Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomized, double-blind, placebo-controlled study evaluating 3 dosing regimens of ATV-1601. Patients completing Part 1 may participate in the Part 2 open-label extension to receive ATV-1601.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
Part 1: This is a Phase 1/2 proof-of-concept, double-blind, multicenter, placebo-controlled study to evaluate the safety, pharmacokinetics and efficacy of 3 oral dosing regimens of ATV-1601. Participants who meet eligibility requirements will be randomized in a double-blind manner to one of 3 doses of ATV-1601 or placebo. Participants will receive double-blind study treatment for a 16-week period.
Part 2: Eligible participants who complete Part 1 may enroll in an open-label extension study to receive up to 2 years of additional treatment. All participants in the open-label extension will receive ATV-1601. Once the recommended Phase 2 dose (RP2D) is determined based on Part 1, all participants in Part 2 will have the option to switch to the RP2D.
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 2 단계
- 1단계
연락처 및 위치
연구 장소
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Arizona
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Phoenix, Arizona, 미국, 85013
- 모병
- Arizona Pulmonary Specialists
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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California
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San Diego, California, 미국, 92161
- 모병
- University of California, San Diego
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Georgia
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Augusta, Georgia, 미국, 30912
- 모병
- Augusta University Medical Center
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Indiana
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Indianapolis, Indiana, 미국, 46260
- 모병
- Innovative Hematology
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Massachusetts
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Boston, Massachusetts, 미국, 02114
- 모병
- Massachusetts General Hospital
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Minnesota
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Rochester, Minnesota, 미국, 55905
- 모병
- Mayo Clinic
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Missouri
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St Louis, Missouri, 미국, 63110
- 모병
- Washington University in St. Louis
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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North Carolina
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Chapel Hill, North Carolina, 미국, 27599
- 모병
- University of North Carolina
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Utah
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Salt Lake City, Utah, 미국, 84132
- 모병
- University of Utah Health Sciences Center
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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Buenos Aires, 아르헨티나, C1199ABB
- 모병
- Hospital Italiano de Buenos Aires
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연락하다:
- Study Director Atavistik
- 전화번호: 857-285-5400
- 이메일: studydirector@atavistikbio.com
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Ability to provide informed consent prior to any study-specific procedures
- Confirmed diagnosis of hereditary hemorrhagic telangiectasia (HHT) based on Curaçao criteria
- Moderate to severe HHT with an ESS ≥ 4
- Anemia at Screening and/or requirement for at least 1 red-cell unit (RUE) in the previous 6 months
- Adequate hematologic, renal, and hepatic function per protocol-defined laboratory criteria
- Use highly effective contraception during the study and for a protocol-defined period after last dose
Exclusion Criteria:
- Clinically significant abnormalities of glucose metabolism including diagnosed Type 1 or uncontrolled Type 2 diabetes
- Chronic cardiac disease, or cardiac rhythm abnormalities
- History of significant cardiovascular, hepatic, renal, or hematologic disease not related to HHT that may confound study results
- Use of prohibited concomitant medications within a protocol-defined washout period prior to first dose (including strong CYP modulators and certain herbal supplements)
- Recent (within 6 weeks) major surgery or local ablative procedures, or procedures on nasal telangiectasias
- Prior AKT inhibitor
- Pregnant or breastfeeding women
Additional Criteria for Open-Label Extension:
- Participants must complete the double-blind treatment period (Part 1)
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 네 배로
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
|
실험적: Part 1: 60 mg QD
Active drug, once daily |
Administered orally, daily
다른 이름들:
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실험적: Part 1: 100 mg QD
Active drug, once daily |
Administered orally, daily
다른 이름들:
|
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실험적: Part 1: 60 BID
Active drug, twice daily |
Administered orally, daily
다른 이름들:
|
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실험적: Part 1: Placebo
Control Arm |
Administered orally, daily
다른 이름들:
|
