Ignite Creation Date:
2024-05-05 @ 7:39 PM
Last Modification Date:
2024-10-26 @ 9:52 AM
Study NCT ID:
NCT00716014
Status:
COMPLETED
Last Update Posted:
2008-11-19
First Post:
2008-07-15
Brief Title:
Study of TD101 a Small Interfering RNA siRNA Designed for Treatment of Pachyonychia Congenita
Sponsor:
Pachyonychia Congenita Project
Organization:
Pachyonychia Congenita Project
Study Overview
Official Title:
A Single-Center Placebo-Controlled Rising Dose to Tolerance and Safety Study of TD101 an siRNA Designed for Treatment of Pachyonychia Congenita
Status:
COMPLETED
Status Verified Date:
2008-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Pachyonychia congenita PC is a rare autosomal dominant keratin disorder affecting the nails skin oral mucosae larynx hair and teeth Pathogenic mutations in keratin K6a K6b K16 or K17 act via a dominant negative mechanism leading to manifestations of the disease The most disabling PC symptom is a painful plantar blistering and keratoderma that requires use of ambulation devices in more than 50 percent of patients Despite our understanding of the molecular basis of PC current treatment is limited to mechanical removal of the thick calluses non-specific topical keratolytics and oral retinoids none of which alleviates blistering or plantar pain satisfactorily A public charity PC Project has been founded to support the development of treatments for PC wwwpachyonychiaorg In collaboration with this charity a small company TransDerm Inc has developed a small interfering RNA siRNA that specifically targets a mutation in one of the PC keratins K6a As this siRNA targets a single nucleotide mutation it will only be effective against PC subjects harboring this specific mutation There are currently only six known patients who carry this mutation in the International Pachyonychia Congenita Research Registry but three of these patients live in Salt Lake City a mother and two of her children We propose to perform a Phase Ib clinical trial to test the safety and tolerability of TD101 in PC patients carrying an N171K mutation We will complete treatment of the adult patient prior to recruitment of the minors
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None