TGM-1 Related Autosomal Recessive Congenital IchthyosisPhase 1/2NCT04047732
Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
A Phase 1/2 study testing KB105 in people with TGM-1 Related Autosomal Recessive Congenital Ichthyosis, run by Krystal Biotech, Inc. at 2 sites in United States.
Trial summary
This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. The primary objectives of this study are to evaluate safety and Investigator Global Assessment (IGA) scale improvement of topically administered KB105. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- TGM-1 Related Autosomal Recessive Congenital Ichthyosis
- What is being tested?
- KB105
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 Aug 2019to Mar 2025
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetic diagnosis of TGM1-deficient ARCI with a null TGM-1 mutation;
- Clinical diagnosis of lamellar ichthyosis;
- Age: 18 years old or older;
- Individual site IGA score of 3 to 4 at the target areas
- Subject is, in the opinion of the Investigator, able to understand thestudy, cooperate with the study procedures, and is willing to return to the clinic for all required follow-up visits;
- Except for their moderate-to-severe ARCI, subject is in good general health; and
- Willing and able to give consent/assent.
Who cannot join
- Medical instability limiting ability to travel to the investigative center;
- Medical illness expected to complicate participation, such as an active infection with: HIV, hepatitis B (as determined by hepatitis B surface antigen screening), hepatitis C (as determined by detection of hepatitis C antibodies or a positive result of hepatitis C);
- Patient has a physical condition or other dermatological disorder (e.g., atopic, seborrheic, or contact dermatitis, psoriasis, tinea infections, etc.) which, in the Investigator's opinion, might impair evaluation of the Target Areas or which exposes the patient to unacceptable risk by study participation;
- Pregnant or breast-feeding women, or women planning to become pregnant or to breast-feed. Women of childbearing potential must have a negative urine pregnancy test at the Screening visit and Day 1 visit and must commit to using an acceptable form of contraception during the entire study period, up to three months after last KB105 administration. Women using oral contraception must also have done so for 3 months prior to Baseline or will be willing to use a combination of barrier methods. To be considered not of childbearing potential, women must be post-menopausal for at least 1 year or surgically sterile
- Known allergy to any of the constituents of the product
- Hypersensitivity to local anesthesia (e.g., lidocaine/prilocainecream)
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- Current enrollment in a clinical trial
- Treatment with an investigational drug or investigational device within 30 days prior to Day 1
- Male who is not surgically sterile nor willing to use effective forms of contraception from Day 1 until 3 months following the last dose of study drug.
- Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Northwestern University
Chicago, Illinois, 60611
Paddington Testing Company, Inc.
Philadelphia, Pennsylvania, 19103
About this study
Official title: A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. The primary objectives of this study are to evaluate safety and Investigator Global Assessment (IGA) scale improvement of topically administered KB105.
Read the detailed description
Up to six adult subjects are planned for the Phase I portion of this study. Subjects are enrolled upon obtaining consent and meeting entry criteria. This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. Patients will be evaluated for safety, and Target Areas will be assessed individually with the IGA and VIIS scales. Target Areas will be imaged and evaluated for safety and efficacy. Subjects will be on-trial for approximately 3.5 months.
- Being tested
- KB105
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB105Biological
Also called HSV1-TGM1
KB105, a replication-incompetent, non-integrating HSV-1 vector expressing human transglutaminase 1 (TGM1) formulated as a topical gel
| Study group | Role | What they receive |
|---|---|---|
| Topical KB105 | Experimental | HSV1-TGM1 vector (KB105) Receives: Biological: KB105 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
Incidence of treatment-emergent adverse events
Measured: Up to 12 weeks
Investigator's Global Assessment (IGA) of disease severity
Improvement of disease severity in the treatment area assessment through Investigator's Global Assessment (IGA)
Measured: Up to 12 weeks
Secondary outcomes (2)
Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard
Improvement of disease severity in the treatment area through use of the Visual Index for Ichthyosis Severity scale, lamellar (VIIS-L) standard assessment. The VIIS-L is a 4-point visual scale with 1 representing normal skin and 5 representing severe ichthyosis.
