blood Pressure
Time frame: 6 months
Absolute Mean Changes of Heart Rate Measure
Time frame: 6 months
Absolute Mean Changes in White Blood Cells Count
Time frame: 6 months
Absolute Mean Changes in Platelets Count
Time frame: 6 months
Absolute Mean Changes in Mean Corpuscular Volume
Time frame: 6 months
Absolute Mean Changes in Mean Corpuscular Haemoglobin Concentration
Time frame: 6 months
Absolute Mean Changes in Mean Corpuscular Haemoglobin
Time frame: 6 months
Absolute Mean Changes in Hemoglobin Count
Time frame: 6 months
Absolute Mean Changes in Foetal Hemoglobin Count
Time frame: 6 months
Absolute Mean Changes in Free Hemoglobin Count
Time frame: 6 months and 12 months for responder patients willing to continue the study after month 6.
Absolute Mean Changes in Dense Red Blood Cells Percentage
Time frame: 6 months and 12 months for responder patients willing to continue the study after month 6.
Absolute Mean Changes in Endogenous Erythropoietin Count
Time frame: 6 months
Absolute Mean Changes in Ferritin Count
Time frame: 6 months
Absolute Mean Changes in Lactate Dehydrogenase
Time frame: 6 months
Absolute Mean Changes in Aspartate Aminotransferase
Time frame: 6 months
Absolute Mean Changes in Alanine Amino Transferase
Time frame: 6 months
Absolute Mean Changes in Blood Urea Nitrogen
Time frame: 6 months
Absolute Mean Changes in Conjugated Bilirubin
Time frame: 6 months
Absolute Mean Changes in Total Bilirubin
Time frame: 6 months
Absolute Mean Changes in Reticulocytes
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pablo Bartolucci, Pr principal investigator
Henri Mondor University Hospital
Vincent Audard, Pr study chair
Henri Mondor University Hospital
21 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxycarbamide in people with Sickle Cell Disease, run by Theravia at 21 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in France, Côte d’Ivoire, Guadeloupe, Mali, Martinique and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2025.