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A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS) (HORIZON)

2026년 10월 23일 업데이트: Biogen

A Randomized, Double-Blind, Placebo-Controlled MAD Study With Open-Label Extension to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of BIIB147 Administered Intrathecally to Adult Participants With ALS

In this study, researchers will learn more about the effects of BIIB147, in participants with amyotrophic lateral sclerosis, also known as ALS. ALS is a rare disease that damages the motor neurons in the brain and spinal cord that help control the movement of muscles in the body. It is a progressive disease, which means that it gets worse with time. Over time, this causes increasing problems with speaking, swallowing, moving the arms and legs, and breathing. In the case of ALS, this eventually leads to death from patients not being able to breathe.

This is a "first-in-human" study. This means that this study drug will be given to people for the very first time in this study. These studies are important because they help researchers learn about the safety of the study drug, how the body processes it, and what dose might be appropriate before testing it in larger groups.

The main goal of the study is to learn about the safety of BIIB147 in adult participants with ALS. The main question researchers want to answer is:

  • How many participants have adverse events and serious adverse events during the study? Adverse events are health problems that may or may not be caused by the study drug.

  • Researchers will also learn more about:

    • How much BIIB147 gets into the blood?
    • What is the highest amount of BIIB147 found in the blood?
    • How long does it take BIIB147 to reach its highest amount in the blood?
    • How much neurofilament light chain (NfL) protein is found in the blood?
    • How longer-term treatment with BIIB147 affects disease worsening and the participants' overall survival?

This study will be done as follows:

  • Participants will join the study after signing an informed consent form, also known as an ICF.
  • Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks.
  • Participants will be randomly assigned to get either the study drug BIIB147 or a placebo during the main treatment period. A placebo is something that looks like the study drug but does not contain any medicine. A placebo is also given the same as the study drug.
  • Neither the participants nor the researchers will know if participants are getting the study drug or the placebo.
  • BIIB147 or the placebo will be given through a lumbar puncture. This is done using a thin needle that is put into the lower part of the spine.
  • During the main treatment period, participants will have regular clinic visits and phone calls. At the visits, the study doctor will do tests to check the participants' overall health, including their brain and nerve function. The participants' breathing function, tongue strength, and muscle strength will also be checked. They will also measure the participants' height, weight, and vital signs and collect blood and urine samples. Participants will also complete questionnaires or answer questions about how they are feeling and about their daily lives.
  • After finishing treatment in the main part of the study, some participants who qualify may be able to join a "long-term extension" part of the study. This may last up to 6 years or more. During this part of the study, all participants will get BIIB147.
  • After the long-term extension is done, participants will join a follow-up safety period. If participants do not enter the long-term extension, they will go directly into the follow-up safety period once the treatment period is done.
  • Each participant will be in the study for up to 8 years or more.

연구 개요

상태

모병

정황

개입 / 치료

상세 설명

The primary objectives of this study are to evaluate the safety and tolerability of BIIB147 during the MAD Period and the long-term safety and tolerability of BIIB147 during the Open Label Extension (OLE) and the Integrated MAD and OLE Periods.

The secondary objectives of MAD Period are to evaluate the pharmacokinetics (PK) profile of total active BIIB147 related species in serum and cerebrospinal fluid (CSF), and to evaluate the impact of BIIB147 on neurodegeneration. The secondary objective for the OLE is to evaluate the long-term PK profile of total active BIIB147 related species in serum and CSF. The secondary objectives of the Integrated MAD Period and OLE are to evaluate the long-term impact of BIIB147 on neurodegeneration, on clinical outcomes of disease progression and survival for participants with earlier initiation of BIIB147 (i.e., start of MAD), compared with delayed initiation of BIIB147 (i.e., at start of the OLE), and long-term PK profile of total active BIIB147 related species in serum and CSF.

연구 유형

중재적

등록 (추정된)

69

단계

  • 2 단계
  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

연구 장소

  • 미국
    • Missouri
      • St Louis, Missouri, 미국, 63110
        • 모병
        • Washington University School of Medicine_St. Louis
        • 연락하다:
        • 수석 연구원:
          • Sean Smith
    • Texas
      • Austin, Texas, 미국, 78759
        • 모병
        • Austin Neuromuscular Center_Austin
        • 수석 연구원:
          • Yessar Hussain
        • 연락하다:
          • 전화번호: 512-920-0140

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Key Inclusion Criteria:

MAD Period

  • Participants must have a diagnosis of ALS consistent with the Gold Coast criteria.
  • No pathogenic variants, likely pathogenic variants, or variants of uncertain significance in the superoxide dismutase 1 (SOD1) or fused in sarcoma (FUS) genes (confirmed by central genetic testing).
  • Greater than or equal to (≥) 50% of predicted value for slow vital capacity (SVC) as adjusted for sex, age, and height (from the sitting position).
  • If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit end of study (EOS)/early termination (ET), unless the Investigator determines that it should be discontinued for medical reasons, in which case it may not be restarted during the study.

OLE

  • Participants must have completed Study 303AS101 MAD period; all pre-dose assessments for the end of MAD treatment period visit (Day 113 for Cohorts A, B, C, and Day 169 for Cohort D) must be completed prior to dosing in OLE.

Key Exclusion Criteria:

MAD Period

  • History or positive test result at screening for human immunodeficiency virus (HIV). The requirement for testing at screening may be omitted if it is not permitted by local regulations.
  • History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NfL, in the opinion of the Investigator.
  • Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
  • Requires permanent ventilation (using > 22 hours per day [h/day] invasive or non-invasive ventilation)
  • History of a deep venous thrombosis or pulmonary embolism ≤2 years of screening, or since the date of ALS diagnosis, whichever is longer.
  • Treatment with an approved ALS disease-modifying therapy other than riluzole or edaravone, or another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, before completion of Screening.
  • Current enrollment or a plan to enroll in any interventional clinical study. Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.

