CAHの成人におけるChronocort®の薬物動態と有効性を特徴付け、調べるためのパイロット研究
先天性副腎過形成症の成人における Chronocort® の薬物動態および疾患バイオマーカー応答を特徴付け、調べるための第 2 相パイロット研究
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.
調査の概要
詳細な説明
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.
研究の種類
入学 (実際)
段階
- フェーズ2
連絡先と場所
研究場所
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Maryland
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Bethesda、Maryland、アメリカ、20892-1932
- National Institutes of Health Clinical Center
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参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
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.
研究計画
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：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：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
他の名前：
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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 |
|---|---|---|
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Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
時間枠：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
時間枠：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
時間枠：24 hours
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Time to maximum plasma concentration (tmax) |
24 hours
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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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)
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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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協力者と研究者
スポンサー
捜査官
- 主任研究者：Deborah P Merke, BS, MS, MD、National Institutes of Health Clinical Center (CC)
出版物と役立つリンク
研究記録日
主要日程の研究
研究開始
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (推定)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- DIUR-003
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