Aromatase Inhibitor in Bone Maturation, Children With Silver Russell or Prader-Willi Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 5 September 2016
• Main ID: NCT01520467
• Date of registration: 25/01/2012
• Prospective Registration: Yes
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Aromatase Inhibitor in Bone Maturation, Children With Silver Russell or Prader-Willi Syndrome ANASILPRA
• Scientific title: Efficacy and Tolerance of Treatment With an Aromatase Inhibitor (Anastrozole) to Limit the Progression of Bone Maturation Related to Pathological Adrenarche in Children With Silver-Russell or Prader-Willi Syndrome
• Date of first enrolment: April 2012
• Target sample size: 27
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01520467
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: N/A

Countries of recruitment

France

Contacts

• Name: Irène Netchine, MD, PhD
• Affiliation: Assistance Publique

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with genetically proven SRS or PWS, under treatment with GH in the usual
context of the disease, presenting with adrenarche (defined either by DHEAS levels as
a function of age or by the appearance of pubic hair) associated with a bone age at
least 6 months greater than chronological age and in the absence of the onset of
central puberty (LH peak = LH peak in prepubertal patients, according to the
standards of the laboratory performing the GnRH stimulation test for LH and FSH, and
dating back to less than 3 months).

- Patients with medical coverage.

- The lower age limit for inclusion is 5 years and the upper age limit is 10 complete
years for girls and 12 complete years for boys.

- The maximum body-mass index (BMI) Z-score for inclusion is +4

- Patients should be capable of swallowing pills of the same size as the experimental
drug.

Exclusion Criteria:

- Renal insufficiency (creatinine clearance, calculated according to the Schwartz
formula, lower than 70ml/min/l, 73 m²),

- Hepatic insufficiency (prothrombin ratio < 50% and factor V < 50%),

- Hepatic cytolysis (liver transaminases levels greater than twice the normal level for
age), cholestasis (gamma-glutamyl transferase (GGT) levels greater than twice the
normal level for age),

- Contraindication to one of the components of Anastrozole or the placebo.

- Patients with scoliosis requiring surgery.



Age minimum: 5 Years
Age maximum: 12 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Silver Russell Syndrome

Prader-Willi Syndrome

Intervention(s)

Drug: Anastrozole

Drug: Placebo

Primary Outcome(s)

The rate of success in each of the two groups, evaluated using an X-ray of the left hand and wrist. Success is defined as a difference in the rate of progression of bone maturation of at least 9 months after 18 months of treatment. [Time Frame: 18 months]

Secondary Outcome(s)

impact on the somatotropic axis (growth rate, IGF-1, IGFBP3). [Time Frame: baseline, 6, 12 and 18 months]

impact on bone (X-ray of the dorsolumbar spine, bi photonic absorptiometry, blood-borne markers of bone remodeling). [Time Frame: 18 months, and earlier in case of bone pain]

impact on the gonadotropic axis [Time Frame: baseline, 6, 12 and 18 months]

metabolic impact (monitoring of body composition by bi photonic absorptiometry, lipid, glucose, HbA1c, insulin and HOMA-IR profiles, leptin). [Time Frame: baseline, 6, 12 and 18 months]

Secondary ID(s)

P 100129

AOM 10093

Source(s) of Monetary Support

Please refer to primary and secondary sponsors