A study looking at growth measurements of children with achondroplasia

Phase 1

• Conditions: Achondroplasia; MedDRA version: 20.0Level: LLTClassification code 10000452Term: AchondroplasiaSystem Organ Class: 100000004850; Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]

• Registration Number: EUCTR2017-000701-21-DE
• Lead Sponsor: BioMarin Pharmaceutical Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-04-27
• Last Update Posted: 22 March 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

Individuals eligible to participate in this study must meet all of the following criteria:
1. Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure.
2. Birth to = 17 years of age at study entry.
3. Have ACH, documented by clinical diagnosis
4. Are ambulatory and able to stand without assistance (not applicable for children who are younger than 5 years of age and less than 104 cm in length)
5. Are willing and able to perform all study procedures as physically possible
Are the trial subjects under 18? yes
Number of subjects for this age range: 500
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

Individuals who meet any of the following exclusion criteria are not
eligible to participate in the study:
1. Have hypochondroplasia or short stature condition other than ACH
(e.g., trisomy 21, pseudoachondroplasia)
2. Have any of the following disorders:
o Hypothyroidism
o Insulin-requiring diabetes mellitus
o Autoimmune inflammatory disease (including celiac disease, lupus
(SLE), juvenile dermatomyositis, scleroderma, and others)
o Inflammatory bowel disease
o Autonomic neuropathy
3. Have an unstable clinical condition likely to lead to intervention during
the course ofthe study, including progressive cervical medullary
compression
4. Growth plates have fused
5. Have a history of any of the following:
o Renal insufficiency
o Anemia
6. Have a history of cardiac or vascular disease, including the following:
o Cardiac dysfunction
o Hypertrophic cardiomyopathy
o Congenital heart disease
o Cerebrovascular disease, aortic insufficiency
o Clinically significant atrial or ventricular arrhythmias
7. Current treatment with antihypertensive medications, ACE inhibitors,
angiotensin IIreceptor blockers, diuretics, beta-blockers, calciumchannel
blockers, cardiacglycosides, systemic anticholinergic agents, any
medication that may impair orenhance compensatory tachycardia, drugs
known to alter renal function that isexpected to continue for the
duration of the study
8. Have been treated with growth hormone, insulin-like growth factor 1
(IGF-1), oranabolic steroids in the previous 6 months or long-term
treatment (> 3 months) at anytime
9. Have had regular long-term treatment (> 1 month) with oral
corticosteroids (low-dose ongoing inhaled steroid for asthma is
acceptable) in the previous 12 months
10. Concomitant medication that prolongs the QT/QTc interval within 14
days or5 half-lives, whichever is longer, before the Screening visit
11. Have used any other investigational product or investigational
medical device for the treatment of ACH or short stature
12. Planned or expected bone-related surgery (ie. surgery involving
disruption of bone cortex), during the study period. Subjects with
previous bone-related surgery may enroll if surgery occurred at least 12
months prior to the study and healing is complete without sequelae.
13. 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 the study and
healing is complete without sequelae.
14. Have any condition that, in the view of the Investigator, places the
subject at high risk of poor compliance with the visit schedule or of not
completing the study.
15. Concurrent disease or condition that, in the view of the Investigator, would interferewith study participation.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified