EUCTR2020-001189-13-DK进行中（未招募）1 期
A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 63
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
- •Age and Sex
- •1. Main cohort: Aged =2 years to <11 years (up to the day before 11th birthday inclusive) at time of enrollment; or exploratory cohort: aged =3 months to <2 years (up to the day before 2nd birthday inclusive) at time of enrollment
- •Type of Participant and Disease Characteristics
- •2. Documented, confirmed genetic diagnosis of achondroplasia from historical medical records prior to entry into this trial (test must have been performed at a laboratory fully accredited for genetic testing under local regulations).
- •3. Completed the C4181001 natural history study with at least 2 valid height/length measurements (at least 3 months apart) prior to enrollment in this study. One of these measurement timepoints must be within the 3 months prior to enrollment in C4181005.
- •4. Tanner stage 1 based on investigator assessment during physical examination (must include assessment of breast development for females, testicular stage for males).
- •5. Able to stand independently for height measurements (if >2 years of age at enrollment).
- •6. If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.
- •Informed Consent:
- •7. Capable of giving signed informed consent/assent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.
- •8. Following receipt of oral and written information about the trial, the child (depending on local institutional review board/independent ethics committee requirements) must provide assent, and one or both (according to local regulations) parents or guardians of the child must provide signed informed consent before any trial-related activity is carried out.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 63
- •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
- •Medical Conditions:
- •1. Presence of co-morbid conditions or circumstances that, in the opinion of the investigator, would affect interpretation of growth data or ability to complete the trial procedures.
- •2. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
- •3. Presence of severe obesity (BMI >95th percentile on Hoover-Fong BMI charts)
- •4. Known closure of long bone growth plates (cessation of height growth).
- •5. Body weight <7 kg or >30 kg.
- •6. Severe renal impairment CrCL GFR <60 mL/min/1.73m2 (Calculated GFR based on updated bedside Schwartz formula for pediatric patients (CrCL (mL/min/1.73 m2) = 0.413 * Height (cms)/ Serum cr (mg/dL) or hepatic impairment (AST/ALT >1.5 ULN)
- •7. History of hypersensitivity to study intervention or any excipients.
- •Prior/Concomitant Therapy:
- •8. History of any prior treatment with human growth hormone or related products (including insulin-like growth factor 1 [IGF-1]).
- •9. History of receipt of any treatment that are known to potentially affect growth (including oral steroids >5 days in the last 6 months, high dose inhaled corticosteroids (>800 mcg/day beclametasone equivalent) and medication for attention deficit hyperactivity disorder).
- •10. History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
- •11. Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
- •12. Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
- •13. Presence of any internal guided growth plates/devices.
- •14. History of removal of internal guided growth plates/devices within less than 6 months.
- •15. History of receipt of any investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
- •16. History of receipt of an investigational product (not for achondroplasia/growth affecting) within the last 30 days or 5 half-lives (whichever is longer).
- •Other Exclusions
- •15. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
研究者
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