EUCTR2017-004972-74-ATActive, not recruitingPhase 1
An Open-label, Ascending Multiple dose Study to Evaluate Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
Amgen Inc.0 sites24 target enrollmentStarted: March 24, 2022Last updated:
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 24
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •Subject's legally acceptable representative has provided informed
- •consent and the subject has provided written assent based on local
- •regulations and/or guidelines prior to initiation of any study-specific
- •activities/procedures
- •Ambulatory male or female children 5 to less than 12 years of age
- •(cohorts 2,4, and 6) or adolescents 12 to less than 18 years of age
- •(cohorts 1, 3 and 5) upon entry into screening
- •Clinical diagnosis of OI defined as a clinical history consistent with type
- •I-IV OI as determined by presence of expected phenotype (eg, facial
- •shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic
- •features, fracture pattern) and lack of additional features unrelated to
- •type I-IV OI (eg, blindness, mental retardation, neuropathy,
- •craniosynostosis, premature exfoliation of deciduous teeth)
- •If familial, also must be autosomal dominant
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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
Exclusion Criteria
- •History of an electrophoresis pattern inconsistent with type I to type IV
- •History of known mutation in a gene other than type I collagen
- •alpha 1/type I collagen alpha 2 (COL1AI/COL1A2) causing OI or other
- •metabolic bone disease
- •History of congenital dislocation of the radial head, interosseous
- •membrane calcification, or exuberant callus formation
- •History of osteomalacia or rickets
- •Body weight less than 10 kg or greater than 90 kg
- •History of other bone diseases that affect bone metabolism (eg,
- •osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis,
- •osteopetrosis, hypophosphatasia)
- •History of Kawasaki disease, rheumatic myocarditis, ischemic
- •cardiomyopathy, inherited cardiomyopathies, valvular heart disease,
- •nephrotic syndrome, familial hypercholesterolemia, stroke, or any
- •thromboembolic disorder
- •Evidence of untreated or unhealed oral cavities or oral infections
- •Unhealed or planned invasive dental or tooth procedure; removal of baby
- •teeth is acceptable and not considered an invasive dental procedure
- •Unhealed fracture as defined by orthopedic opinion
- •Osteotomy, rodding surgery or spinal fusion surgery within 5 months
- •prior to screening, or not yet healed per orthopedic surgeon
- •Any planned major surgery, including skeletal surgery (eg, rodding
- •surgery, spinal surgery) within the next 6 months from day 1 that would
- •interfere with study procedures or would require missing of any IP
- •Symptoms associated with skull abnormalities such as basilar
- •invagination, basilar impression or Chiari malformation (headache
- •induced by coughing or straining for stool, or parasthesia or weakness)
- •History of malabsorption (in children with serum albumin < lower limit
- •normal [LLN]), malabsorption should be clinically ruled out by the
- •investigator to confirm eligibility)
- •History of long QT syndrome
- •History of malignancy
- •History of any solid organ or bone marrow transplant
- •Positive blood screen for hepatitis B surface antigen (HbsAg), hepatitis B
- •core antibody (HBcAb), or hepatitis C antibody (HepCAb), human
- •immunodeficiency virus (HIV) -1 or -2 antibody
- •History of hyper- or hypothyroidism, unless subject is on stable therapy
- •> 6 months and has supporting laboratory documentation within 6
- •months prior to or at screening indicating normal serum thyroidstimulating
- •hormone [TSH] value
- •Evidence of any of the following:
- •Current hyper- or hypoparathyroidism (parathyroid hormone outside the
- •normal range)
- •Renal disease: Estimated glomerular filtration rate (eGFR) < 60
- •mL/min/1.73 m2 (calculated by the bedside Schwartz equation at
- •eGFR (mL/min/1.73 m2)= 0.413 X (height/serum creatinine)
- •(Height is in centimeters, and serum creatinine is in mg/dL)
- •Current hypocalcemia (albumin-adjusted serum calcium < LLN of the
- •laboratory's reference range) or hypercalcemia (albumin-adjusted serum
- •calcium > ULN of the laboratory's reference range) at the time of
- +12 more not shown
Investigators
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