基本情報/Basic information |
一般向け試験名/Public title |
小児を含む、先天性胆汁酸代謝異常症患者を対象としたRM1319の第Ⅲ相試験及び製造販売後臨床試験
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Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn error of bile acid metabolism, including children |
一般向け試験名略称/Acronym |
先天性胆汁酸代謝異常症患者を対象とした臨床試験 |
Clinical study for patients with inborn errors of bile acid metabolism |
科学的試験名/Scientific Title |
小児を含む、先天性胆汁酸代謝異常症患者を対象としたRM1319の第Ⅲ相試験及び製造販売後臨床試験
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Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn errors of bile acid metabolism, including children |
科学的試験名略称/Scientific Title:Acronym |
小児を含む、先天性胆汁酸代謝異常症患者を対象としたRM1319の第Ⅲ相試験及び製造販売後臨床試験
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Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn errors of bile acid metabolism, including children |
試験実施地域/Region |
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評価/Assessment |
主要アウトカム評価項目/Primary outcomes |
投与26週後(中間評価時)における各病型に特徴的にみられる尿(随時尿)中の異常代謝産物の量(濃度) |
Amount (concentration) of abnormal metabolites in urine (spot urine) which are characteristic of each disease type at 26 weeks after the first administration of RM1319 (interim evaluation) |
副次アウトカム評価項目/Key secondary outcomes |
1. 治験薬評価期及び継続投与期の各Visitにおける各病型に特徴的にみられる尿中及び血清中異常代謝産物の量(濃度)
2. 治験薬評価期及び継続投与期の各Visitにおける臨床検査値(血清):
AST、ALT、D-Bil、PT、ビタミンD
3. 投与開始時(Visit1)、投与26週後、継続投与期の6ヵ月ごと、終了時(中止又は中断時)の肝臓の超音波画像所見及び肝硬度(フィブロスキャン検査等のエラストグラフィ)
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1. Amount (concentration) of abnormal metabolites in urine and serum that are characteristic of each disease type at each visit during the investigational drug evaluation period and continuous administration period
2. Clinical test values (serum) at each visit during the investigational drug evaluation period and continuous administration period:
AST, ALT, D-Bil, PT, Vitamin D
3. Ultrasonic image findings and liver stiffness (Elastrography examination such as FibroScan) at base line of administration (Visit1), 26 weeks after the first administration, every 6 months during the continuous administration period at the time of completion (including withdrawal or interruption). |
介入/Intervention |
群数/No. of arms |
1 |
介入の目的/Purpose of intervention |
治療・ケア/Treatment |
介入の種類/Type of intervention |
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介入1/Interventions/Control_1 |
RM1319の継続投与 |
Continuous administration of RM1319 |
介入2/Interventions/Control_2 |
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介入3/Interventions/Control_3 |
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介入4/Interventions/Control_4 |
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介入5/Interventions/Control_5 |
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介入6/Interventions/Control_6 |
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介入7/Interventions/Control_7 |
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介入8/Interventions/Control_8 |
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介入9/Interventions/Control_9 |
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介入10/Interventions/Control_10 |
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適格性/Eligibility |
年齢(下限)/Age-lower limit |
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年齢(上限)/Age-upper limit |
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性別/Gender |
男女両方/Male and Female |
選択基準/Key inclusion criteria |
以下1から7のすべての条件を満たす患者
1. 1)又は2)に該当する患者
1) 先天性胆汁酸代謝異常症
2) 先天性胆汁酸代謝異常症の急性期症状がみられ、胆汁酸分析等の特殊生化学検査により先天性胆汁酸代謝異常症が強く疑われる患者
2. 上記1) に該当し、現在、一次胆汁酸(ケノデオキシコール酸)による治療を受けている患者の場合は、本治験の登録時までの12週間以上、用法・用量が概ね一定となっている患者
3. 同意取得時年齢が75歳未満の患者(男性及び女性)
4. 治験で計画されている全ての観察項目の実施に同意した患者
5. 治験責任医師又は分担医師の指示に従うことに同意した患者
6. 同意取得時年齢が20歳以上の患者については、治験の目的及び研究内容について理解し、副作用についても理解し、治験に参加することについて本人又は代諾者、必要に応じて公正な立会人による文書同意が得られている患者
7. 国内在住の日本人患者
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Patients who meet all of the following criteria
1. Patients who meet 1) or 2)
1) Inborn errors of bile acid metabolism
2) Patients with acute symptoms of inborn errors of bile acid metabolism and strongly suspected inborn errors of bile acid metabolism by special biochemical tests such as bile acid analysis
2. For patients with above 1), if the patients are currently undergoing treatment with primary bile acid (chenodeoxycholic acid), the dosage and administration should be generally constant for at least 12 weeks until the registration of this study
3. Patients not older than 75 years old at the time of informed consent (male and female)
4. Patients who have consented to carry out all the observation items planned in the clinical trial
5. Patients who have consented to follow the instructions of the investigator or sub-investigator
6. For patients aged 20 years or older at the time of informed consent, patients who understand the purpose, content of this study and side effects, and agree to participate in this study and have signed the consent form by themselves or by a legal representative, if necessary, with a fair attendance.
7. Japanese patients living in Japan
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除外基準/Key exclusion criteria |
以下の基準のいずれかに該当する患者は、本治験から除外する。
1. 過去にコール酸及び本治験薬に含まれる成分を投与して過敏症又は重篤な副作用を発現したことがあり、治験責任医師又は分担医師が治験参加不適切と判断する患者
2. ガラクトース不耐症、ラップラクターゼ欠損症、又はグルコース・ガラクトース吸収不全症の疾患を有する患者
3. 高トリグリセリド血症又は家族歴を有する患者で、ケノデオキシコール酸の高用量を投与しているにも関わらず効果が確認出来ず、有効な用量が確認出来ずにいる患者
4. 同意取得前12週間以内に他の臨床研究又は臨床試験に参加した患者
5. アルコール依存症又は薬物依存症のある患者
6. 妊娠中又は授乳中の女性。妊娠可能な患者においては、治験期間中に妊娠を希望する患者又は適切な避妊(子宮内避妊器具・殺精子剤、パートナーによるコンドーム装着等)に同意いただけない患者(治験登録時及び治験期間中の定められた時点において尿中HCG検査による確認を必須とする)
7. その他、治験責任医師又は分担医師が本治験への参加を不適切と判断した患者
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Patients with any of the following criteria will be excluded from this study.
1. Among patients who have experienced hypersensitivity or serious side effects after administration of cholic acid or components contained in this investigational drug, patients who are determined to be unsuitable by the investigator or sub-investigator
2. Patients with galactose intolerance, Lapp lactase deficiency, or glucose/galactose malabsorption
3. Among patients with hypertriglyceridemia or family history, patients who are unable to confirm the effective dose even though they are administering a high dose of chenodeoxycholic acid
4. Patients who have participated in other clinical studies or clinical trials within the past 12 weeks
5. Patients with alcohol dependence or drug dependence
6. Pregnant or breast-feeding women. Among women of childbearing potential, who wish to become pregnant during this study or who cannot consent appropriate contraception (intrauterine contraceptive device/spermicide, wearing a condom by a partner, etc.)
Check by urinary HCG test is mandatory at registration and specified timing during this study)
7. Other patients who are determined to be unsuitable by the investigator or sub-investigator |
目標参加者数/Target sample size |
5 |