PORT-77 for Healthy Adults

Overseen By Portal Therapeutics, Chief Medical Officer

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Portal Therapeutics, Inc.

Disqualifiers: Pregnancy, Lactation, Psychiatric condition, others

Compensation: Varies

Number of Visits: 1

108 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment called PORT-77 to assess its effects on healthy adults. Researchers seek to understand the impact of different doses and how food might alter its effects. Participants will receive either PORT-77 or a placebo, a harmless pill resembling the treatment but inactive. The trial seeks healthy adults without significant medical issues who can adhere to the study's procedures. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects on people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, since the trial is for healthy adults with no significant medical history, it's likely that participants should not be on any regular medications.

Is there any evidence suggesting that PORT-77 is likely to be safe for humans?

Research has shown that PORT-77 is being tested for safety and tolerance in healthy adults. The FDA has given the medication special designations, suggesting it might be promising for treating conditions like EPP and XLP, though it hasn't received approval yet.

In early studies, researchers administer PORT-77 to healthy individuals to assess their tolerance. Although specific safety data from these studies is not yet available, participation in a first-in-human trial involves close monitoring for any side effects. The primary goal of these early trials is to ensure the treatment's safety for humans before evaluating its effectiveness.

For those considering joining a trial, it's important to understand that early-phase trials prioritize safety over efficacy. Researchers will closely monitor participants to identify any potential side effects or risks. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about PORT-77 because it offers a novel approach to treatment by using a new chemical formulation that acts differently from existing options. While most current treatments rely on mechanisms like targeting specific proteins or pathways, PORT-77 introduces a unique active ingredient that might provide benefits in just a single oral dose. This could potentially lead to quicker results and improved patient compliance compared to existing multi-dose therapies.

What evidence suggests that PORT-77 could be effective?

PORT-77 is a new treatment being tested in healthy adults, and little information exists about its effectiveness in people. The current study focuses on assessing its safety and understanding how the body processes it. Participants in this trial will receive either a single oral dose of PORT-77 or a matching placebo. Although direct proof of its effectiveness for any specific condition is not yet available, the research examines how well participants tolerate the treatment and how their bodies respond. The treatment's design suggests potential benefits, but more data from human studies is needed to confirm this. ^{6 7 8 9 10}

Who Is on the Research Team?

Portal Therapeutics, Chief Medical Officer

Principal Investigator

Portal Therapeutics

Are You a Good Fit for This Trial?

This trial is for healthy adults who can participate in a study to test the safety and effects of a new medication, PORT-77. Specific eligibility criteria are not provided, but typically participants must meet certain health standards.

Inclusion Criteria

Participants must be healthy based on no clinically significant medical history, physical examination, laboratory profiles, vital signs, and Electrocardiograms (ECGs), as deemed by the Principal Investigator (PI) or designee

All healthy adult participants must be willing and able to follow protocol-specified assessments

Exclusion Criteria

I am mentally capable and emotionally stable to participate in the study.

I am not pregnant or breastfeeding.

Participation in another clinical study within 60 days or within 10 half-lives (if known), prior to the first dosing, whichever is longer, or previous participation in another PORT 77 study or study part

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose

Participants receive a single oral dose of PORT-77 or placebo

1 day

1 visit (in-person)

Multiple Ascending Dose

Participants receive multiple doses of PORT-77 or placebo to assess safety and tolerability

2-4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PORT-77

Trial Overview The trial is testing PORT-77 against a placebo (a substance with no therapeutic effect). It's designed to see how people react to different doses of PORT-77 and what happens when they take it with food.

How Is the Trial Designed?

Group I: PORT-77 Experimental Treatment 1 Intervention

Healthy adult participants in each cohort will receive a single oral dose of PORT 77 or matching placebo on Day 1.

Group II: Placebo Placebo Group 1 Intervention

Healthy adult participants in each cohort will receive a single oral dose of PORT 77 or matching placebo on Day 1.

Who Is Running the Clinical Trial?

Portal Therapeutics, Inc.

Lead Sponsor

Trials

1

Recruited

110+

Published Research Related to This Trial

The web-based adverse event tracking system (eAETS) has effectively supported 175 clinical protocols over four years, capturing 2,440 adverse event reports, including minor symptoms that could indicate serious issues.

