- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00716014
Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
November 18, 2008 updated by: Pachyonychia Congenita Project
A Single-Center, Placebo-Controlled, Rising Dose to Tolerance and Safety Study of TD101, an 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 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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
1
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Utah
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Salt Lake City, Utah, United States, 84112
- Huntsman Cancer Institute
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
8 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- A positive genetic identification of the N171K mutation in the keratin 6a gene from a CLIA certified laboratory;
- Witnessed, signed informed consent approved by Institutional Review Board/Ethics Committee;
- A signed Health Information Portability and Accountability Act (HIPAA) authorization form which permits the use and disclosure of patient's individually identifiable health information for those enrolled in the United States of America;
- Male or female subjects of any race 10 years of age and older;
- Complete physical examination and medical history indicating no abnormalities that will interfere with study objectives;
- Normal or not clinically significant baseline laboratory tests, including hemogram, ANA, serum chemistry panel, urinalysis, C3a, Bb, and APTT,PT;
- Negative pregnancy test (females only).
Exclusion Criteria:
- Females of childbearing potential not using a highly effective method of birth control (e.g. implants, injectables, combined oral contraceptives, some intrauterine contraceptive devices) during the study;
- Diabetes mellitus requiring treatment other than diet and exercise;
- Treatment of any type for cancer within the last six months;
- History of any significant internal disease;
- Subjects who are known to be allergic to any of the test product(s) or any components in the test product(s) or history of hypersensitivity or allergic reactions to any of the study preparations;
- History of street drug or alcohol abuse;
- Any patient not able to meet the study attendance requirements;
- Subjects who have participated in any other trial of an investigational drug or device within 60 days prior to enrollment or participation in a research study concurrent with this study.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Active Comparator: Foot 1
An active drug injection of TD101 is injected into a callus on the bottom of one foot.
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TD101 is injection into a callus on the bottom of one of the patient's feet
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Placebo Comparator: Foot 2
An injection of placebo (normal saline) is injected into a callus on the bottom of one foot.
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A normal saline solution (placebo) is injected into one of the patient's feet.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Determine safety/toxicity of TD101
Time Frame: 18 weeks, followed by 3-month wash out period
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18 weeks, followed by 3-month wash out period
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Determine efficacy of TD101
Time Frame: 18 weeks, followed by 3-month wash out period
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18 weeks, followed by 3-month wash out period
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Sancy A Leachman, MD, PhD, PC Project
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2008
Primary Completion (Actual)
August 1, 2008
Study Completion (Actual)
August 1, 2008
Study Registration Dates
First Submitted
July 15, 2008
First Submitted That Met QC Criteria
July 15, 2008
First Posted (Estimate)
July 16, 2008
Study Record Updates
Last Update Posted (Estimate)
November 19, 2008
Last Update Submitted That Met QC Criteria
November 18, 2008
Last Verified
November 1, 2008
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 24013
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Pachyonychia Congenita
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Palvella Therapeutics, Inc.RecruitingPachyonychia CongenitaUnited States, United Kingdom
-
Pachyonychia Congenita ProjectRecruiting
-
Kamari Pharma LtdRecruiting
-
Palvella Therapeutics, Inc.RecruitingPachyonychia CongenitaUnited States
-
Palvella Therapeutics, Inc.Active, not recruitingPachyonychia CongenitaUnited States
-
TransDerm, Inc.Pachyonychia Congenita ProjectCompletedPachyonychia CongenitaUnited States
-
Palvella Therapeutics, Inc.UnknownPachyonychia CongenitaUnited States
-
Tel-Aviv Sourasky Medical CenterUnknownPachyonychia Congenita
-
Johns Hopkins UniversityCompletedEpidermolysis Bullosa Simplex | Pachyonychia CongenitaUnited States
-
Uppsala UniversityUnknownEpidermolysis Bullosa SimplexSweden