Measured: Up to 12 weeks
Immunofluorescence microscopy
Level of transglutaminase 1 in KB105-administered skin as measured by immunofluorescence microscopy
Measured: Up to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2019
- Study start27 Aug 2019
- Primary completionOct 2022 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TGM-1 Related Autosomal Recessive Congenital IchthyosisStudies of the same condition, open and under way.
- More trials run by Krystal Biotech, Inc.The sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04047732
- Sponsor's protocol ID
- KB105-001
- Status last verified
- Sep 2021
- First submitted
- 29 Jul 2019
- Last updated
- 13 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
- ClinicalTrials.gov ID
- NCT04047732
- Acronym / sponsor ID
- KB105-001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Aug 2019 (actual)
- Primary completion
- Oct 2022 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 7 Aug 2019 (actual)
- 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.
This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. The primary objectives of this study are to evaluate safety and Investigator Global Assessment (IGA) scale improvement of topically administered KB105.
Detailed description
Up to six adult subjects are planned for the Phase I portion of this study. Subjects are enrolled upon obtaining consent and meeting entry criteria. This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. Patients will be evaluated for safety, and Target Areas will be assessed individually with the IGA and VIIS scales. Target Areas will be imaged and evaluated for safety and efficacy. Subjects will be on-trial for approximately 3.5 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Topical KB105 | Experimental | Biological: KB105HSV1-TGM1 vector (KB105) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Genetic diagnosis of TGM1-deficient ARCI with a null TGM-1 mutation;
- Clinical diagnosis of lamellar ichthyosis;
- Age: 18 years old or older;
- Individual site IGA score of 3 to 4 at the target areas
- Subject is, in the opinion of the Investigator, able to understand thestudy, cooperate with the study procedures, and is willing to return to the clinic for all required follow-up visits;
Key exclusion criteria (10 listed)
- Medical instability limiting ability to travel to the investigative center;
- Medical illness expected to complicate participation, such as an active infection with: HIV, hepatitis B (as determined by hepatitis B surface antigen screening), hepatitis C (as determined by detection of hepatitis C antibodies or a positive result of hepatitis C);
- Patient has a physical condition or other dermatological disorder (e.g., atopic, seborrheic, or contact dermatitis, psoriasis, tinea infections, etc.) which, in the Investigator's opinion, might impair evaluation of the Target Areas or which exposes the patient to unacceptable risk by study participation;
- Pregnant or breast-feeding women, or women planning to become pregnant or to breast-feed. Women of childbearing potential must have a negative urine pregnancy test at the Screening visit and Day 1 visit and must commit to using an acceptable form of contraception during the entire study period, up to three months after last KB105 administration. Women using oral contraception must also have done so for 3 months prior to Baseline or will be willing to use a combination of barrier methods. To be considered not of childbearing potential, women must be post-menopausal for at least 1 year or surgically sterile
- Known allergy to any of the constituents of the product
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 |
|---|---|---|
| KB105Also: HSV1-TGM1KB105, a replication-incompetent, non-integrating HSV-1 vector expressing human transglutaminase 1 (TGM1) formulated as a topical gel | Biological | Topical KB105 |
Primary outcome
Safety and tolerability
Time frame: Up to 12 weeks
Incidence of treatment-emergent adverse events
Investigator's Global Assessment (IGA) of disease severity
Time frame: Up to 12 weeks
Improvement of disease severity in the treatment area assessment through Investigator's Global Assessment (IGA)
2 secondary outcomes
Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard
Time frame: Up to 12 weeks
Immunofluorescence microscopy
Time frame: Up to 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2021.
- Individual participant dataSharing planned: No
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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing KB105 in people with TGM-1 Related Autosomal Recessive Congenital Ichthyosis, run by Krystal Biotech, Inc. at 2 sites 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 10 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: status not confirmed, as of the sponsor's last update on 13 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Safety and tolerability (time frame: Up to 12 weeks). The study lists 2 primary outcomes in all. 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 13 Sep 2021.