Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 105
- 主要终点
- Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs.
研究概览
简要总结
The Increlex® Global Registry is a descriptive, multicenter, observational, prospective, open-ended, non interventional, post-authorisation surveillance registry.
The main purpose of this global registry is to collect, analyse and report safety data during and up to at least 5 years after the end of treatment in children and adolescents receiving Increlex® therapy for SPIGFD according to the locally approved product information.
详细描述
This registry is a Post-Authorisation Safety Study called the Increlex® Global Registry which is intended primarily to monitor the safety of Increlex® therapy in children and adolescents with Severe Primary IGF-1 Deficiency and secondly to follow the effectiveness of this treatment. Patients who have already started Increlex® therapy before entering this registry may be included and data will be collected retrospectively.
The countries participating in this registry are Austria, France, Germany, Italy, Poland, Spain, Sweden, United Kingdom and the USA
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For US : patients starting or planning to start or currently receiving treatment with Increlex® therapy for severe primary IGF-1 deficiency as defined by the US Increlex® prescribing information or for growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.For EU : patients starting or planning to start or currently receiving treatment with Increlex® therapy according to the locally approved product information.
- •Parents or legally authorized representatives if applicable must give signed informed consent before any registry-related activities are conducted. Assent from the subject should also be obtained as appropriate
排除标准
- •Subject currently participating in an Increlex® clinical trial
- •Subject currently participating in any clinical trial for growth retardation
- •Patient with any contraindication to Increlex® or any condition subject to special warning as per the locally approved label
- •For US patients, these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia and patients with closed epiphyses.
- •For EU patients: these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia or any condition or medical history which increases the risk of benign or malignant neoplasia and patients with closed epiphyses
结局指标
主要结局
Incidence of SAEs (including AESI of neoplasia) and all AEs, targeted AEs, deaths and withdrawals due to AEs.
时间窗: During the treatment period up to 30 days after the last dose.
Targeted AE includes hypersensitivity; scoliosis; immunogenicity (presence of antibodies if available); slipped capital femoral epiphysis, headache, otitis media, papilloedema, hypoglycaemia (suspected or documented - documented means blood level glucose \< 50 mg/dL or 2.78 mmol/L), acromegalic facial changes, gynaecomastia, hearing loss, intracranial hypertension, lipohypertrophy at injection sites, sleep apnoea, tonsillar hypertrophy, cardiomegaly, oedema and myalgia.
次要结局
- Description of neoplasia (benign and malignant) and hypoglycaemia(Within the first 3 years after treatment start, between 3 and 5 years and over 5 years.)
- Incidence of special situations and concomitant medications(During the treatment period an average of 5 years and within 5 years post-treatment)
- Modelisation to identify predictive factors of height SDS change(From baseline at least up to 5 years or until the final adult height is achieved.)
- Modelisation to identify predictive factors of bone age development(From baseline at least up to 5 years or until the final adult height is achieved)
- Body mass index (BMI)(From baseline at least up to 5 years or until the final adult height is achieved.)
- Modelisation to identify predictive factors of pubertal (Tanner) stage(From baseline at least up to 5 years or until the final adult height is achieved)
- Changes in QoL assessment using EQ-5D in participant aged 4 and over.(At baseline, at year one, at least up to 5 years, at Final Adult Height.)
- Height velocity(From baseline at least up to 5 years or until the final adult height is achieved.)
- Bone age development(From baseline at least up to 5 years or until the final adult height is achieved)
- Biological assessment : baseline GH concentrations, IGF-1 levels, IGFBP-3 levels and binding proteins.(Throughout study at least up to 5 years.)
- Incidence of SAEs (including AESI of neoplasia), targeted AEs, all AEs, deaths, withdrawals due to AEs, special situations and concomitant medications(Within 5 years post-treatment)
- Changes in height Standard Deviation Score (SDS)(From baseline at least up to 5 years or until the final adult height is achieved.)
- Estimation of differences between predicted adult height (PAH) and final adult height (FAH)(From baseline at least up to 5 years or until the final adult height is achieved.)
- Modelisation to identify predictive factors of Height velocity(From baseline at least up to 5 years or until the final adult height is achieved)
- Dose of Increlex® administrated(Periodically assessed during the study until treatment stop at least up to 5 years.)
- Presence or absence of gene deletion/mutation(Throughout study at least up to 5 years.)
- Pubertal stage(From baseline at least up to 5 years or until the final adult height is achieved.)
- Modelisation to identify predictive factors of FAH(From baseline at least up to 5 years or until the final adult height is achieved)
- Duration of Increlex exposure(Periodically assessed during the study until treatment stop at least up to 5 years.)
- Description of effectiveness parameters height SDS according to average dose received and according to dose ranges (e.g. 4 dose ranges (≤50, ]50-80], ]80-110], > 110 μg/kg BID)).(Periodically assessed during the study until treatment stop at least up to 5 years.)
- Description of effectiveness parameters height velocity according to average dose received and according to dose ranges (e.g. 4 dose ranges (≤50, ]50-80], ]80-110], > 110 μg/kg BID)).(Periodically assessed during the study until treatment stop at least up to 5 years.)