QTORIN Rapamycin Gel for Pachyonychia Congenita

Overseen By Emily Cook

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Palvella Therapeutics, Inc.

Must not be taking: Sirolimus

Disqualifiers: Pregnancy, Breastfeeding, Significant conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

45 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the long-term safety and effectiveness of a gel called PTX-022 for adults with Pachyonychia Congenita (PC), a condition affecting nails and skin, particularly in those with specific gene mutations. Participants must have completed a previous related study, PALV-05, to qualify. The primary goal is to assess how the body absorbs and reacts to PTX-022. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

You will need to stop taking any medications that contain sirolimus, as the trial excludes participants using these drugs.

Is there any evidence suggesting that PTX-022 is likely to be safe for humans?

Research has shown that PTX-022, a topical gel, is generally safe and well-tolerated. In studies involving adults with Pachyonychia Congenita and conditions like Microcystic Lymphatic Malformations and Cutaneous Venous Malformations, researchers have closely monitored its safety.

Most participants experienced mild to moderate side effects, typically at the application site, such as redness or skin irritation. Serious side effects have been rare, indicating that PTX-022 is relatively safe for long-term skin use.

These studies are in advanced stages, providing stronger evidence of safety. Additionally, the active ingredient in PTX-022, rapamycin, has already received FDA approval for other uses, further supporting its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for pachyonychia congenita, which often involve managing symptoms with pain relievers and topical creams, QTORIN Rapamycin 3.9% Anhydrous Gel offers a direct approach by delivering rapamycin, an active ingredient, directly to the affected area. Rapamycin is known for its ability to inhibit a key pathway involved in cell growth and inflammation, potentially addressing the root cause of the condition rather than just alleviating symptoms. Researchers are excited about this gel because it promises a more targeted treatment with potentially fewer side effects compared to systemic therapies.

What evidence suggests that PTX-022 might be an effective treatment for Pachyonychia Congenita?

Research has shown that PTX-022, a gel applied to the skin containing rapamycin, may help treat Pachyonychia Congenita (PC). In earlier studies, patients using PTX-022 experienced significant symptom improvements. Reports from doctors and patients, along with photos, demonstrated clear progress. Although researchers continue to study this treatment, these findings suggest PTX-022 could effectively manage PC. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

Inclusion Criteria

Completed the PALV-05 (VAPAUS) study

Agree to contraceptive use

Exclusion Criteria

Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study

Females who are pregnant or breastfeeding

Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive QTORIN rapamycin 3.9% anhydrous gel to evaluate safety and pharmacokinetics

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PTX-022

How Is the Trial Designed?

Group I: PTX-022 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Palvella Therapeutics, Inc.

Lead Sponsor

Trials

8

Recruited

380+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03920228

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX- ... Phase 2/​3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita. ClinicalTrials.gov ID NCT03920228. Sponsor ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05050149

Study Evaluating the Safety and Efficacy of PTX-022 ... This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations.

3. trialx.com trialx.com/clinical-trials/listings/300704/a-phase-2-baseline-controlled-study-evaluating-the-safety-and-efficacy-of-ptx-022-sirolimus-topical-gel-39-in-the-treatment-of-cutaneous-venous-malformations/

A Phase 2, Baseline-Controlled Study Evaluating the ... Overview. A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX022 (sirolimus) Topical Gel 3.9% in the Treatment of ...

4. stanfordhealthcare.org stanfordhealthcare.org/trials/s/NCT05050149.html

Study Evaluating the Safety and Efficacy of PTX-022 ... Purpose. This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations.

5. journals.lww.com journals.lww.com/jova/fulltext/2024/12000/phase_2_study_of_the_safety_and_efficacy_of_qtorin.8.aspx

Phase 2 study of the safety and efficacy of QTORIN... Efficacy indicated statistically significant improvements in endpoints with treatment. Clinician and patient reports were supported by visual ...

6. mayo.edu mayo.edu/research/clinical-trials/cls-20582792

Clinical Trials The purpose of this study is to evaluate the safety and efficacy of PTX022 (sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations.

7. ctv.veeva.com ctv.veeva.com/study/phase-2-3-study-evaluating-the-safety-and-efficacy-of-ptx-022-in-treatment-of-adults-with-pachyonych

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX ... This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia ...

8. ctv.veeva.com ctv.veeva.com/study/valo-2-study-evaluating-the-safety-and-efficacy-of-ptx022-in-the-treatment-of-adults-with-pachyonyc

VALO-2: Study Evaluating the Safety and Efficacy of PTX022 ... The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study ...