Trial Outcomes & Findings for SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial) (NCT NCT03806452)
NCT ID: NCT03806452
Last Updated: 2025-05-14
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
86 participants
Primary outcome timeframe
6 months
Results posted on
2025-05-14
Participant Flow
From 28 May 2019 to 30 May 2024, 86 patients were included in the study in the mITT population and analysed in France and Africa (Mali, Ivory Coast and Senegal).
Participant milestones
| Measure |
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
|
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
|
|---|---|---|
|
Initial Treatment Period
STARTED
|
46
|
40
|
|
Initial Treatment Period
COMPLETED
|
44
|
38
|
|
Initial Treatment Period
NOT COMPLETED
|
2
|
2
|
|
Responders Extended Treatment Period
STARTED
|
18
|
15
|
|
Responders Extended Treatment Period
COMPLETED
|
15
|
9
|
|
Responders Extended Treatment Period
NOT COMPLETED
|
3
|
6
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
one patient with missing information
Baseline characteristics by cohort
| Measure |
Hydroxycarbamide
n=46 Participants
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
|
Placebo
n=40 Participants
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
|
Total
n=86 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
30.3 years
STANDARD_DEVIATION 10.0 • n=45 Participants • one patient with missing information
|
30.0 years
STANDARD_DEVIATION 8.7 • n=40 Participants • one patient with missing information
|
30.2 years
STANDARD_DEVIATION 9.3 • n=85 Participants • one patient with missing information
|
|
Sex: Female, Male
Female
|
32 Participants
n=46 Participants
|
27 Participants
n=40 Participants
|
59 Participants
n=86 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=46 Participants
|
13 Participants
n=40 Participants
|
27 Participants
n=86 Participants
|
|
Race/Ethnicity, Customized
Continent of origin · Africa - America
|
45 Participants
n=46 Participants
|
40 Participants
n=40 Participants
|
85 Participants
n=86 Participants
|
|
Race/Ethnicity, Customized
Continent of origin · Other
|
1 Participants
n=46 Participants
|
0 Participants
n=40 Participants
|
1 Participants
n=86 Participants
|
|
Region of Enrollment
Senegal
|
17 participants
n=46 Participants
|
12 participants
n=40 Participants
|
29 participants
n=86 Participants
|
|
Region of Enrollment
Mali
|
12 participants
n=46 Participants
|
8 participants
n=40 Participants
|
20 participants
n=86 Participants
|
|
Region of Enrollment
Guadeloupe
|
0 participants
n=46 Participants
|
1 participants
n=40 Participants
|
1 participants
n=86 Participants
|
|
Region of Enrollment
Côte D'Ivoire
|
5 participants
n=46 Participants
|
4 participants
n=40 Participants
|
9 participants
n=86 Participants
|
|
Region of Enrollment
Martinique
|
1 participants
n=46 Participants
|
0 participants
n=40 Participants
|
1 participants
n=86 Participants
|
|
Region of Enrollment
France
|
11 participants
n=46 Participants
|
15 participants
n=40 Participants
|
26 participants
n=86 Participants
|
|
SCD genotype
HbSS
|
43 Participants
n=46 Participants
|
37 Participants
n=40 Participants
|
80 Participants
n=86 Participants
|
|
SCD genotype
HbSbeta0
|
3 Participants
n=46 Participants
|
3 Participants
n=40 Participants
|
6 Participants
n=86 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: 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
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.
Outcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value
|
18 Participants
|
15 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in eGFR Value
|
3.2 ml/min/1.73m2
Standard Deviation 18.5
|
-1.0 ml/min/1.73m2
Standard Deviation 13.8
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in ACR Value
|
3.5 mg/mmol
Standard Error 19.7
|
1.0 mg/mmol
Standard Error 13.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=8 Participants
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
|
|---|---|---|
|
Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria
|
10 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Proportion of Patients With a Shift From Microalbuminuria to Macroalbuminuria
|
3 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=40 Participants
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
|
Placebo
n=34 Participants
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
|
|---|---|---|
|
Absolute Mean Changes of Systolic Blood Pressure
|
2.8 mmHg
Standard Deviation 10.7
|
2.2 mmHg
Standard Deviation 13.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=37 Participants
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
|
|---|---|---|
|
Absolute Mean Changes of Body Weight
|
2.2 kg
Standard Deviation 3.0
|
-0.3 kg
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=40 Participants
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
|
Placebo
n=35 Participants
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
|
|---|---|---|
|
Absolute Mean Changes of Diastolic Blood Pressure
|
2.5 mmHg
Standard Deviation 13.3
|
-1.0 mmHg
Standard Deviation 12.9
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=41 Participants
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
|
Placebo
n=37 Participants
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
|
|---|---|---|
|
Absolute Mean Changes of Heart Rate Measure
|
-2.1 beats/minute
Standard Deviation 11.3
|
2.4 beats/minute
Standard Deviation 12.5
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=37 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in White Blood Cells Count
|
-3659.6 cells/mm3
Standard Deviation 3524.4
|
-228.4 cells/mm3
Standard Deviation 2982.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=37 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Platelets Count
|
-49.3 10^9 platelets per liter
Standard Deviation 150.9
|
-16.0 10^9 platelets per liter
Standard Deviation 83.5
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=37 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Mean Corpuscular Volume
|
15.1 fL
Standard Deviation 10.8
|
-0.3 fL
Standard Deviation 3.4
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=33 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Mean Corpuscular Haemoglobin Concentration
|
1.7 g/L
Standard Deviation 15.8
|
-4.8 g/L
Standard Deviation 16.4
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=34 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Mean Corpuscular Haemoglobin
|
5.5 pg
Standard Deviation 4.0
|
-0.8 pg
Standard Deviation 1.6
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=44 Participants
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
|
Placebo
n=36 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Hemoglobin Count
|
12 g/L
Standard Deviation 11.3
|
-2.2 g/L
Standard Deviation 6.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=24 Participants
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
|
Placebo
n=24 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Foetal Hemoglobin Count
|
8.5 percentage of hemoglobin
Standard Deviation 8.8
|
0.3 percentage of hemoglobin
Standard Deviation 1.9
|
SECONDARY outcome
Timeframe: 6 months and 12 months for responder patients willing to continue the study after month 6.Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 months and 12 months for responder patients willing to continue the study after month 6.Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 monthsPopulation: Very few patients have an evaluation of endogenous erythropoietin count. Only 3 in hydroxycarbamide group and 6 in placebo group
Outcome measures
| Measure |
Hydroxycarbamide
n=3 Participants
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
|
Placebo
n=6 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Endogenous Erythropoietin Count
|
-8.2 U/L
Standard Deviation 22.1
|
17.4 U/L
Standard Deviation 37.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=41 Participants
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
|
Placebo
n=35 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Ferritin Count
|
28.1 µg/L
Standard Deviation 664.2
|
-8.0 µg/L
Standard Deviation 404.3
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=42 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Lactate Dehydrogenase
|
-170.5 U/L
Standard Deviation 349.4
|
-1.8 U/L
Standard Deviation 220.9
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Aspartate Aminotransferase
|
-8.7 U/L
Standard Deviation 19.2
|
-1.0 U/L
Standard Deviation 10.9
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=43 Participants
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
|
