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A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment.

Mandel J, Bertrand V, Lehert P, Attarian S, Magy L, Micallef J, Chumakov I, Scart-Grès C, Guedj M, Cohen D - Orphanet J Rare Dis (2015)

Bottom Line: CMT1A is the most common inherited peripheral neuropathy.There is currently no approved treatment.Patients treated with AA were stable after one year but not significantly different from Placebo.

View Article: PubMed Central - PubMed

Affiliation: Pharnext SAS, Issy-les-Moulineaux, France. jmandel@pharnext.com.

ABSTRACT
CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.

No MeSH data available.


Related in: MedlinePlus

Results of the meta-analysis on the change from baseline after one year. Fixed-effect meta-analysis, with treatment as moderator variable. Difference in changes from baseline between Placebo, AA and PXT3003 were assessed through contrast tests. a Change from baseline in CMTNS under Placebo, AA and PXT3003; b Change from baseline in ONLS under Placebo, AA and PXT3003. *p < 0.05; NS = not-significant
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Fig1: Results of the meta-analysis on the change from baseline after one year. Fixed-effect meta-analysis, with treatment as moderator variable. Difference in changes from baseline between Placebo, AA and PXT3003 were assessed through contrast tests. a Change from baseline in CMTNS under Placebo, AA and PXT3003; b Change from baseline in ONLS under Placebo, AA and PXT3003. *p < 0.05; NS = not-significant

Mentions: Results obtained for CMTNS and ONLS scales were consistent (Fig. 1a and b). After one year, CMT1A patients showed a slight deterioration under Placebo of 0.16 point in CMTNS and 0.06 point in ONLS. The progression of patients under AA appeared stable (−0.04 point in CMTNS and −0.01 point in ONLS) and not significant when compared to Placebo (p = 0.390 for CMTNS and p = 0.387 for ONLS). Patients taking PXT3003 showed an amelioration in both measures (−0.68 point in CMTNS and −0.21 point in ONLS), significant when compared to Placebo (p = 0.048 for CMTNS and p = 0.044 for ONLS).Fig. 1


A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment.

Mandel J, Bertrand V, Lehert P, Attarian S, Magy L, Micallef J, Chumakov I, Scart-Grès C, Guedj M, Cohen D - Orphanet J Rare Dis (2015)

Results of the meta-analysis on the change from baseline after one year. Fixed-effect meta-analysis, with treatment as moderator variable. Difference in changes from baseline between Placebo, AA and PXT3003 were assessed through contrast tests. a Change from baseline in CMTNS under Placebo, AA and PXT3003; b Change from baseline in ONLS under Placebo, AA and PXT3003. *p < 0.05; NS = not-significant
© Copyright Policy - open-access
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4482281&req=5

Fig1: Results of the meta-analysis on the change from baseline after one year. Fixed-effect meta-analysis, with treatment as moderator variable. Difference in changes from baseline between Placebo, AA and PXT3003 were assessed through contrast tests. a Change from baseline in CMTNS under Placebo, AA and PXT3003; b Change from baseline in ONLS under Placebo, AA and PXT3003. *p < 0.05; NS = not-significant
Mentions: Results obtained for CMTNS and ONLS scales were consistent (Fig. 1a and b). After one year, CMT1A patients showed a slight deterioration under Placebo of 0.16 point in CMTNS and 0.06 point in ONLS. The progression of patients under AA appeared stable (−0.04 point in CMTNS and −0.01 point in ONLS) and not significant when compared to Placebo (p = 0.390 for CMTNS and p = 0.387 for ONLS). Patients taking PXT3003 showed an amelioration in both measures (−0.68 point in CMTNS and −0.21 point in ONLS), significant when compared to Placebo (p = 0.048 for CMTNS and p = 0.044 for ONLS).Fig. 1

Bottom Line: CMT1A is the most common inherited peripheral neuropathy.There is currently no approved treatment.Patients treated with AA were stable after one year but not significantly different from Placebo.

View Article: PubMed Central - PubMed

Affiliation: Pharnext SAS, Issy-les-Moulineaux, France. jmandel@pharnext.com.

ABSTRACT
CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.

No MeSH data available.


Related in: MedlinePlus