Clinical Trials

Multiple clinical trials evaluate (2-Hydroxypropyl)-β-cyclodextrin exclusively for Niemann-Pick disease type C1, a rare lysosomal storage disorder. Sponsored by industry sponsor Cyclo Therapeutics, Inc., these Phase I and combined Phase I/II studies include completed protocols assessing intravenous safety and pharmacokinetics alongside an extension study with unknown recruitment status. Together, these clinical investigations aim to establish the safety, pharmacokinetic, and long-term efficacy profile of the compound in affected patients.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03893071 UNKNOWN
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc.
 2019-05-23 PHASE1
NCT02912793 COMPLETED
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc.
 2017-03-20 PHASE1; PHASE2
NCT02939547 COMPLETED
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc.
 2017-10-11 PHASE1
NCT02912793 Completed
Niemann-Pick Disease Type C1
Cyclo Therapeutics Inc.
 2017-03-20 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2021-06-18)

Check the (2-Hydroxypropyl)-β-cyclodextrin (HP-β-CD) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

(2-Hydroxypropyl)-β-cyclodextrin encapsulates lipophilic molecules and binds intracellular cholesterol, facilitating its egress from lysosomal compartments and preventing amyloid-β aggregation. This cholesterol mobilization alleviates pathologically entrapped lipid accumulation within the endosomal-lysosomal system, mitigating neurovisceral damage in patients with Niemann-Pick disease type C1.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.