CClinicalTrials.gg
Sign In
CompletedNCT00876200WilliamsUpdated Sep 30, 2025Results posted

Efficacy of Minoxidil in Children With Williams-Beuren Syndrome

A Phase 2 interventional study of Minoxidil and Placebo in Williams Beuren Syndrome, sponsored by Hospices Civils de Lyon. Completed at 18 sites in France. Open to participants aged 6 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-09-30.

Sponsored by Hospices Civils de Lyon · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
21
Allocation
Randomized
Ages
6 Years to 18 Years
Sex
All
01

Study summary

The Williams-Beuren syndrome (WBS) is a sporadic congenital disorder characterized by a multisystem developmental impairment. This syndrome is caused by a microdeletion in chromosome 7q11.23 that encompasses loss of the elastin locus.

Elastin, which is part of the extracellular matrix, controls proliferation of vascular smooth muscle cells (VSMCs) and stabilizes arterial structure. Loss of elastin gene in WBS patients has been claimed to provide a biological basis for the abnormal elastic fibre properties leading to cardiovascular abnormalities like supravalvular aortic stenosis (SVAS), hypertension, arteriosclerosis and stenosis in more than 50% of WBS children.

These cardiovascular pathologies result in important consequences and neither curative nor preventive medicinal treatments exist at this time. Surgery is needed in more than half cases, while it is often leading to complications.

Minoxidil is a well-known antihypertensive drug used in adults and children. Furthermore, according to animal studies, minoxidil seems to increase arterial elastin content by decreasing elastase activity in these tissues. Other data demonstrate that minoxidil specifically stimulate elastin synthesis.

Working Hypothesis:If insufficient elastin synthesis leads to vascular complications and arterial hypertension in children with WBS, restoration of sufficient quantity of elastin should then result in prevention or inhibition of vascular malformations and improvement in arterial tension. Therefore, as a pharmacological agent capable to stimulate elastin expression, minoxidil might be a useful drug for the treatment of abnormal elastin metabolism in WBS children.

Objective:To evaluate the efficacy of minoxidil on cardiovascular structure in children with Williams Beuren syndrome.

Methodology: randomized controlled trial on two parallel group (23 patients in each arm) Main criterion:variation of carotid Intima-media thickness (IMT) before and after 12 months of treatment with Minoxidil versus placebo Secondary intermediate criteria of the vascular properties are arterial stiffness, cardiac and renal stenosis, arterial tension.

Total study duration:30 months including a 12 month-recruitment period

02

Conditions studied

  • Williams Beuren Syndrome

Keywords

  • Williams Beuren syndrome
  • Cardiovascular abnormalities
  • Cardiovascular structure
03

In context

Williams Syndrome

29 studies on the registry are indexed under Williams Syndrome; 11 are open to participants now.

This study's enrollment of 21 is below the median of 60 across 12 interventional studies indexed under Williams Syndrome.

Browse Williams Syndrome studies →

Lead sponsor

Hospices Civils de Lyon is the lead sponsor of 1,826 studies on the registry; 439 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • proven diagnosis of Williams Beuren syndrome (genetic test)
  • normotension or hypertension, treated or not
  • male or female,
  • 6\< age \<18,
  • negative pregnancy test for childbearing potential female
  • effective birth control for sexually active female
  • signed consent form collected from parents or legal guardian

Exclusion criteria

Exclusion Criteria:

  • pulmonary hypertension secondary to mitral stenosis
  • myocardial infarction within 1 month prior randomization
  • known allergies to minoxidil or any of the components of Lonoten.
  • asthma
  • renal failure (creatinine clearance \<40ml/min)
  • no affiliation to a national health insurance program (social security)
  • intolerance to lactose
  • current vasodilator anti hypertensive treatment
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    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.

    Drug: Minoxidil

  • Placebo comparator
    Placebo

    Placebo = lactose

    Drug: Placebo

Interventions

  • DrugMinoxidil

    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.