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Part 1: Safety and tolerability
기간: 16 weeks
|
Number and severity of treatment-emergent adverse events (TEAEs) and study drug-related TEAEs |
16 weeks
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Part 2: Safety and tolerability
기간: 24 months
|
Type, incidence, severity, timing, seriousness and relatedness of AEs and laboratory abnormalities |
24 months
|
2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Part 1: Change in Epistaxis duration
기간: 16 weeks
|
28-day total duration compared to baseline |
16 weeks
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Part 1: Epistaxis frequency
기간: 16 weeks
|
28-day frequency of nosebleeds compared to baseline |
16 weeks
|
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Part 1: Epistaxis intensity
기간: 16 weeks
|
28-day average epistaxis intensity (6-point scale) of nosebleeds compared to baseline |
16 weeks
|
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Part 1: Intensity-weighted epistaxis duration
기간: 16 weeks
|
28-day intensity-weighted duration of nosebleeds |
16 weeks
|
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Part 1: Epistaxis Severity Score (ESS)
기간: 16 weeks
|
The Epistaxis Severity Score (ESS) is a validated 6-question instrument with scores ranging from 0 to 10, where higher scores indicate more severe epistaxis symptoms. |
16 weeks
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Part 1: Change in Hemoglobin
기간: 16 weeks
|
Hemoglobin levels compared to baseline |
16 weeks
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Part 1: Change in Parenteral iron use
기간: 16 weeks
|
Amount of parenteral iron administered compared to 16-weeks prior to treatment initiation |
16 weeks
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Part 1: Change in Blood transfusion requirements
기간: 16 Weeks
|
Amount of packed red blood cell (PRBC) transfusions and rate of transfusion independence compared to 16-weeks prior to treatment initiation |
16 Weeks
|
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Part 1: Pharmacokinetics - Maximum observed concentration (Cmax)
기간: 16 Weeks
|
Maximum plasma concentration |
16 Weeks
|
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Part 1: Pharmacokinetics - Area under the concentration-time curve over the dosing interval (AUCtau)
기간: 16 Weeks
|
Systemic exposure of ATV-1601 over the dosing interval |
16 Weeks
|
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Part 1: Pharmacokinetics - Area under the concentration-time curve extrapolated to infinity (AUCinf)
기간: 16 Weeks
|
Total systemic exposure of ATV-1601 extrapolated to infinite time |
16 Weeks
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Part 1: Pharmacokinetics - Time to maximum concentration (Tmax)
기간: 16 Weeks
|
Time to reach maximum plasma concentration |
16 Weeks
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Part 1: Pharmacokinetics - minimum concentration (Cmin)
기간: 16 Weeks
|
Pre-dose trough plasma concentration |
16 Weeks
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Part 1: Pharmacokinetics - Half-life (t½)
기간: 16 Weeks
|
Time required for plasma concentration to decrease by half |
16 Weeks
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Part 2: Epistaxis duration
기간: Up to 2 years
|
28-day total duration every 4 weeks |
Up to 2 years
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Part 2: Epistaxis frequency
기간: Up to 2 years
|
Total number of nosebleeds every 4 weeks |
Up to 2 years
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Part 2: Epistaxis Severity Score (ESS)
기간: At 12 weeks and every 12 weeks thereafter up to study completion
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Severity of nosebleeds using a score of 0-10 automatically calculated based on responses to 6 questions. |
At 12 weeks and every 12 weeks thereafter up to study completion
|
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Part 2: Change in Hemoglobin
기간: Monthly during Part 2
|
Hemoglobin levels compared to baseline |
Monthly during Part 2
|
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Part 2: Parenteral iron use
기간: At 12 weeks and every 12 weeks thereafter up to study completion
|
Total amount of parenteral iron infused (mg) compared to baseline (12 weeks prior to treatment initiation |
At 12 weeks and every 12 weeks thereafter up to study completion
|
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Part 2: Blood transfusion requirements
기간: At 12 weeks and every 12 weeks thereafter during part 2
|
Total number of packed red blood cell (PRBC) transfusions (units) compared to baseline |
At 12 weeks and every 12 weeks thereafter during part 2
|
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Part 2: Transfusion independence
기간: At 12 weeks and every 12 weeks thereafter during part 2
|
Proportion of participants who do not require PRBC transfusions |
At 12 weeks and every 12 weeks thereafter during part 2
|
공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- ATV-1601-102
- Harmony-HHT (기타 식별자: Atavistik Bio Inc)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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