EUCTR2015-003836-11-GB进行中（未招募）1 期
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 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
- 110
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
入选标准
- •1. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, subjects under the age of 18 are willing and able to provide written assent (if required by local regulations or the IRB/EC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of 18 years while the study is ongoing will be asked to provide their own written consent.
- •2. 5 to < 18 years old at study entry
- •3. Have ACH, documented by clinical grounds and confirmed by genetic testing
- •4. Have at least a 6-month period of pretreatment growth assessments, including standing height, and are currently active participants in 111-901.
- •5. Females = 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study
- •6. If sexually active, willing to use a highly effective method of contraception while participating in the study
- •7. Are ambulatory and able to stand without assistance
- •8. Are willing and able to perform all study procedures as physically possible
- •9. Caregivers are willing to administer daily injections to the subjects and complete the required training
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 110
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 2
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia)
- •2. Have any of the following: Hypothyroidism or hyperthyroidism; Insulin-requiring diabetes mellitus; Autoimmune
- •inflammatory disease (including celiac disease, lupus (SLE), juvenile dermatomyositis, scleroderma, and others); Inflammatory bowel disease; Autonomic neuropathy
- •3. Have a history of any of the following: Renal insufficiency defined as serum creatinine > 2 mg/dl; Chronic anemia; Baseline systolic blood pressure (BP) < 70 millimeters of mercury (mm Hg) or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms i.e., dizziness, fainting) or recurrent symptomatic orthostatic hypotension; Cardiac or vascular disease (including Cardiac dysfunction (abnormal echocardiogram) at Screening Visit; Hypertrophic cardiomyopathy; Pulmonary hypertension; Congenital heart disease; Cerebrovascular disease; Aortic insufficiency or other clin significant valvular dysfunction; Clinically significant atrial or ventricular arrhythmias)
- •4. Have a clinically significant finding or arrhythmia on screening electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
- •5. Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea)
- •6. Evidence of decreased growth velocity (AGV<1.5 cm/y) as assessed over a period of at least 6 mths or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays including both AP and lateral views
- •7. Documented Vitamin D deficiency (concentration of blood 25-hydroxy-vitamin D <12 ng/mL or <30 nmol/L)
- •8. Require any investigational agent prior to completion of study period9. Have received another investigational product or investigational medical device within 6 mths before the Screening Visit
- •10. Have used any investigational product or investigational medical device for the treatment of ACH or short stature at any time, including BMN 111
- •11. Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
- •12. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time
- •13. Have had > 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months
- •14. Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to screening and healing is complete without sequelae.
- •15. Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
研究者
相似试验
进行中（未招募）
1 期
A Study in Patients with Chronic Cluster HeadacheChronic Cluster HeadacheMedDRA version: 18.0 Level: LLT Classification code 10009698 Term: Cluster headaches System Organ Class: 100000004852EUCTR2014-005429-11-FREli Lilly and Company162
进行中（未招募）
1 期
A Study in Patients with Chronic Cluster HeadacheChronic Cluster HeadacheMedDRA version: 19.0 Level: LLT Classification code 10009698 Term: Cluster headaches System Organ Class: 100000004852EUCTR2014-005429-11-GREli Lilly and Company162
进行中（未招募）
1 期
A Clinical Study to evaluate the efficacy and safety of GS-5745 in combination with standard of care in patients with stomach cancer.EUCTR2015-001526-42-HUGilead Sciences, Inc.430
进行中（未招募）
1 期
A Study in Patients with Chronic Cluster HeadacheChronic Cluster HeadacheMedDRA version: 19.1Level: LLTClassification code 10009698Term: Cluster headachesSystem Organ Class: 100000004852EUCTR2014-005429-11-NLEli Lilly and Company237
已完成
3 期
A Phase 3 study of Abatacept in Patients with Primary Sjogrens SyndromePrimary Sjogrens SyndromeJPRN-jRCT2080223466Bristol-Myers Squibb K.K.172