  • DrugPlacebo

    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.

06

What researchers measure

Primary outcomes

  1. Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography

    Time frame: 12 months

Secondary outcomes

  1. Efficacy of Minoxidil on Humeral IMT Assessed by Vascular Echography

    Time frame: 18 months

  2. Efficacy of Minoxidil on Arterial Stiffness (Pulse Wave Velocity and Vascular Compliance at J0, M12 and M18)

    Time frame: 18 months

  3. Efficacy of Minoxidil on Supravalvular Stenosis, Pulmonary Stenosis, Aortic Stenosis and Renal Stenosis (Cardiac and Renal Echodoppler at J0, and M12)

    Time frame: 12 months

  4. Efficacy of Minoxidil on Arterial Tension (24H-Holter at J0 and M12)

    Time frame: 12 months

  5. Effect of Minoxidil on Neurohumoral Mechanisms of Cardiovascular Regulation and on Plasmatic Markers of the Extracellular Matrix.

    Time frame: 12 months

  6. Genetic Study: Characterization of Deletions Responsible for WBS (Size Deletion, DNA Sample at Inclusion).

    Time frame: Day 0

07

Results

Posted Jul 26, 2019

Participant flow

Participant flow — Overall Study
MilestoneMinoxidilPlacebo
Started912
Completed89
Not completed13

Outcome measures

PrimaryVariation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography
Time frame:
12 months
Reported as:
Mean · mm
Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography
mmMinoxidilPlacebo
Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography0.028 (0.002 to 0.058)0.012 (0.0017 to 0.040)
SecondaryEfficacy of Minoxidil on Humeral IMT Assessed by Vascular Echography
Time frame:
18 months

Results for this outcome have not been posted.

SecondaryEfficacy of Minoxidil on Arterial Stiffness (Pulse Wave Velocity and Vascular Compliance at J0, M12 and M18)
Time frame:
18 months

Results for this outcome have not been posted.

SecondaryEfficacy of Minoxidil on Supravalvular Stenosis, Pulmonary Stenosis, Aortic Stenosis and Renal Stenosis (Cardiac and Renal Echodoppler at J0, and M12)
Time frame:
12 months

Results for this outcome have not been posted.

SecondaryEfficacy of Minoxidil on Arterial Tension (24H-Holter at J0 and M12)
Time frame:
12 months

Results for this outcome have not been posted.

SecondaryEffect of Minoxidil on Neurohumoral Mechanisms of Cardiovascular Regulation and on Plasmatic Markers of the Extracellular Matrix.
Time frame:
12 months

Results for this outcome have not been posted.

SecondaryGenetic Study: Characterization of Deletions Responsible for WBS (Size Deletion, DNA Sample at Inclusion).
Time frame:
Day 0

Results for this outcome have not been posted.

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Minoxidil—1/9 (11.1%)5/9 (55.6%)
Placebo—1/12 (8.3%)0/12 (0%)
Most frequent serious events
Most frequent serious events
EventMinoxidilPlacebo
Strabismus correctionSurgical and medical procedures1/90/12
Flat footMusculoskeletal and connective tissue disorders0/91/12
Most frequent other events
Most frequent other events
EventMinoxidilPlacebo
HypertrichosisSkin and subcutaneous tissue disorders5/90/12

Baseline characteristics

Age, Continuous
Age, Continuous(years)MinoxidilPlaceboTotal
Mean12.33 ± 4.4210.75 ± 3.7711.43 ± 4.03
Sex: Female, Male
Sex: Female, Male(Participants)MinoxidilPlaceboTotal
Female549
Male4812
Race and Ethnicity Not Collected
Race and Ethnicity Not Collected(Participants)MinoxidilPlaceboTotal
Count of participants——0
History of hypertension
History of hypertension(Participants)MinoxidilPlaceboTotal
Count of participants224
History of cardiovascular disease
History of cardiovascular disease(Participants)MinoxidilPlaceboTotal
Count of participants4610
Systolic Blood Pressure
Systolic Blood Pressure(mmHg)MinoxidilPlaceboTotal
Mean128.11 ± 16.78120.83 ± 12.68123.95 ± 14.65
Diastolic Blood Pressure
Diastolic Blood Pressure(mmHg)MinoxidilPlaceboTotal
Mean78.22 ± 15.5873.17 ± 8.9775.33 ± 12.16
BMI
BMI(Kg/cm2)MinoxidilPlaceboTotal
Mean17.93 ± 3.8817.54 ± 3.3017.71 ± 3.47
08

