SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinic... | NCT03806452

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Hydroxycarbamide	Drug	Hydroxycarbamide tablets of 100 and 1000 mg	Hydroxycarbamide

Measure	Description	Time Frame
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

Measure	Description	Time Frame
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

Name	Affiliation	Role
Pablo Bartolucci, Pr	Henri Mondor University Hospital	Principal Investigator
Vincent Audard, Pr	Henri Mondor University Hospital	Study Chair

Facility	Status	City	State	ZIP	Country	Contacts
Centre Suisse de Recherches Scientifiques en Côte d'Ivoire (CSRS)		Abidjan			Côte d’Ivoire
CHU d'Angers

ID	Title	Description
FG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Dec 22, 2022

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

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.

3 patients in the hydroxycarbamide arm and 2 patients in the placebo arm do not have any measure at 6 months and so were not part of this analysis

Posted

Count of Participants

Participants

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants	OG00043 OG00138

Title

Denominators

Categories

Title	Measurements
	OG00018 OG00115

Secondary

Absolute Mean Changes in eGFR Value

Posted

Mean

Standard Deviation

ml/min/1.73m2

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in ACR Value

Posted

Mean

Standard Error

mg/mmol

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria

Posted

Count of Participants

Participants

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria

Posted

Count of Participants

Participants

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria

Posted

Count of Participants

Participants

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Proportion of Patients With a Shift From Microalbuminuria to Macroalbuminuria

Posted

Count of Participants

Participants

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes of Systolic Blood Pressure

Posted

Mean

Standard Deviation

mmHg

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes of Body Weight

Posted

Mean

Standard Deviation

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes of Diastolic Blood Pressure

Posted

Mean

Standard Deviation

mmHg

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes of Heart Rate Measure

Posted

Mean

Standard Deviation

beats/minute

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in White Blood Cells Count

Posted

Mean

Standard Deviation

cells/mm3

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Platelets Count

Posted

Mean

Standard Deviation

10^9 platelets per liter

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Mean Corpuscular Volume

Posted

Mean

Standard Deviation

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Mean Corpuscular Haemoglobin Concentration

Posted

Mean

Standard Deviation

g/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Mean Corpuscular Haemoglobin

Posted

Mean

Standard Deviation

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Hemoglobin Count

Posted

Mean

Standard Deviation

g/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Foetal Hemoglobin Count

Posted

Mean

Standard Deviation

percentage of hemoglobin

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Free Hemoglobin Count

Not Posted

6 months and 12 months for responder patients willing to continue the study after month 6.

Participants

Secondary

Absolute Mean Changes in Dense Red Blood Cells Percentage

Not Posted

6 months and 12 months for responder patients willing to continue the study after month 6.

Participants

Secondary

Absolute Mean Changes in Endogenous Erythropoietin Count

Very few patients have an evaluation of endogenous erythropoietin count. Only 3 in hydroxycarbamide group and 6 in placebo group

Posted

Mean

Standard Deviation

U/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts

Secondary

Absolute Mean Changes in Ferritin Count

Posted

Mean

Standard Deviation

µg/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Lactate Dehydrogenase

Posted

Mean

Standard Deviation

U/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Aspartate Aminotransferase

Posted

Mean

Standard Deviation

U/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Alanine Amino Transferase

Posted

Mean

Standard Deviation

U/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Blood Urea Nitrogen

Posted

Mean

Standard Deviation

mmol/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Conjugated Bilirubin

Posted

Mean

Standard Deviation

µmol/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Total Bilirubin

Posted

Mean

Standard Deviation

µmol/L

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

Secondary

Absolute Mean Changes in Reticulocytes

Posted

Mean

Standard Deviation

cells/mm3

6 months

ID	Title	Description
OG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
OG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets

Units	Counts
Participants

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg	0	46	5	46	30	46
EG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets	2	40	7	40	29	40

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
SICKLE CELL ANAEMIA WITH CRISIS	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0002 events2 affected46 at risk EG0012 events2 affected40 at risk
ANAEMIA	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk
ACUTE CHEST SYNDROME	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0012 events2 affected40 at risk
DYSPNOEA	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk
CHOLECYSTITIS	Hepatobiliary disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0010 events0 affected40 at risk
CHOLELITHIASIS	Hepatobiliary disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0010 events0 affected40 at risk
HEPATIC PAIN	Hepatobiliary disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0010 events0 affected40 at risk
INFLUENZA	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk
PNEUMONIA	Infections and infestations	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk
ABDOMINAL PAIN	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0010 events0 affected40 at risk
PYREXIA	General disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0010 events0 affected40 at risk
HEPATOBILIARY PROCEDURAL COMPLICATION	Injury, poisoning and procedural complications	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk
BONE INFARCTION	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0011 events1 affected40 at risk

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
ARTHRALGIA	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0006 events5 affected46 at risk EG0016 events6 affected40 at risk
BACK PAIN	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0003 events3 affected46 at risk EG0013 events3 affected40 at risk
PAIN IN EXTREMITY	Musculoskeletal and connective tissue disorders	MedDRA 27.1	Systematic Assessment	EG0004 events3 affected46 at risk EG0011 events1 affected40 at risk
ASTHENIA	General disorders	MedDRA 27.1	Systematic Assessment	EG0002 events2 affected46 at risk EG0015 events5 affected40 at risk
CHEST PAIN	General disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0015 events4 affected40 at risk
PYREXIA	General disorders	MedDRA 27.1	Systematic Assessment	EG0006 events4 affected46 at risk EG0011 events1 affected40 at risk
FATIGUE	General disorders	MedDRA 27.1	Systematic Assessment	EG0001 events1 affected46 at risk EG0013 events3 affected40 at risk
ABDOMINAL PAIN UPPER	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0006 events4 affected46 at risk EG0013 events3 affected40 at risk
ABDOMINAL PAIN	Gastrointestinal disorders	MedDRA 27.1	Systematic Assessment	EG0002 events2 affected46 at risk EG0013 events3 affected40 at risk
HEADACHE	Nervous system disorders	MedDRA 27.1	Systematic Assessment	EG0009 events8 affected46 at risk EG0016 events5 affected40 at risk
SICKLE CELL ANAEMIA WITH CRISIS	Blood and lymphatic system disorders	MedDRA 27.1	Systematic Assessment	EG0003 events3 affected46 at risk EG0017 events7 affected40 at risk
VERTIGO	Ear and labyrinth disorders	MedDRA 27.1	Systematic Assessment	EG0003 events3 affected46 at risk EG0015 events5 affected40 at risk
ACUTE CHEST SYNDROME	Respiratory, thoracic and mediastinal disorders	MedDRA 27.1	Systematic Assessment	EG0000 events0 affected46 at risk EG0013 events3 affected40 at risk

ID	Term
D000755	Anemia, Sickle Cell
D000419	Albuminuria

ID	Term
D000745	Anemia, Hemolytic, Congenital
D000743	Anemia, Hemolytic
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D006453	Hemoglobinopathies
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D011507	Proteinuria
D014555	Urination Disorders
D014570	Urologic Diseases
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D020924	Urological Manifestations
D012816	Signs and Symptoms
D013568	Pathological Conditions, Signs and Symptoms

ID	Title	Description
BG000	Hydroxycarbamide	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. Hydroxycarbamide: Hydroxycarbamide tablets of 100 and 1000 mg
BG001	Placebo	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. Placebo Oral Tablet: Placebo tablets of 100 and 1000 mg to mimic hydroxycarbamide tablets
BG002	Total	Total of all reporting groups