A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Phase 2

Completed

• Conditions: Achondroplasia

• Registration Number: JPRN-jRCT2080224833
• Lead Sponsor: BioMarin Pharmaceutical Inc.(ICCC:EPS Corporation)

Brief Summary

Preliminary data from the sentinel group showed improvements in height Z-scores in all cohorts. Adverse events occurred in all 1 patients in the sentinel group and 64 patients in the randomized group. SAEs occurred in 2 patients (oxygen saturation decreased, and petit mal epilepsy in 1 patient each) in the randomized group in Cohort 1, 2 patients (autism spectrum disorder, and gastroenteritis in 1 patient each) in the randomized group in Cohort 2, and 5 patients (otitis media and skull fracture in 1 patient, parainfluenzae virus infection, pneumonia, respiratory syncytial (RS) virus bronchiolitis, dyspnoea, and vomiting in 1 patient each) in the randomized group in Cohort 3. All these events were assessed as not related to the investigational product. No AEs leading to discontinuation of the study or discontinuation of treatment with the investigational product or deaths were reported by the time of data cut-off.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-08-19
• Study Completion: 2022-04-15
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 62

Inclusion Criteria

Diagnosis of ACH,confirmed by genetic testing
-Age 0 to < 60 months at study entry (Day1)
-At least 6 month period of pretreatment growth assessment in Study 111-901 immediately before study entry (for cohort 1 and 2) or at least 3 months of observation in either Study 111-901 or Study 111-206 prior to treatment (for cohort 3)

Exclusion Criteria

1.Have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.)
2.Have any of the following:
-Hypothyroidism or hyperthyroidism
-Insulin-requiring diabetes mellitus
-Autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus, juvenile dermatomyositis, scleroderma, etc.)
-Inflammatory bowel disease
-Autonomic neuropathy
3.Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F > 450 msec on screening ECG
4.Have evidence of cervicomedullary compression (CMC) likely to require surgical intervention within 60 days of Screening as determined by the Investigator and informed by the following assessments:
-Physical exam (eg, neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
-Polysomnography (eg, severe central sleep apnea)
-MRI indicating presence of severe CMC or spinal cord damage
5.Subject weight < 5.0 kg (cohort 1 & 2) or < 4.0 kg (cohort 3)
6.Treatment with growth hormone within 6-months prior to screening or prolonged treatment (> 3 months) at any time
7.Any history of spine or long-bone surgery or any bone-related surgery with chronic complications
8.Any history of limb-lengthening surgery or planned limb-lengthening during the study
9.Fracture of the long bones within 6 months prior to screening

Study & Design

• Study Type: Interventional
• Study Design: Not specified