Out of the reported AEs, 1,053 did not align with the initial risk profiles, leading to corrective recommendations in 13% of the protocols, highlighting the system's role in enhancing subject safety by identifying unanticipated patterns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The impact of minor adverse event tracking on subject safety: a web-based system. Shenvi, NV., Gebhart, SS.[2009]

In a comprehensive review of 54 phase-I studies involving 1015 healthy volunteers over 10 years, the overall incidence of adverse events was found to be 12.8%, with a higher rate for active drugs (13.7%) compared to placebo (7.9%).

Most adverse events were minor, with only 3% classified as severe, and no deaths or life-threatening events reported, highlighting that while adverse events are common in phase-I trials, they are typically not serious.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in phase-I studies: a report in 1015 healthy volunteers. Sibille, M., Deigat, N., Janin, A., et al.[2019]

In Australia, the increasing requirements for reporting adverse events (AEs) and serious AEs (SAEs) in medical research are leading to unnecessary burdens on researchers, particularly affecting less funded investigator-initiated trials.

The authors argue that individual AE reports are often not useful in blinded studies, and suggest that a trial data safety and monitoring committee should handle safety data analysis, which would streamline reporting and focus on meaningful safety evaluations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Serious adverse event reporting in investigator-initiated clinical trials. Wallace, S., Myles, PS., Zeps, N., et al.[2022]

In a five-year analysis of 1,559 healthy volunteers across 142 phase I studies, the overall incidence of adverse events (AEs) was 8.8%, with most AEs being mild or moderate in severity, indicating that while AEs are common, they are generally not severe.

The incidence of AEs was significantly higher in multiple-dose studies compared to single-dose trials, highlighting the importance of study design in assessing the safety profile of active drugs, with a notable increase in AEs on the first day of drug administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in volunteers participating in phase I clinical trials: a single-center five-year survey in 1,559 subjects. Lutfullin, A., Kuhlmann, J., Wensing, G.[2019]

The study demonstrated that it is feasible and acceptable to collect self-reported symptoms, function, and quality of life (QOL) data from pediatric cancer patients aged 8 to 18 enrolled in phase 1/2 clinical trials, with a high enrollment rate of 75.9%.

The findings indicated that while most measures were reliable, the validity of the results was limited; specifically, as adverse events increased, patients reported decreased mobility and increased fatigue, suggesting that patient-reported outcomes could serve as valuable endpoints in clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new trial endpoint? Hinds, PS., Wang, J., Stern, ED., et al.[2020]

Citations

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VIR-2482 Trial: Safety & Efficacy for Influenza A Prevention Unvaccinated healthy adults were selected as the trial population in this study to better understand the tolerability profile and clinical activity of VIR-2482 ...

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A Phase 2a Study of PORT-77 in Adults With Erythropoietic ... Unable to refrain from or anticipates the use of medications/supplements known to confound PORT-77 beginning 28 days (or 5 half-lives, whichever is longer) ...

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An mRNA-based seasonal influenza vaccine in adults In this article, we report on the safety, immunogenicity, and vaccine efficacy findings on the original mRNA-1010 formulation from the P301 and ...

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PORT-77 for Healthy Adults · Recruiting Participants for Phase ... This trial is for healthy adults who can participate in a study to test the safety and effects of a new medication, PORT-77. Specific eligibility criteria are ...

7. porphyrianews.com porphyrianews.com/news/port-77-gets-2-fda-designations-potential-treatment-epp-xlp/

PORT-77 gets 2 FDA designations as treatment for EPP ... In a first-in-human Phase 1 trial (NCT06346509), PORT-77 s' safety and tolerability are being evaluated in up to 108 healthy adult volunteers.

8. businesswire.com businesswire.com/news/home/20251014131763/en/GondolaBio-Receives-U.S.-FDA-Orphan-Drug-and-Fast-Track-Designations-for-PORT-77-for-the-Treatment-of-Erythropoietic-Protoporphyria-EPP-and-X-Linked-Protoporphyria-XLP

GondolaBio Receives U.S. FDA Orphan Drug and Fast ... PORT-77 has the potential to be the first therapy in EPP/XLP that may prevent both skin and liver damage with a rapid onset of action by ...

9. trialx.com trialx.com/clinical-trials/listings/323969/randomized-double-blind-placebo-controlled-single-and-multiple-ascending-dose-study-of-port-77-administered-to-healthy-adult-participants/

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A Phase 2a study of PORT-77 in adults with erythropoietic ... Major Inclusion Criteria: * BMI ≥18.0 and ≤35.0 kg/m2 and weight ≥50 kg * Known diagnosis of EPP and is currently symptomatic even if receiving treatment ...