FDA Investigational New Drug Application Documents for Cyclodextrin Treatment for Niemann Pick Type C Disease

Here you will find detailed documents outlining Addi and Cassi’s cyclodextrin infusion treatments. We filed a compassionate use IND application to the US FDA in early 2009 which was approved.  The girls’ treatment is currently being monitored by the FDA.

As of mid-Sept, we are giving Addi and Cassi hydroxy propel beta cyclodextrin (HPBCD) infusions treatments two times per week on Monday’s/Thursday’s for 8 hours. Addi and Cassi are currently at a dose of 2100 mg/kg/day.

The FDA is particularly interested in tracking creatinine levels and kidney function. So far, we have not experienced any negative sides effect from cyclodextrin treatments and feel it is having a positive benefit for our twins.

Addi and Cassi’s Cyclodextrin Infusion Treatments Submitted to FDA – First Protocol Extension

Dr. Caroline Hastings Submission Letter to FDA – May 2009
Cyclodextrin Protocol Extension Letter To Dr. Ruyi He, Acting Director of FDA
Addi and Cassi Hempel FDA Cyclodextrin IND Extension
Cyclodextrin Specimen Collection Document
Addi and Cassi Hempel BioSampling Protocol

Addi and Cassi’s Compassionate Use IND Documents Submitted To FDA For Cyclodextrin Treatment

FDA Submission Documents for Addi and Cassi’s Cyclodextrin Treatment
Ruyi He FDA response letter to clinical hold – 1-17-09
FDA Type A Meeting Request Letter from Dr. Hastings
FDA Letter Releasing Clinical Hold on Cyclodextrin Treatment For Addi and Cassi Hempel
Hydroxy Propel Beta Cyclodextrin (HPBCD) Summary/Overview
Cyclodextrin Safety Study in Rats and Dogs
PNAS paper by UT Southwestern researchers on cyclodextrin

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