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A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) (PATHWAY)

2026年9月10日 更新者：Portal Therapeutics, Inc.

A Phase 2b/3, Quadruple-Blinded, Randomized, Placebo-Controlled Study of PORT-77 Administered Orally to Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria and an Open-Label Extension

PATHWAY is a Phase 2b/3 clinical study with an Open-Label Extension (OLE). It tests PORT-77 in patients with EPP, EPP2, and XLP.

The study has three parts:

Part 1: Phase 2b

  • Find the right dose of PORT-77 to lower PPIX levels in the blood
  • Test if PORT-77 lowers PPIX levels better than placebo
  • Check the safety of each dose

Part 2: Phase 3

  • Test if PORT-77 lowers PPIX levels better than placebo
  • Test if PORT-77 increases the amount of time patients spend in sunlight each day without pain compared to placebo
  • Check the safety of PORT-77

Part 3: Open-Label Extension (OLE)

  • Test the long-term effect of PORT-77 on PPIX levels and time in sun
  • Monitor long-term safety

調査の概要

状態

募集

条件

介入・治療

詳細な説明

PATHWAY is a Phase 2b/3, multiregional, multicenter clinical study of PORT-77 in adults and adolescents with EPP or XLP. The study consists of 3 parts: Part 1 (Phase 2b), Part 2 (Phase 3), and Part 3 (OLE).

Part 1 and Part 2 are randomized, quadruple-blinded, placebo-controlled studies. Part 1 is a Phase 2b study evaluating the safety, tolerability, and preliminary efficacy of 2 dose levels of PORT-77. Part 2 is a Phase 3 study designed to confirm the efficacy of the selected PORT-77 dose and to further characterize safety in a larger cohort. All participants who complete Part 1 or Part 2 of the study will have the opportunity to enroll in Part 3 (OLE).

研究の種類

介入

入学 (推定)

225

段階

  • フェーズ2
  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究場所

  • アメリカ
    • California
      • Huntington Beach、California、アメリカ、92647
        • 募集
        • Investigative Site
        • コンタクト：
          • Site Contact
    • Ohio
      • Cincinnati、Ohio、アメリカ、43215
        • 募集
        • Investigative Site
        • コンタクト：
          • Site Contact

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 子
  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

Inclusion Criteria:

  • Aged 12 years or older.
  • Clinical history of EPP, XLP, or EPP2 supported by genetic confirmation or historical laboratory test results
  • History of consistent, non-painful prodrome within approximately 45 minutes of sunlight exposure and prior to phototoxic attacks
  • Demonstrates ≥85% compliance with daily symptom diary during run-in period.

  • Body weight or BMI at Screening as follows:

    1. For participants aged 12 to <18 years: body weight ≥32 kg
    2. For participants aged ≥18 years: BMI ≥18.5 kg/m2
  • AST and ALT <3 × ULN and total bilirubin <2 × ULN (unless documented Gilbert syndrome) at Screening
  • Willing and able to provide informed consent and/or assent for the study.
  • Willing and able to comply with study visits, study procedures, and contraception guidance
  • Intends to remain in the same approximate geographic latitude for the duration of the placebo-controlled period, with no more than 14 days spent outside this region.

Exclusion Criteria:

  • Diagnosis of another porphyria or another photodermatosis that may confound the evaluation of PORT-77
  • Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory assessments
  • Major surgery within 8 weeks before Screening, incomplete recovery from any previous surgery, or major surgery planned to occur during the study
  • History of, or anticipated need for, liver transplantation or history of bone marrow transplantation
  • Active infection with hepatitis B or C
  • Unable to swallow tablets or has a disease that significantly affects gastrointestinal function
  • Any other disease, condition, or circumstance that, at the discretion of the Investigator or Sponsor, would interfere with the evaluation of PORT-77 or study participation, or would make study participation not in the best interest of the participant
  • History of drug or alcohol abuse within the last 12 months, or current or planned use of prohibited or illegal substances
  • Has taken any medication, vitamin, or supplement that alters sensitivity to light exposure (eg, afamelanotide, melanotan, beta carotene, dersimelagon) within 90 days of Day 1; or has taken bitopertin within 120 days of Day 1; or has taken iron within 30 days of Day 1
  • Drugs or supplements that may impact or be impacted by PORT-77
  • Concurrent or anticipated participation in an interventional clinical trial during the study period.
  • Received another investigational therapy within 5 half-lives, if the half-life is known, or within 30 days, if the half-life is unknown, prior to Day 1.
  • Known hypersensitivity to PORT-77 or excipients
  • Female who has a positive pregnancy test at Screening or Day 1 or is breastfeeding

