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Peptide
BMN 111 for Achondroplasia
Phase 3
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Female >= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to at least 16 years of age for females and 18 years of age for males
Awards & highlights
Study Summary
This trial is testing if a new drug, BMN 111, is a safe and effective treatment for children with achondroplasia.
Who is the study for?
This trial is for children with Achondroplasia who completed Study 111-301. They must be willing to use contraception if sexually active, and girls over 10 or menstruating need a negative pregnancy test. Participants should commit to study procedures, and minors require consent from guardians.Check my eligibility
What is being tested?
The Phase 3 study tests BMN 111's effectiveness and safety in treating Achondroplasia in children. It aims to provide an additional therapeutic option by extending previous research findings from an earlier phase of the study.See study design
What are the potential side effects?
While specific side effects are not listed here, potential risks may include reactions related to growth patterns since BMN 111 targets bone development disorders like Achondroplasia.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am a female over 10 or have started my periods and can take pregnancy tests.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to at least 16 years of age for females and 18 years of age for males
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to at least 16 years of age for females and 18 years of age for males
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from baselines in mean annualized growth velocity
Secondary outcome measures
BMN 111 Activity Biomarkers
Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire
Characterize maximum concentration (Cmax) of BMN 111 in plasma
+10 moreOther outcome measures
Optional exploratory genomic biomarker analysis
Side effects data
From 2019 Phase 3 trial • 121 Patients • NCT0319776673%
Injection site reaction
68%
Injection site erythema
38%
Injection site swelling
27%
Vomiting
27%
Nasopharyngitis
23%
Headache
17%
Pyrexia
15%
Arthralgia
13%
Injection site urticaria
13%
Upper respiratory tract infection
12%
Blood pressure decreased
12%
Cough
10%
Ear pain
10%
Oropharyngeal pain
10%
Otitis media
10%
Diarrhoea
10%
Ear infection
8%
Pain in extremity
8%
Viral infection
8%
Injection site bruising
8%
Influenza
7%
Fatigue
7%
Gastroenteritis
7%
Gastroenteritis viral
7%
Injection site mass
7%
Seasonal allergy
7%
Dizziness
7%
Fall
5%
Arthropod bite
5%
Nausea
5%
Tonsillitis
5%
Injection site rash
5%
Otorrhoea
5%
Abdominal pain
5%
Nasal congestion
5%
Dry skin
5%
Vitamin D deficiency
3%
Ligament sprain
3%
Procedural anxiety
3%
Neck pain
3%
Bone contusion
3%
Injection site induration
3%
Body temperature increased
3%
Injection site vesicles
3%
Bronchitis
3%
Otitis externa
3%
Injection site pain
3%
Injection site haemorrhage
3%
Enterobiasis
3%
Injection site inflammation
3%
Abdominal pain upper
3%
Paraesthesia
3%
Presyncope
3%
Rhinorrhoea
3%
Tympanic membrane perforation
2%
Skin abrasion
2%
Pallor
2%
Pharyngitis
2%
Localised infection
2%
Streptococcal infection
2%
Viral pharyngitis
2%
Gingival pain
2%
Back injury
2%
Arthropod sting
2%
Procedural dizziness
2%
Thermal burn
2%
Nasal obstruction
2%
Drug eruption
2%
Dermatitis allergic
2%
Rash pruritic
2%
Hyperhidrosis
2%
Adenoidal hypertrophy
2%
Impetigo
2%
Migraine
2%
Otitis media acute
2%
Viral upper respiratory tract infection
2%
Scarlet fever
2%
Tooth abscess
2%
Pruritus
2%
Enuresis
2%
Cellulitis
2%
Influenza like illness
2%
Lower respiratory tract infection
2%
Back pain
2%
Contusion
2%
Sleep apnoea syndrome
2%
Ocular hyperaemia
2%
Arthropathy
2%
Sinusitis
2%
Radius fracture
2%
Varicella
2%
Constipation
2%
Pulmonary congestion
2%
Gait disturbance
2%
Lethargy
2%
Injection site discolouration
2%
Injection site pruritus
2%
Croup infectious
2%
Hand-foot-and-mouth disease
2%
Disturbance in attention
2%
Hyperreflexia
2%
Hypotension
2%
Scratch
2%
Ear discomfort
2%
Excessive cerumen production
2%
External ear inflammation
2%
Middle ear effusion
2%
Acanthosis nigricans
2%
Acne
2%
Rash
2%
Malaise
2%
Pain
2%
Lip swelling
2%
Malpositioned teeth
2%
Device expulsion
2%
Vulvovaginal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
BMN 111 - 15 μg/kg
Trial Design
1Treatment groups
Experimental Treatment
Group I: BMN 111Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
BMN 111
2016
Completed Phase 3
~310
Find a Location
Who is running the clinical trial?
BioMarin PharmaceuticalLead Sponsor
156 Previous Clinical Trials
190,606 Total Patients Enrolled
13 Trials studying Achondroplasia
2,996 Patients Enrolled for Achondroplasia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am taking medication that affects my kidney function.I stopped taking BMN 111 or placebo before finishing the 111-301 study.My guardian or I have agreed to participate in the study and signed the consent form.My growth has slowed to less than 1.5 cm/year or my growth plates have closed.I am willing and able to follow all study requirements.I am a female over 10 or have started my periods and can take pregnancy tests.
Research Study Groups:
This trial has the following groups:- Group 1: BMN 111
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any similar investigations to BMN 111?
"The first clinical trials for BMN 111 took place in 2016 at Murdoch Children's Research Institute. As of now, 4 trials have completed with 5 more active trials underway, a majority of which are based in Chicago, Illinois."
Answered by AI
From how many physical sites is this trial being coordinated?
"In total, there are 14 sites where this trial is running. These locations are: Ann and Robert H. Lurie Children's Hospital of Chicago in Chicago, Illinois, Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio, Johns Hopkins University in Baltimore, Maryland, as well as 11 other hospitals."
Answered by AI
Does BMN 111 have FDA backing?
"This particular medication, BMN 111, is estimated to be safe based on its Phase 3 status. This means that, while there is some data supporting its efficacy, there are also multiple rounds of data supporting its safety."
Answered by AI
How many individuals are being monitored as part of this clinical investigation?
"This particular medical trial is no longer recruiting patients. The clinical trial was first posted on December 12th, 2017 and was most recently edited on June 30th, 2022. However, if you are exploring other studies, there are 12 clinical trials actively recruiting patients with achondroplasia and 5 trials for BMN 111 actively enrolling patients."
Answered by AI
To your knowledge, has something similar to this been done before?
"BMN 111 has undergone extensive research since 2016, when the first Phase 1 clinical trial was sponsored by BioMarin Pharmaceutical. After the initial study involving 30 patients, BMN 111 received Phase 2 drug approval in 2016. There are currently 5 active studies being conducted for BMN 111 across 16 cities and 8 countries."
Answered by AI
Who else is applying?
What site did they apply to?
Seattle Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
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