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NCT02094222
Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease
trial testing RAVICTI in Byler Disease. No longer available.
Quick facts
| Lead sponsor | Robert Squires, Jr. |
|---|---|
| Status | NO LONGER AVAILABLE |
| Study type | EXPANDED_ACCESS |
Drugs / interventions tested
- RAVICTI (GLYCEROL PHENYLBUTYRATE) — full drug profile →
Conditions studied
- Byler Disease — all drugs for Byler Disease →
Sponsor
Robert Squires, Jr.
Who can join
Adults 130 Days to 21, any sex, with Byler Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Byler Disease is the result of a homozygous missense (G308V) mutation in the ATP8B1 gene. The disease is typically manifest in the first year of life on the basis of complications of cholestasis; common presentations include jaundice, poor growth, bleeding related to vitamin K deficiency, and/or weak bones related to vitamin D deficiency. Early management of Byler Disease is directed at nutritional issues which tend to be responsive to medical intervention, unlike the pruritus/scratching which remains a devastating problem. Progressive liver disease develops in Byler Disease and can lead to cirrhosis and end-stage liver disease. This is an open label expanded access protocol of RAVICTI in children with Byler Disease. The primary hypothesis is that the administration of RAVICTI in these children is feasible, well tolerated and safe. It is also hypothesized that RAVICTI treatment leads to an improvement in biochemical markers of liver disease and it may ameliorates or prevents the development of scratching behavior as a manifestation of pruritus attributed to the liver disease.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Compassionate use of drugs and medical devices in the United States, the European Union and Japan.
Tsuyuki K, Yano K, Watanabe N, Aruga A, et al · · 2016 · cited 13× · PMID 31245484 · DOI 10.1016/j.reth.2015.11.002 -
Latent disease similarities and therapeutic repurposing possibilities uncovered by multi-modal generative topic modeling of human diseases.
Kozawa S, Yokoyama H, Urayama K, Tejima K, et al · · 2023 · cited 1× · PMID 37123453 · DOI 10.1093/bioadv/vbad047
Verify or expand the search:
- PubMed search for NCT02094222
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of RAVICTI
Trials testing the same drug.
- NCT03335488 — Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCD · Phase 4 · completed
- NCT02246218 — A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of A · Phase 4 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT02094222 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Robert Squires, Jr.
- Last refreshed: 18 February 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02094222.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing