CompletedPhase 2

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

United States75 participantsStarted 2018-06-13

Plain-language summary

Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.

Who can participate

Age range59 Months

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of achondroplasia (ACH), confirmed by genetic testing. If subjects had previous genetic testing, subjects must have a lab report from a certified laboratory with the study specific mutation documented.
✓. Age 0 to \< 60 months, at study entry (Day 1)
✓. Cohort 1 and 2 subjects must have at least a 6-month period of pretreatment growth assessment in Study 111 901 immediately before screening, and have one documented measurement of height/body length a minimum of 6 months prior to the screening visit for 111-206. Cohort 3 subjects must have a minimum of 3 months of observation prior to treatment. This observational period can be obtained either (1) via prior enrollment in Study 111-901 or (2) via enrollment in this Study 111 206 for a minimum of 3 months of non-treatment observation prior to commencement of treatment.
✓. Parent(s) or guardian(s) (and the subjects themselves, if required by local regulations or ethics committee) 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
✓. Willing and able to perform all study procedures as physically possible
✓. Parent(s) or caregiver(s) are willing to administer daily injections to the subjects and complete the required training

Exclusion criteria

✕. Have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.)
✕. Subject weighs \< 5.0 kg (Cohort 1 and 2) or \< 4.0 kg (Cohort 3)
✕. Have any of the following:
✕. Have a history of any of the following:
✕. Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or Fridericia's corrected QT interval (QTc-F) \>450 msec on screening ECG
✕. Have evidence of cervicomedullary compression (CMC) likely to require surgical intervention within 60 days of Screening as determined by the Investigator based on the following assessments
✕. Have an unstable medical condition likely to require surgical intervention in the next 6 months, or planned spine or long-bone surgery (i.e., surgery involving significant disruption of bone cortex) during the study period
✕. Have documented uncorrected Vitamin D deficiency: 25(OH)D \<=15 ng/mL (37.5 nmol/L)

What they're measuring

Number of Participants With Adverse Events (AEs) by Severity Grade and Study Drug Treatment-emergent Adverse Events (TEAEs)

Timeframe: Up to Week 56 (Safety Follow-Up +/-7d)

Change From Baseline in Height Z-score at Week 52.

Timeframe: Baseline to Week 52

Trial details

NCT IDNCT03583697

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-01-26

Results submitted2023-11-14

View on ClinicalTrials.gov More Achondroplasia trials