ISRCTN67285394进行中（未招募）1 期
Phase Ib, open label study to evaluate the safety, tolerability, and efficacy of a 1% topical formulation of KM-001 for the treatment of type I punctate palmoplantar keratoderma or pachyonychia congenita
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 16
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Read, understood, and signed an informed consent form (ICF) before any investigational procedure(s) are performed.
- •2. Male and female and aged 18 – 65 years (inclusive) at the time of screening.
- •3. Clinical diagnosis of:
- •Punctate palmoplantar keratoderma type I disease with confirmed heterozygous mutation in AAGAB gene.
- •PC with confirmed heterozygous mutation in either KRT16, KRT17, KRT6A, KRT6B or KRT6C mutations.
- •4. The target treatment region is 0.5% to 4% BSA including target lesion.
- •5. CGI-S score (as assessed by the CI at the screening visit) of =2.
- •6. Female patients of childbearing potential1 must use a highly effective birth control method2 (failure rate ?1% per year when used consistently and correctly) (28) throughout the trial and for at least 4 weeks after last application of IMP.
- •In addition to the hormonal contraception, female patients must agree to use a supplemental barrier method during intercourse with a male partner (i.e., male condom) throughout the trial and for at least 4 weeks after last application of IMP.
- •Female patients must be having regular menstrual periods (interval of 21 to 35 days, duration of 2 to 7 days for several months) at the baseline visit (as reported by the patient); exception: patients using hormonal contraceptives that preclude regular menstrual periods, menopausal or hysterectomised patients.
- •A male patient with a pregnant or non-pregnant female partner of childbearing potential1 must use adequate contraceptive methods (adequate contraceptive measures as required by local regulation or practice; as a minimum, the male patient must agree to use condom during treatment and until the end of relevant systemic exposure in the male patient (7 days post-treatment).
- •7. Female patients must refrain from donating eggs throughout the trial and for 4 weeks after the last IMP administration.
- •Male patients must refrain from sperm donation throughout the trial and for 7 days after the last IMP administration.
- •8. Female patients of non-childbearing potential must meet 1 of the following criteria:
- •8.1. Absence of menstrual bleeding for 1 year prior to screening without any other medical reason.
- •8.2. Documented hysterectomy or bilateral oophorectomy at least 3 months before the trial.
- •9. Patient is willing and able to comply with all the time commitments and procedural requirements of the protocol.
排除标准
- •1. History of drug or alcohol abuse in the past 2 years.
- •2. Regular alcohol consumption in males >21 units per week and females >14 units per week (1 unit = ½ pint beer, 25 mL of 40% spirit or a 125 mL glass of wine).
- •3. Positive hepatitis B surface antigen [HbsAg], hepatitis B core antibody [HbcAb], hepatitis C antibody, or human immunodeficiency virus (HIV) antibody serology results at the screening visit.
- •4. Known hypersensitivity or any suspected cross-allergy to the API and/or excipients.
- •5. Any medical or active psychological condition or any clinically relevant laboratory abnormalities, such as, but not limited, to elevated alanine aminotransferase (ALT) or aspartate aminotransferase (AST) (>3 × upper limit of normal [ULN]) in combination with elevated bilirubin (>2 × ULN), at the screening/baseline visit.
- •6. Planned or expected major surgical procedure during the clinical trial.
- •7. Patient is unwilling to refrain from using prohibited medications during the clinical trial.
- •8. Currently participating or participated in any other clinical trial of an IMP or device, within the past 4 months before the screening visit.
- •9. Cutaneous infection or another underlying condition of the skin which may impact the assessments or trial participation.
- •10. Cutaneous infection of the area to be treated with IMP within 2 weeks before the screening visit or any infection of treatment area requiring treatment with oral, parenteral antibiotics, antivirals, antiparasitics or antifungals or any topical within 2 weeks before the screening visit.
- •11. Pregnant or breastfeeding patient.
- •12. Failure to satisfy the investigator of fitness to participate for any other reason.
- •13. Having received any of the prohibited treatments within the specified timeframe before the baseline visit.
研究者
相似试验
进行中（未招募）
1 期
A Phase I/II, open label study to assess the safety and efficacy of expanded autologous bone marrow (BM) derived Mesenchymal Stromal Cells (MSC) seeded on to decellularised allogeneic patch of an airway scaffold in subjects with clinically significant Bronchopleural Fistula.EUCTR2019-004939-24-GBCell and Gene Therapy Catapult5
进行中（未招募）
1 期
Study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 in participants with myelofibrosis.Primary (PMF) or secondary (SMF) myelofibrosis.MedDRA version: 20.0Level: PTClassification code 10077161Term: Primary myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 21.1Level: LLTClassification code 10074691Term: Post polycythaemia vera myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 21.0Level: LLTClassification code 10074690Term: Post essential thrombocythemia myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)EUCTR2020-003087-45-ITGalecto Biotech AB24
招募中
1 期
Study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 in participants with myelofibrosis.EUCTR2020-003087-45-DEGalecto Biotech AB24
已完成
不适用
An open-label, Phase I study to assess safety, tolerability, radiation dosimetry, and imaging properties of 89Zr-labelled girentuximab (89Zr-girentuximab) for in vivo detection of clear cell renal carcinoma (CCRC) by positron emission tomography (PET) using different PET imaging methodologies.clear cell renal carcinomakidney cancer10038364NL-OMON44583TELIX International Pty. Ltd.10
招募中
1 期
An Open-Label Exploratory Study of ABBV-CLS-7262 in Subjects with Vanishing White Matter DiseaseCTIS2023-505704-30-00Calico Life Sciences LLC14