Study of the Efficacy of Topiramate in Patients With Prader Willi Syndrome Over 8 Weeks
Status:
Terminated
Trial end date:
2016-06-01
Target enrollment:
Participant gender:
Summary
There is no specific treatment for core symptoms of PWS. Regarding behavioral and psychiatric
symptoms (hyperphagia, imulsivity and self-mutilations), one of the only drug options
consists in antipsychotics, that are not efficient and might be responsible for a worsening
of the weight gain (major issue in PWS). An alternative therapeutic approach for behavioral
disturbances has been suggested by some authors with topiramate (Epitomax®), an antiepileptic
drug that can be used as a mood stabilizer and anti-impulsive. In addition, topiramate is
used as a treatment for eating disorders because it induces loss of weight and appetite. This
last effect might be useful in the case of SPW.
Except for some clinical case reports, the investigators only found one open study for
topiramate in SPW 8 patientssuggesting promising effects. There si however no placebo
controlled study..
Objective:
To evaluate the efficacy of topiramate (200 mg / d) on Eating disorders (E), self Mutilations
(M), irritability and Impulsivity (I), metabolic status, and tolerance among of PWS patients.
Methodology:
This is a multicenter (out-patients in Toulouse, Reims, Nantes and Paris and in-patients in
Hendaye) 8 weeks double-blind placebo controlled study .
Subjects (n = 125 for 112 analyzable) all having PWS, aged 12 years-old and more should have
any of the following symptoms: E, M and U (see above). All subjects will be randomly
allocated into two groups one taking a placebo, the other taking topiramate (50mg / day
initially, increasing up to 50mg per week 200mg / day).
The population of analyzable patients in and out patient will be of equal size (n = 56). The
inclusion period is two years..
Are excluded subjects with antipsychotic or mood stabilizer medication or topiramate.
The primary endpoint will be the rate of responders, with response defined by obtaining a
score of 1 or 2 on the CGI improvement after 8 weeks of treatment
Other assessments, secondary endpoints :
- Clinic: Weight / Size / Self-injury behavior (french Echelle des Conduites Auto et
Hétéro Aggressives, ECAHA))
- Psychometric: C-SHARP and A-SHARP / Conners (Impulsivity) / Dickens (Eating behavior for
PWS)
- Organic: NFS, serum electrolytes, creatinine, ammonia plasma, serum bicarbonate, AST /
ALT / GGT, ghrelin, fasting glucose, lipid profile and insulin, leptin, TG and HbA1c.
- Side effects of topiramate: SAPS / SANS and BPRS (hallucinations), anxiety scales and
laboratory tests.