EUCTR2022-002954-25-DK进行中（未招募）1 期
ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children with Achondroplasia followed by an Open Label Extension period - ApproaCH
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Participants are eligible to be included in the trial only if all of the following criteria apply:
- •1. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
- •2. Male or female, between 2 and 11 years of age (inclusive) at the time of Screening.
- •3. Clinical diagnosis of ACH with documented genetic confirmation available.
- •4. Able to stand without assistance.
- •5. Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and to follow the protocol.
- •6. At least six months of growth and disease history from ACHieve (TCC-NHS-01) trial or comparable growth and disease history available from medical records (pending confirmation by Medical Monitor).
- •7. Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG and clinical laboratory tests performed during the Screening period.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 80
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Participants are excluded from the trial if any of the following criteria apply:
- •1. Participation (i.e., signed informed consent) in any interventional clinical trial before within 3 months prior to screening.
- •2. Closed epiphysis.
- •3. Known or suspected hypersensitivity to the IMP or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and mPEG).
- •4. Have a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, pre-diabetes, or diabetes mellitus.
- •5. Have received any dose of prescription medications and IMP or surgical intervention intended to affect stature, growth, or body proportionality at any time.
- •6. Requires, or anticipated to require, chronic (> 4 weeks) or repeated treatment (more than twice/year and >3 weeks/year) with systemic corticosteroids during participation in the trial. Chronic use of high-dose inhaled corticosteroids is not allowed.
- •7. Known history of presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
- •8. Known history of any bone-related surgery affecting growth potential of long bones, such as:
- •Orthopedic reconstructive surgery for bone lengthening (e.g., procedures for leg bowing such as 8-plate are not exclusionary).
- •Cervicomedullary decompression surgery without anticipated need for repeat decompression during the time of the trial are allowed with minimum of 6 months of bone healing.
- •Ventriculoperitoneal (VP) shunt and laminectomy with full recovery are allowed with minimum of 6 months of bone healing.
- •Bone fracture within 6 months prior to screening (within 2 months for fracture of digits and buckle fractures).
- •9. Clinically significant findings at Screening, such as:
- •Expected to require surgical intervention during participation in the trial. Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement, are permitted.
- •Severe untreated sleep apnea or newly initiated sleep apnea treatment (e.g., Continuous Positive Airway Pressure [CPAP] in the previous 2 months prior to Screening.
- •Musculoskeletal disease, such as Salter-Harris fractures or clinical and/or radiographic evidence of severe hip pathology, or
- •Otherwise, are considered by the Investigator and Medical Monitor to make a participant unfit to receive trial treatment or undergo trial related procedures.
- •10. Have evidence at Screening that are consistent with severe cervicomedullary junction compression based on clinical and/or radiologic findings that indicate immediate surgical intervention is required.
- •11. Have a clinically significant finding or arrhythmia as determined by the investigator in consultation with the medical monitor that indicates abnormal cardiac function or conduction that includes, but is not exclusive to:
- •Repaired or unrepaired coarctation.
- •Moderate or greater complexity congenital heart disease including tetralogy of Fallot, Atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease.
- •12. QTcF = 450 msec at the Screening Visit.
- •13. Known history or presence of condition that impacts hemodynamic stability (s
研究者
相似试验
招募中
1 期
A phase 2b clinical trial to evaluate efficacy and safety of weekly doses of TransCon CNP compared with placebo in participants with achondroplasia aged 2 to 11 years of ageAchondroplasia (ACH) in prepubertal childrenMedDRA version: 25.0Level: LLTClassification code 10000452Term: AchondroplasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2022-002954-25-IEAscendis Pharma Growth Disorders A/S80
进行中（未招募）
1 期
A Clinical Study to Investigate the Efficacy and Safety of Different Combination Regimens Including JNJ-73763989 and/or JNJ-56136379 for the Treatment of Chronic Hepatitis B Virus InfectioChronic Hepatitis B Virus InfectionMedDRA version: 20.1Level: PTClassification code 10008910Term: Chronic hepatitis BSystem Organ Class: 10021881 - Infections and infestationsEUCTR2019-000622-22-ESJanssen Sciences Ireland UC450
进行中（未招募）
1 期
A Clinical Study to Investigate the Efficacy and Safety of Different Combination Regimens Including JNJ-73763989 and/or JNJ-56136379 for the Treatment of Chronic Hepatitis B Virus InfectioEUCTR2019-000622-22-DEJanssen Sciences Ireland UC450
进行中（未招募）
1 期
A Clinical Study to Investigate the Efficacy and Safety of Different Combination Regimens Including JNJ-73763989 and/or JNJ-56136379 for the Treatment of Chronic Hepatitis B Virus InfectioChronic Hepatitis B Virus InfectionMedDRA version: 20.1Level: PTClassification code 10008910Term: Chronic hepatitis BSystem Organ Class: 10021881 - Infections and infestationsEUCTR2019-000622-22-GBJanssen Sciences Ireland UC470
进行中（未招募）
1 期
A Clinical Study to Investigate the Efficacy and Safety of Different Combination Regimens Including JNJ-73763989 and/or JNJ-56136379 for the Treatment of Chronic Hepatitis B Virus InfectioChronic Hepatitis B Virus InfectionMedDRA version: 20.1Level: PTClassification code 10008910Term: Chronic hepatitis BSystem Organ Class: 10021881 - Infections and infestationsEUCTR2019-000622-22-ITJanssen Sciences Ireland Unlimited Company450