Placebo
n=38 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Alanine Amino Transferase
|
-1.7 U/L
Standard Deviation 51.2
|
-0.2 U/L
Standard Deviation 13.7
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=42 Participants
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
|
Placebo
n=36 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Blood Urea Nitrogen
|
-0.5 mmol/L
Standard Deviation 6.4
|
0.5 mmol/L
Standard Deviation 2.6
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=39 Participants
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
|
Placebo
n=35 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Conjugated Bilirubin
|
-0.7 µmol/L
Standard Deviation 33.8
|
-1.9 µmol/L
Standard Deviation 9.8
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=40 Participants
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
|
Placebo
n=35 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Total Bilirubin
|
-8.9 µmol/L
Standard Deviation 106.5
|
4.7 µmol/L
Standard Deviation 30.1
|
SECONDARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Hydroxycarbamide
n=40 Participants
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
|
Placebo
n=36 Participants
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
|
|---|---|---|
|
Absolute Mean Changes in Reticulocytes
|
-116082.5 cells/mm3
Standard Deviation 168548.6
|
-35336.1 cells/mm3
Standard Deviation 108156.3
|
Adverse Events
Hydroxycarbamide
Serious events: 5 serious events
Other events: 30 other events
Deaths: 0 deaths
Placebo
Serious events: 7 serious events
Other events: 29 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Hydroxycarbamide
n=46 participants at risk
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
|
Placebo
n=40 participants at risk
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
|
|---|---|---|
|
Blood and lymphatic system disorders
SICKLE CELL ANAEMIA WITH CRISIS
|
4.3%
2/46 • Number of events 2 • 6 months
|
5.0%
2/40 • Number of events 2 • 6 months
|
|
Blood and lymphatic system disorders
ANAEMIA
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
ACUTE CHEST SYNDROME
|
0.00%
0/46 • 6 months
|
5.0%
2/40 • Number of events 2 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
Hepatobiliary disorders
CHOLECYSTITIS
|
2.2%
1/46 • Number of events 1 • 6 months
|
0.00%
0/40 • 6 months
|
|
Hepatobiliary disorders
CHOLELITHIASIS
|
2.2%
1/46 • Number of events 1 • 6 months
|
0.00%
0/40 • 6 months
|
|
Hepatobiliary disorders
HEPATIC PAIN
|
2.2%
1/46 • Number of events 1 • 6 months
|
0.00%
0/40 • 6 months
|
|
Infections and infestations
INFLUENZA
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
Infections and infestations
PNEUMONIA
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
2.2%
1/46 • Number of events 1 • 6 months
|
0.00%
0/40 • 6 months
|
|
General disorders
PYREXIA
|
2.2%
1/46 • Number of events 1 • 6 months
|
0.00%
0/40 • 6 months
|
|
Injury, poisoning and procedural complications
HEPATOBILIARY PROCEDURAL COMPLICATION
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
Musculoskeletal and connective tissue disorders
BONE INFARCTION
|
0.00%
0/46 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
Other adverse events
| Measure |
Hydroxycarbamide
n=46 participants at risk
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
|
Placebo
n=40 participants at risk
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
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
10.9%
5/46 • Number of events 6 • 6 months
|
15.0%
6/40 • Number of events 6 • 6 months
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
6.5%
3/46 • Number of events 3 • 6 months
|
7.5%
3/40 • Number of events 3 • 6 months
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
6.5%
3/46 • Number of events 4 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
General disorders
ASTHENIA
|
4.3%
2/46 • Number of events 2 • 6 months
|
12.5%
5/40 • Number of events 5 • 6 months
|
|
General disorders
CHEST PAIN
|
2.2%
1/46 • Number of events 1 • 6 months
|
10.0%
4/40 • Number of events 5 • 6 months
|
|
General disorders
PYREXIA
|
8.7%
4/46 • Number of events 6 • 6 months
|
2.5%
1/40 • Number of events 1 • 6 months
|
|
General disorders
FATIGUE
|
2.2%
1/46 • Number of events 1 • 6 months
|
7.5%
3/40 • Number of events 3 • 6 months
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
8.7%
4/46 • Number of events 6 • 6 months
|
7.5%
3/40 • Number of events 3 • 6 months
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
4.3%
2/46 • Number of events 2 • 6 months
|
7.5%
3/40 • Number of events 3 • 6 months
|
|
Nervous system disorders
HEADACHE
|
17.4%
8/46 • Number of events 9 • 6 months
|
12.5%
5/40 • Number of events 6 • 6 months
|
|
Blood and lymphatic system disorders
SICKLE CELL ANAEMIA WITH CRISIS
|
6.5%
3/46 • Number of events 3 • 6 months
|
17.5%
7/40 • Number of events 7 • 6 months
|
|
Ear and labyrinth disorders
VERTIGO
|
6.5%
3/46 • Number of events 3 • 6 months
|
12.5%
5/40 • Number of events 5 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
ACUTE CHEST SYNDROME
|
0.00%
0/46 • 6 months
|
7.5%
3/40 • Number of events 3 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place