Trial Outcomes & Findings for Efficacy of Minoxidil in Children With Williams-Beuren Syndrome (NCT NCT00876200)

NCT ID: NCT00876200

Last Updated: 2025-09-30

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

12 months

Results posted on

2025-09-30

Participant Flow

Participant milestones

Participant milestones
Measure
Minoxidil
Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more. Minoxidil: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Placebo
Placebo = lactose Placebo: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Overall Study
STARTED
9
12
Overall Study
COMPLETED
8
9
Overall Study
NOT COMPLETED
1
3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Minoxidil
n=9 Participants
Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more. Minoxidil: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Placebo
n=12 Participants
Placebo = lactose Placebo: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Total
n=21 Participants
Total of all reporting groups
Age, Continuous
12.33 years
STANDARD_DEVIATION 4.42 • n=9 Participants
10.75 years
STANDARD_DEVIATION 3.77 • n=12 Participants
11.43 years
STANDARD_DEVIATION 4.03 • n=21 Participants
Sex: Female, Male
Female
5 Participants
n=9 Participants
4 Participants
n=12 Participants
9 Participants
n=21 Participants
Sex: Female, Male
Male
4 Participants
n=9 Participants
8 Participants
n=12 Participants
12 Participants
n=21 Participants
Race and Ethnicity Not Collected
0 Participants
Race and Ethnicity were not collected from any participant.
History of hypertension
2 Participants
n=9 Participants
2 Participants
n=12 Participants
4 Participants
n=21 Participants
History of cardiovascular disease
4 Participants
n=9 Participants
6 Participants
n=12 Participants
10 Participants
n=21 Participants
Systolic Blood Pressure
128.11 mmHg
STANDARD_DEVIATION 16.78 • n=9 Participants
120.83 mmHg
STANDARD_DEVIATION 12.68 • n=12 Participants
123.95 mmHg
STANDARD_DEVIATION 14.65 • n=21 Participants
Diastolic Blood Pressure
78.22 mmHg
STANDARD_DEVIATION 15.58 • n=9 Participants
73.17 mmHg
STANDARD_DEVIATION 8.97 • n=12 Participants
75.33 mmHg
STANDARD_DEVIATION 12.16 • n=21 Participants
BMI
17.93 Kg/cm2
STANDARD_DEVIATION 3.88 • n=9 Participants
17.54 Kg/cm2
STANDARD_DEVIATION 3.30 • n=12 Participants
17.71 Kg/cm2
STANDARD_DEVIATION 3.47 • n=21 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure
Minoxidil
n=9 Participants
Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more. Minoxidil: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Placebo
n=9 Participants
Placebo = lactose Placebo: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography
0.028 mm
Interval 0.002 to 0.058
0.012 mm
Interval 0.0017 to 0.04

SECONDARY outcome

Timeframe: 18 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 18 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 0

Outcome measures

Outcome data not reported

Adverse Events

Minoxidil

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Minoxidil
n=9 participants at risk
Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more. Minoxidil: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Placebo
n=12 participants at risk
Placebo = lactose Placebo: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Musculoskeletal and connective tissue disorders
Flat foot
0.00%
0/9
8.3%
1/12 • Number of events 1
Surgical and medical procedures
Strabismus correction
11.1%
1/9 • Number of events 1
0.00%
0/12

Other adverse events

Other adverse events
Measure
Minoxidil
n=9 participants at risk
Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more. Minoxidil: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Placebo
n=12 participants at risk
Placebo = lactose Placebo: Normotension: 0.2mg/kg/day for children under 12 and 5mg/day for children aged 12 or more. Hypertension: 0.2mg/kg/day, increasing up to a maximal dosage of 1 mg/kg) for children under 12. 5mg/day, increasing as needed of 0.1 mg/kg/day (up to a maximal dosage of 40 mg/day) for children aged 12 or more.
Skin and subcutaneous tissue disorders
Hypertrichosis
55.6%
5/9 • Number of events 5
0.00%
0/12

Additional Information

Dr Behrouz KASSAI-KOUPAI

Hospices Civils de Lyon

Phone: 472116911

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place