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Hydroxypropyl-beta-cyclodextrin
Hydroxypropyl-beta-cyclodextrin is a solubilizing excipient that forms inclusion complexes with lipophilic drugs to improve their bioavailability and enable intravenous delivery.
Hydroxypropyl-beta-cyclodextrin is a solubilizing excipient that forms inclusion complexes with lipophilic drugs to improve their bioavailability and enable intravenous delivery. Used for Niemann-Pick disease type C (NPC).
At a glance
| Generic name | Hydroxypropyl-beta-cyclodextrin |
|---|---|
| Also known as | Hydroxypropyl-beta-cyclodextrin (HP-β-CD), Trappsol Cyclo |
| Sponsor | Cyclo Therapeutics, Inc. |
| Drug class | Cyclodextrin derivative / Excipient |
| Modality | Small molecule |
| Therapeutic area | Rare Genetic Disorders / Lysosomal Storage Diseases |
| Phase | Phase 3 |
Mechanism of action
Cyclodextrins are cyclic oligosaccharides with a hydrophobic cavity that can encapsulate poorly water-soluble compounds, increasing their solubility and stability. Hydroxypropyl-beta-cyclodextrin (HPβCD) is used as a pharmaceutical excipient to facilitate the formulation and delivery of drugs that would otherwise have limited aqueous solubility. In the context of Cyclo Therapeutics' development, HPβCD is being investigated as a therapeutic agent itself for certain lysosomal storage disorders where it may help mobilize and clear accumulated lipids.
Approved indications
- Niemann-Pick disease type C (NPC)
Common side effects
- Infusion-related reactions
- Headache
- Nausea
- Diarrhea
Key clinical trials
- Evaluating Safety and Immune Response of Janssen, Moderna, Pfizer/BNT, and Novavax COVID-19 Vaccines for Same and Mixed Boosters in Adolescents and Adults Aged 12-64 With and Without HIV in Kenya, DRC, and Rwanda (PHASE2)
- Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C (PHASE1, PHASE2)
- The Androtriol Injection for the Treatment of Acute Ischemic Stroke (PHASE2)
- A First in Human Study of the Safety, Tolerability and Pharmacokinetics of PRV-002 in Healthy Volunteers (PHASE1)
- Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (PHASE3)
- Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System (PHASE3)
- Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 (PHASE2, PHASE3)
- Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Hydroxypropyl-beta-cyclodextrin CI brief — competitive landscape report
- Hydroxypropyl-beta-cyclodextrin updates RSS · CI watch RSS
- Cyclo Therapeutics, Inc. portfolio CI