OLE

  • Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the ability of the participant to undergo study procedures.
  • Clinically significant abnormalities (based on most recent assessments in MAD period) in hematology, blood chemistry parameters, or 12-lead electrocardiogram (ECG) prior to first OLE visit that would make the participant unsuitable for inclusion as assessed by the Investigator.

Note: Other protocol-defined Inclusion/Exclusion criteria do apply.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 순차적 할당
  • 마스킹: 네 배로

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: MAD: Cohort A

Participants will either receive Dose A of BIIB147 or a placebo for 12 weeks.
의약품: BIIB147

Administered intrathecally (IT)

의약품: Placebo

Administered IT
실험적: MAD: Cohort B

Participants will either receive Dose B of BIIB147 or a placebo for 12 weeks.
의약품: BIIB147

Administered intrathecally (IT)

의약품: Placebo

Administered IT
실험적: MAD: Cohort C

Participants will either receive Dose C of BIIB147 or a placebo for 12 weeks.
의약품: BIIB147

Administered intrathecally (IT)

의약품: Placebo

Administered IT
실험적: MAD: Cohort D

Participants will either receive Dose D of BIIB147 or a placebo for 20 weeks.
의약품: BIIB147

Administered intrathecally (IT)

의약품: Placebo

Administered IT
실험적: OLE

Participants from Cohorts A-D who complete the MAD period will enter a 4-week blinded loading-dose period. During this period, participants randomized to BIIB147 in the MAD period will receive placebo, followed by BIIB147, while participants randomized to placebo during the MAD period will receive BIIB147. Upon completion of the blinded loading-dose period, all participants will enter the open-label maintenance dose period and receive BIIB147 for up to 340 weeks.
의약품: BIIB147

Administered intrathecally (IT)

의약품: Placebo

Administered IT

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
기간
MAD, OLE, Integrated MAD and OLE Periods: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
기간: Up to Week 416
Up to Week 416

2차 결과 측정

결과 측정
측정값 설명
기간
MAD, OLE, Integrated MAD and OLE Periods: Serum Concentrations of Total Active BIIB147 Related Species
기간: Pre-dose and at multiple time points post-dose [MAD: Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D); OLE and Integrated MAD and OLE: Up to Week 340
Pre-dose and at multiple time points post-dose [MAD: Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D); OLE and Integrated MAD and OLE: Up to Week 340
MAD, OLE, Integrated MAD and OLE Periods: Cerebrospinal Fluid (CSF) Concentrations of Total Active BIIB147 Related Species
기간: Pre-dose at multiple timepoints [MAD: Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D); OLE and Integrated MAD and OLE: Up to Week 340]
Pre-dose at multiple timepoints [MAD: Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D); OLE and Integrated MAD and OLE: Up to Week 340]
MAD Period: Area Under the Serum Concentration-Time Curve From Time Zero to Infinity (AUCinf) of BIIB147
기간: Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
MAD Period: Area Under the Serum Concentration-Time Curve From Time Zero to Time of the Last Measurable Concentration (AUC0-last) of BIIB147
기간: Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
MAD Period: Maximum Observed Serum Concentration (Cmax) of BIIB147
기간: Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
MAD Period: Time to Reach Maximum Observed Serum Concentration (Tmax) of BIIB147
기간: Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
MAD Period: Elimination Half-Life (t1/2) of BIIB147
기간: Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
Pre-dose and at multiple time points post-dose [Up to Week 16 (Cohorts A, B, and C) and Up to Week 24 (Cohort D)]
MAD, Integrated MAD and OLE Periods: Change From Baseline in Plasma Concentration of Neurofilament Light Chain (NfL)
기간: Baseline, Week 16 (Cohorts A, B, and C), Week 24 (Cohort D), and Up to Week 340 (Integrated MAD and OLE)
Baseline, Week 16 (Cohorts A, B, and C), Week 24 (Cohort D), and Up to Week 340 (Integrated MAD and OLE)
Integrated MAD and OLE Period: Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) Score
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Change From Baseline in Percent Predicted Slow Vital Capacity (SVC)
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Change From Baseline in Muscle Strength as Measured by Handheld Dynamometry (HHD)
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Change From Baseline in Bulbar Strength as Measured by Iowa Oral Pressure Instrument (IOPI)
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Change From Baseline in Rasch Overall Amyotrophic Lateral Sclerosis (ALS) Disability (ROADS).
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Change From Baseline in Weight
기간: Baseline, Up to Week 340
Baseline, Up to Week 340
Integrated MAD and OLE Period: Time to Death or Permanent Ventilation
기간: Up to Week 416

Permanent ventilation is defined as ≥ 22 hours of mechanical ventilation [invasive or noninvasive] per day for ≥ 21 consecutive days.
Up to Week 416
Integrated MAD and OLE Period: Time to Death
기간: Up to Week 416
Up to Week 416

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Biogen

수사관

  • 연구 책임자: Medical Director, Biogen

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2026년 10월 7일

기본 완료 (추정된)

2034년 1월 23일

연구 완료 (추정된)

2034년 1월 23일

연구 등록 날짜

최초 제출

2026년 9월 28일

QC 기준을 충족하는 최초 제출

2026년 9월 28일

처음 게시됨 (실제)

2026년 10월 2일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 10월 26일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 10월 23일

마지막으로 확인됨

2026년 10월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • 303AS101
  • 2026-527124-27 (기타 식별자: EU Trial (CTIS) number)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다