CAH가 있는 성인에서 Chronocort®의 약동학 및 효능을 특성화하고 조사하기 위한 파일럿 연구
선천성 부신 비대증이 있는 성인에서 Chronocort®의 약동학 및 질병 바이오마커 반응을 특성화하고 조사하기 위한 2상 파일럿 연구
The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH). Hydrocortisone is the man-made version of the hormone cortisol, which is released in the body following a regular daily pattern. The objective of the study is to measure the levels of hydrocortisone that are absorbed into the bloodstream once Chronocort® is taken and what affects it has on other hormones in the body. Since Chronocort® is anticipated to mimic the same release pattern of cortisol in the body, it is hoped that patients with CAH will be treated more effectively to manage their disease.
연구 개요
상세 설명
This study is a Phase 2 pilot study to characterize and examine the pharmacokinetics and efficacy profile of Chronocort® in adults with congenital adrenal hyperplasia (CAH). It is designed as a two-part, single cohort, open label, multiple dose Phase 2 pilot study to: (Part A) characterize and examine the pharmacokinetics (PK) and disease bio-marker behavior following short-term dosing with Chronocort®; and to (Part B) examine the disease control after six months dose titration with Chronocort® in adults with CAH.
연구 유형
등록 (실제)
단계
- 2 단계
연락처 및 위치
연구 장소
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Maryland
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Bethesda, Maryland, 미국, 20892-1932
- National Institutes of Health Clinical Center
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참여기준
자격 기준
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건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Known CAH due to 21-hydroxylase deficiency (classic CAH) based on hormonal and genetic testing currently treated with hydrocortisone, prednisone, prednisolone or dexamethasone on a stable dosage for a minimum of 3 months.
- Male or female patients aged 18 and above.
- Provision of signed written informed consent.
- Good general health.
- Females of childbearing potential must have a negative pregnancy test initially and at all visits. Females who are engaging in sexual intercourse must be using a medically acceptable method of contraception (as defined in the protocol, section 10.5).
- Plasma renin activity must be within the clinically acceptable range at screening (less than 1.5 times upper normal range).
Exclusion Criteria:
- Co-morbid condition requiring daily administration of a medication that induces hepatic enzymes or interferes with the metabolism of glucocorticoids.
- Clinical or biochemical evidence of hepatic or renal disease. Creatinine above the normal range or elevated liver function tests (ALT or AST) > 2 times the upper limits of normal.
- Females who are pregnant or lactating.
- Women taking an estrogen-containing oral contraceptive pill and who have taken it within 6 weeks of recruitment.
- Patients taking spironolactone.
- Patients on inhaled or oral steroids apart from treatment for CAH.
- Patients with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the trial.
- Participation in another clinical trial of an investigational or licensed drug or device within the 3 months prior to inclusion in this study.
- Patients with history of bilateral adrenalectomy.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Hydrocortisone Modified Release Capsules
Chronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response |
Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
기간: 24 hours
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The maximum plasma concentration (Cmax) of chronocort |
24 hours
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Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
기간: 24 hours (at 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs)
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Area under the curve (AUC) from 0 to 24 hours (sampling occurs at the following timepoints: 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs) |
24 hours (at 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs)
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Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
기간: 24 hours
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Time to maximum plasma concentration (tmax) |
24 hours
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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The Percentage of Patients With 17-OHP and Androstenedione Levels at 0700h Within Proposed Optimal Ranges Whilst on Chronocort and Whilst on Standard Therapy (at Baseline)
기간: Specific time point (0700hrs)
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Proposed optimal ranges of 17-OHP: 300-1200ng/dl Proposed optimal ranges of androstenedione: 40-150ng.dl for males and 30-200ng/dl for females |
Specific time point (0700hrs)
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17-OHP Levels at 0700h, 1700h and 2300h
기간: Specified time points (0700h, 1700h and 2300h)
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17-OHP levels at 0700h, 1700h and 2300h |
Specified time points (0700h, 1700h and 2300h)
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Androstenedione Levels at 0700h, 1700h and 2300h
기간: Specified time points (0700h, 1700h and 2300h)
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Androstenedione levels at 0700h, 1700h and 2300h |
Specified time points (0700h, 1700h and 2300h)
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ACTH Levels at 0700h, 1700h and 2300h
기간: Specified time points (0700h, 1700h and 2300h)
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ACTH levels at 0700h, 1700h and 2300h |
Specified time points (0700h, 1700h and 2300h)
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AUC Values (Nmol*h/L) for Androstenedione
기간: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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AUC values (nmol*h/L) for Androstenedione for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h |
Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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AUC Values (Nmol*h/L) for 17-OHP
기간: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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AUC values (nmol*h/L) for 17-OHP for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h |
Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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AUC Values (Pmol*h/L) for ACTH
기간: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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AUC values (pmol*h/L) for ACTH for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h |
Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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공동 작업자 및 조사자
수사관
- 수석 연구원: Deborah P Merke, BS, MS, MD, National Institutes of Health Clinical Center (CC)
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (추정된)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- DIUR-003
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
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