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NCT00716014 · ClinicalTrials.gov registry record · Phase 1
Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
A Phase 1 study, sponsored by Pachyonychia Congenita Project.
- Completed
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
NCT00716014 is a Phase 1 study that has completed, run by Pachyonychia Congenita Project. The registered enrollment target is 1 participants.
The verdict
NCT00716014, a Phase 1 study, has completed, sponsored by Pachyonychia Congenita Project.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
Study 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 (www.pachyonychia.org). 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.
Interventions
- DRUG TD101
- DRUG Normal saline (placebo)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2008-01 |
| Est. Completion | 2008-08 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00716014
The ClinicalTrials.gov registry entry for NCT00716014 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Pachyonychia Congenita Project, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which TD101 is the first listed.
NCT00716014 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00716014 about?
NCT00716014 is a clinical study titled "Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita". 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 symp...
What is the current status of trial NCT00716014?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2008-01. Estimated completion is 2008-08.
What interventions are being tested in trial NCT00716014?
The interventions under investigation include: TD101 (DRUG), Normal saline (placebo) (DRUG).
Who is sponsoring clinical trial NCT00716014?
This trial is sponsored by Pachyonychia Congenita Project, which has 2 total clinical trials registered on ClinicalTrials.gov.
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