Study locations

18 sites
  • Service de Cardiologie Pédiatrique, CHU Angers
    Angers, 49033, France
  • Service de Cardiologie, Hôpital Saint-André, CHU Bordeaux
    Bordeaux, 33075, France
  • Service de Néphrologie Pédiatrique, Hôpital Pellegrin, CHU Bordeaux
    Bordeaux, 33076, France
  • Service de Génétique Médicale, Hôpital Pellegrin, CHU Bordeaux
    Bordeaux, France
  • Département de Pédiatrie, Hôpital Femme Mère Enfant
    Bron, 69677, France
  • Service de Cardiologie Pédiatrique, Hôpital Cardiovasculaire L. Pradel
    Bron, 69677, France
  • Service Cardiologie, CHU St Jacques
    Clermont-Ferrand, 63000, France
  • Département de Pédiatrie- Service de Cardiologie, CHU Grenoble
    Grenoble, 38043, France
  • Service de Néphrologie Pédiatrique, CHRU de Lille
    Lille, 59000, France
  • Service des Maladies Cardiovasculaires Infantiles et Congénitales, CHRU Lille
    Lille, 59000, France
  • Service de Cardiologie Infantile, CHU Nancy
    Nancy, 54511, France
  • Service de Cardiologie Pédiatrique, Hôpital Necker Enfants Malades
    Paris, 75015, France
  • Service de Physiologie, Explorations Fonctionnelles, Hôpital Robert Debré
    Paris, 75019, France
  • Unité de Pharmacologie Clinique, Hôpital Robert Debré
    Paris, 75019, France
  • Service de Pathologie Cardiaque Congénitale du Fœtus, de l'Enfant et de l'Adulte, Hôpital Haut Lévêque, CHU de Bordeaux
    Pessac, 33604, France
  • Service de Génétique Médicale, CHU La Milétrie
    Poitiers, 86021, France
  • Service de Cardiologie - Hôpital des Enfants
    Toulouse, 31059, France
  • Service de Néphrologie Pédiatrique - Hôpital des Enfants, CHU Toulouse
    Toulouse, 31059, France
09

References and documents

Publications

  • Kassai B, Bouye P, Gilbert-Dussardier B, Godart F, Thambo JB, Rossi M, Cochat P, Chirossel P, Luong S, Serusclat A, Canterino I, Mercier C, Rabilloud M, Pivot C, Pirot F, Ginhoux T, Coopman S, Grenet G, Gueyffier F, Di-Fillippo S, Bertholet-Thomas A. Minoxidil versus placebo in the treatment of arterial wall hypertrophy in children with Williams Beuren Syndrome: a randomized controlled trial. BMC Pediatr. 2019 May 28;19(1):170. doi: 10.1186/s12887-019-1544-1. PubMed 31138170 ↗

Related links

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 30, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00876200
Lead sponsor
Hospices Civils de Lyon
Responsible party
Sponsor
First posted
Apr 6, 2009
Start date
Mar 2009
Primary completion
Feb 2015
Completion
Aug 2015
Results posted
Jul 26, 2019
Last update
Sep 30, 2025

Study contacts

Behrouz KASSAI, MD
principal investigator · Hospices Civils de Lyon

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jan 2016. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Williams Syndrome→

Other studies from this sponsor

Hospices Civils de Lyon

Start the discussion