Pachyonychia CongenitaPhase 1NCT00716014
Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
A Phase 1 study testing TD101 in people with Pachyonychia Congenita, run by Pachyonychia Congenita Project at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Pachyonychia Congenita
- What is being tested?
- TD101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Aug 2008
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
About this study
Official title: 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.
- Conditions
- Pachyonychia Congenita
- Being tested
- TD101, Normal saline (placebo)
- Keywords
- Pachyonychia Congenita, siRNA
- Medical subject headings
- Pachyonychia Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TD101Drug
TD101 is injection into a callus on the bottom of one of the patient's feet
Normal saline (placebo)Drug
A normal saline solution (placebo) is injected into one of the patient's feet.
| Study group | Role | What they receive |
|---|---|---|
| Foot 1 | Active comparator | An active drug injection of TD101 is injected into a callus on the bottom of one foot. Receives: Drug: TD101 |
| Foot 2 | Placebo comparator | An injection of placebo (normal saline) is injected into a callus on the bottom of one foot. Receives: Drug: Normal saline (placebo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine safety/toxicity of TD101
Measured: 18 weeks, followed by 3-month wash out period
Secondary outcomes (1)
Determine efficacy of TD101
Measured: 18 weeks, followed by 3-month wash out period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2008 (estimated)
- Study startJan 2008
- Primary completionAug 2008
- Study completionAug 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pachyonychia CongenitaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pachyonychia Congenita Project
- Sponsor type
- University, hospital or other
- Investigators
- Sancy A Leachman, MD, PhD, Principal investigator, PC Project
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00716014
- Sponsor's protocol ID
- 24013
- Status last verified
- Nov 2008
- First submitted
- 15 Jul 2008
- Last updated
- 19 Nov 2008
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Center, Placebo-Controlled, Rising Dose to Tolerance and Safety Study of TD101, an siRNA Designed for Treatment of Pachyonychia Congenita
- ClinicalTrials.gov ID
- NCT00716014
- Acronym / sponsor ID
- 24013
- Conditions
- Pachyonychia Congenita
- Keywords
- Pachyonychia Congenita, siRNA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- Pachyonychia Congenita ProjectUniversity, hospital or other
- Study start
- Jan 2008
- Primary completion
- Aug 2008 (actual)
- Study completion
- Aug 2008 (actual)
- First posted
- 16 Jul 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Foot 1 | Active comparator | Drug: TD101An active drug injection of TD101 is injected into a callus on the bottom of one foot. |
| Foot 2 | Placebo comparator | Drug: Normal saline (placebo)An injection of placebo (normal saline) is injected into a callus on the bottom of one foot. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (8 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TD101TD101 is injection into a callus on the bottom of one of the patient's feet | Drug | Foot 1 |
| Normal saline (placebo)A normal saline solution (placebo) is injected into one of the patient's feet. | Drug | Foot 2 |
Primary outcome
Determine safety/toxicity of TD101
Time frame: 18 weeks, followed by 3-month wash out period
1 secondary outcome
Determine efficacy of TD101
Time frame: 18 weeks, followed by 3-month wash out period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2008.
Related trials for Pachyonychia Congenita
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Epidermal Growth Factor Receptor Inhibition for Keratinopathies
Epidermolytic Ichthyosis, Palmoplantar Keratoderma
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A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
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RecruitingObservational study
International Pachyonychia Congenita Research Registry
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sancy A Leachman, MD, PhD principal investigator
PC Project
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TD101 in people with Pachyonychia Congenita, run by Pachyonychia Congenita Project at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Nov 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine safety/toxicity of TD101 (time frame: 18 weeks, followed by 3-month wash out period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2008.