Niemann-Pick disease type C (NPC) is a rare disease caused by a defected NPC protein. In a healthy individual, this protein helps the body process cholesterol. Those diagnosed with NPC experience symptoms in their lungs, liver, spleen, brain, and other parts of their body.
While there is an approved treatment in Europe for the condition, there remain to be no approved therapies in the U.S.
Thankfully, there are dedicated researchers trying to change that.
A New Trial
Cyclo Therapeutics is investigating Trappsol Cyclo as a treatment for NPC. This medication its administered intravenously. Currently, the therapy is in a Phase 1 trial where researchers are investigating its safety and tolerability for patients. The Co-Principal Investigators for this trial are Caroline Hastings and Benny Liu. These individuals and the rest of their team at UCSF Benioff Children’s Hospital Oakland in California worked tirelessly to bring this trial to fruition.
As evidence of their hardwork, this trial has just completed enrollment. This is a significant milestone.
12 NPC patients over the age of 18 are enrolled in this trial. All of the patients were randomized to receive 7 doses of Trappsol Cyclo. Some of the patients are being given 1500 mg/kg and some of the patients are being given 2500 mg/kg. The following are being documented:
- Adverse events
- Symptomatic changes utilizing a NPC specific severity scoring tool
- Cholesterol metabolism markers
After data is collected from the last patient (estimated date is February 2020), the results will be unblinded and analyzed.
These clinical trial results will be compared side by side with the data from the Phase I/II study which is being conducted in EU-Israel. These data will be utilized in combination to support Cyclo Therapeutics’ market registration applications.
Initial Data
The company has collected preliminary data from the Phase 1 study which shows promising results. These include an analysis of:
- It’s safety profile
- Cholesterol level in the cells
- Drug presence in cerebrospinal fluid
- Reduction of a tau, which is a neuron-specific biomarker linked to cell degeneration
You can read more about this exciting trial here.