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：4倍

武器と介入

参加者グループ / アーム
介入・治療
実験的：Phase 2b (Treatment Group 1)
PORT-77 Dose 1
薬：PORT-77
Oral tablets
実験的：Phase 2b (Treatment Group 2)
PORT-77 Dose 2
薬：PORT-77
Oral tablets
プラセボコンパレーター：Phase 2b (Treatment Group 3)
Placebo
薬：プラセボ
経口錠剤
実験的：Phase 3 (Treatment Group 1)
PORT-77
薬：PORT-77
Oral tablets
プラセボコンパレーター：Phase 3 (Treatment Group 2)
Placebo
薬：プラセボ
経口錠剤
実験的：Phase 3 (Open-Label Extension)
PORT-77
薬：PORT-77
Oral tablets

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Plasma metal-free PPIX concentration change
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Adverse Events
時間枠：Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12
Evaluate safety and tolerability of each dose of PORT-77 over the course of the study
Part 1 - Day 1 through Day 84; Part 2 - through Day 182; Part 3 - through Month 12
Average daily time in sunlight without pain
時間枠：Part 2 - Day 155 through 182
Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Part 2 - Day 155 through 182

二次結果の測定

結果測定
メジャーの説明
時間枠
Average daily time in sunlight without pain
時間枠：Part 1 - Days 57 through 84
Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Part 1 - Days 57 through 84
Change from baseline in daily time in sunlight before first prodromal symptom
時間枠：Part 1 - Day 1 through Day 84; Part 2 - through Day 182
Assess the effect of different doses of PORT-77 on average daily time in sunlight before first prodromal symptom compared to placebo
Part 1 - Day 1 through Day 84; Part 2 - through Day 182
Cumulative total time in sunlight without pain
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Assess the effect of different doses of PORT-77 on cumulative total time in sunlight without pain compared to placebo
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Number and severity of phototoxic reactions
時間枠：Part 1 - Day 1 through 84; Part 2 - through Day 182; through Month 12
Assess the effect of different doses of PORT-77 on the rate and severity of phototoxic reactions compared to placebo
Part 1 - Day 1 through 84; Part 2 - through Day 182; through Month 12
Change from baseline in validated QoL scores - PGI-C
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12

Evaluate the effect of PORT-77 on the Patient Global Impressions - Change in clinical status (PGI-C) in adults compared to placebo.

The PGI-C measures Change in clinical status. Higher numerical scores (1-7) in PGI-C indicate worsening of clinical status.
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - PGI-S
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12

Evaluate the effect of PORT-77 on the Patient Global Impressions - Severity of clinical status.

The PGI-S measures Severity of clinical status. Higher numerical scores in PGI-S (1-5) indicate greater symptom severity.
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - SF36
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12

Evaluate the effect of PORT-77 on the Short Form 36 Health Survey (SF-36) in adults compared to placebo.

The SF-36 covers 36 individual items that evaluate functional health and well-being. It produces eight health domain scores and two summary component scores (Physical Component Summary and Mental Component Summary) where higher values (0-100) represent better health.
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Change from baseline in validated QoL scores - PedsQL
時間枠：Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12

Evaluate the effect of PORT-77 on the Pediatric Quality of Life Inventory (PedsQL) in adolescents compared to placebo.

The (PedsQL) evaluates four main domains: physical functioning, emotional functioning, social functioning, and school functioning. It uses a 0 to 100 scale where higher scores show a better health-related quality of life.
Part 1 - Days 1 through 84; Part 2 - through Day 182; Part 3 - through Month 12
Area under the curve (AUCtau) of PORT-77
時間枠：Part 1 - Day 1 through 84; Part 2 - through Day 182
Model derived area under the curve of PORT-77 during dosing interval at steady state
Part 1 - Day 1 through 84; Part 2 - through Day 182
Cmax of PORT-77
時間枠：Part 1 - Day 1 through 84; Part 2 - through Day 182
Model derived maximal concentration after dosing of PORT-77 at steady state
Part 1 - Day 1 through 84; Part 2 - through Day 182
Ctrough of PORT-77
時間枠：Part 1 - Day 1 through 84; Part 2 - through Day 182
Model derived trough concentration after dosing of PORT-77 at steady state
Part 1 - Day 1 through 84; Part 2 - through Day 182

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Portal Therapeutics, Inc.

捜査官

  • スタディディレクター：Chief Medical Officer、Portal Therapeutics, Inc.

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年9月1日

一次修了 (推定)

2029年3月1日

研究の完了 (推定)

2029年9月1日

試験登録日

最初に提出

2026年8月25日

QC基準を満たした最初の提出物

2026年9月10日

最初の投稿 (実際)

2026年9月15日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月15日

QC基準を満たした最後の更新が送信されました

2026年9月10日

最終確認日

2026年9月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • PORT-77-202
  • 2026-525555-86 (EudraCT番号)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

未定

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

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