AngiokeratomasPhase 2LOTUNCT07552077
A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Angiokeratomas (LOTU)
A Phase 2 study testing PTX-022 in people with Angiokeratomas, run by Palvella Therapeutics, Inc. at 4 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety and effectiveness of the experimental drug PTX-022 in the treatment of angiokeratomas Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Angiokeratomas
- What is being tested?
- PTX-022
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 4 sitesUnited States
- Study dates
- Apr 2026to Sep 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants must be 6 years or older
- Diagnosed with angiokeratoma
- Able and willing to comply with all protocol-related activities
- Willing and able to provide written informed consent
Who cannot join
- Any significant concurrent condition that could adversely affect participation
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
- Patients deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Cleaver Medical Group
Dawsonville, Georgia, 30534
Dana Anderson, 770.800.3455 ext. 1014, danderson@cleavermedicalgroup.com
Nathan Cleaver, MD
- Not yet recruiting
Vividia Dermatology
Las Vegas, Nevada, 89148
Graham Lichtman, MD, graham.litchman@gmail.com
Graham Lichtman, MD
- Not yet recruiting
Unified Health
Sherman Oaks, California, 91403
Jamie Goodnight, (833) 525-CURA (2872), jgoodnight@curaclinicalresearch.com
Michael Lin, MD
- Recruiting
Epiphany Dermatology
Southlake, Texas, 76092
Moraima Avalos, (214) 884 - 7558, chintan@stryderesearch.com
Todd Plott, MD
About this study
The purpose of this study is to evaluate the safety and effectiveness of the experimental drug PTX-022 in the treatment of angiokeratomas
- Conditions
- Angiokeratomas
- Being tested
- PTX-022
- Keywords
- angiokeratoma
- Medical subject headings
- Angiokeratoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTX-022Drug
topical administration of PTX-022
| Study group | Role | What they receive |
|---|---|---|
| Open label | Experimental | Not described Receives: Drug: PTX-022 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
Measured: 3 months
Overall Angiokeratoma Investigator Global Assessment (AK-IGA)
Measured: 3 months
Overall Patient Global Impression of Change (PGI-C)
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2026
- Study startApr 2026 (estimated)
- Primary completionAug 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AngiokeratomasStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07552077
- Sponsor's protocol ID
- PALV-12
- Acronym
- LOTU
- Status last verified
- Apr 2026
- First submitted
- 20 Apr 2026
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Angiokeratomas (LOTU)
- ClinicalTrials.gov ID
- NCT07552077
- Acronym / sponsor ID
- LOTU / PALV-12
- Conditions
- Angiokeratomas
- Keywords
- angiokeratoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2026 (estimated)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 27 Apr 2026 (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.
The purpose of this study is to evaluate the safety and effectiveness of the experimental drug PTX-022 in the treatment of angiokeratomas
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Experimental | Drug: PTX-022 |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Participants must be 6 years or older
- Diagnosed with angiokeratoma
- Able and willing to comply with all protocol-related activities
- Willing and able to provide written informed consent
Key exclusion criteria (3 listed)
- Any significant concurrent condition that could adversely affect participation
- Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
- Patients deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities
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 |
|---|---|---|
| PTX-022topical administration of PTX-022 | Drug | Open label |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 3 months
Overall Angiokeratoma Investigator Global Assessment (AK-IGA)
Time frame: 3 months
Overall Patient Global Impression of Change (PGI-C)
Time frame: 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2026.
- Individual participant dataSharing planned: Yes
Related trials for Angiokeratomas
RecruitingObservational study
Fabry Disease Registry & Pregnancy Sub-registry
Fabry Disease
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.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTX-022 in people with Angiokeratomas, run by Palvella Therapeutics, Inc. at 4 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 4 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 27 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Emily Cook (267-738-6366, emily.cook@palvellatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: 3 months). The study lists 3 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 27 Apr 2026.