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Peptide

BMN 111 for Achondroplasia (ACH Trial)

Phase 2

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

Parents/caregivers willing to administer daily injections to the subjects and complete the required training

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

ACH Trial Summary

This trial is to study the long-term effects of a medication given to children with achondroplasia, a form of dwarfism. The study will track the children's safety and tolerability of the medication over a period of time.

Who is the study for?

This trial is for children with Achondroplasia who have already completed 24 months of BMN 111 treatment in a previous study. They must be able to perform study procedures, use contraception if sexually active, and not be pregnant. Those reaching legal adult age during the study will need to consent again.Check my eligibility

What is being tested?

The trial tests the long-term safety and effectiveness of daily injections of BMN 111 in children with Achondroplasia over an extended period after they've been treated for two years in a prior study (111-202).See study design

What are the potential side effects?

While specific side effects are not listed here, the trial aims to monitor any adverse reactions from long-term use of BMN 111 through daily injections administered by parents or caregivers.

ACH Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My caregiver is ready to give me daily injections and has completed the necessary training.

ACH Trial 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

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to at least 16 years of age for females and 18 years of age for males for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Secondary outcome measures

Body Proportions (Efficacy)

Final Adult Height

Growth Parameters (Efficacy)

+1 more

Side effects data

From 2019 Phase 3 trial • 121 Patients • NCT03197766

73%

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

Pain in extremity

Viral infection

Injection site bruising

Influenza

Fatigue

Gastroenteritis

Gastroenteritis viral

Injection site mass

Seasonal allergy

Dizziness

Fall

Arthropod bite

Nausea

Tonsillitis

Injection site rash

Otorrhoea

Abdominal pain

Nasal congestion

Dry skin

Vitamin D deficiency

Ligament sprain

Procedural anxiety

Neck pain

Bone contusion

Injection site induration

Body temperature increased

Injection site vesicles

Bronchitis

Otitis externa

Injection site pain

Injection site haemorrhage

Enterobiasis

Injection site inflammation

Abdominal pain upper

Paraesthesia

Presyncope

Rhinorrhoea

Tympanic membrane perforation

Nasal obstruction

Skin abrasion

Localised infection

Streptococcal infection

Arthropod sting

Thermal burn

Drug eruption

Back injury

Procedural dizziness

Pallor

Viral pharyngitis

Gingival pain

Pharyngitis

Dermatitis allergic

Rash pruritic

Hyperhidrosis

Adenoidal hypertrophy

Impetigo

Migraine

Otitis media acute

Viral upper respiratory tract infection

Scarlet fever

Tooth abscess

Pruritus

Enuresis

Cellulitis

Influenza like illness

Lower respiratory tract infection

Back pain

Contusion

Sleep apnoea syndrome

Ocular hyperaemia

Arthropathy

Sinusitis

Radius fracture

Varicella

Constipation

Pulmonary congestion

Gait disturbance

Lethargy

Injection site discolouration

Injection site pruritus

Croup infectious

Hand-foot-and-mouth disease

Disturbance in attention

Hyperreflexia

Hypotension

Scratch

Ear discomfort

Excessive cerumen production

External ear inflammation

Middle ear effusion

Acanthosis nigricans

Acne

Rash

Malaise

Pain

Lip swelling

Malpositioned teeth

Device expulsion

Vulvovaginal pain

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

BMN 111 - 15 μg/kg

ACH Trial Design

1Treatment groups

Experimental Treatment

Group I: BMN 111 - Subcutaneous InjectionExperimental Treatment1 Intervention

111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

BMN 111

2016

Completed Phase 3

~310

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor

156 Previous Clinical Trials

190,695 Total Patients Enrolled

13 Trials studying Achondroplasia

3,085 Patients Enrolled for Achondroplasia

Medical Director, MDStudy DirectorBioMarin Pharmaceutical

76 Previous Clinical Trials

16,214 Total Patients Enrolled

6 Trials studying Achondroplasia

914 Patients Enrolled for Achondroplasia

Media Library

BMN 111 (Peptide) Clinical Trial Eligibility Overview. Trial Name: NCT02724228 — Phase 2

Achondroplasia Research Study Groups: BMN 111 - Subcutaneous Injection

Achondroplasia Clinical Trial 2023: BMN 111 Highlights & Side Effects. Trial Name: NCT02724228 — Phase 2

BMN 111 (Peptide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02724228 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the potential dangers of taking BMN 111?

"While there is some data indicating BMN 111's safety, it has not been proven effective yet. Therefore, it received a score of 2."

Answered by AI

How many test subjects are in this experiment?

"As of right now, this clinical trial has completed recruitment for participants. The study was originally posted on 1/26/2016 and ended on 6/30/2022. If you are looking to participate in other studies, there are 12 trials for achondroplasia and 5 for BMN 111 that are still admitting patients."

Answered by AI

Are patients still needed for this clinical trial?

"This particular clinical trial is no longer enrolling patients. The study was first posted on 1/26/2016 and the most recent update was 6/30/2022. There are 12 other trials currently looking for candidates with achondroplasia and 5 recruiting for BMN 111."

Answered by AI

Are there any other sites outside of North America conducting this research?

"Currently, this study is being hosted at six different locations. These include the Baylor College of Medicine in Houston, Ann and Robert H. Lurie Childrens Hospital of Chicago in Chicago, and Johns Hopkins McKusick - Institute of Genetic Medicine in Baltimore."

Answered by AI

What other research studies have included BMN 111 as a variable?

"Five separate clinical trials are currently studying BMN 111. One of these studies has progressed to Phase 3. The majority of research for BMN 111 is conducted in Osaka and Texas; however, there are 53 total research sites for this medication."

Answered by AI

Have there been similar investigations like this one in the past?

"Since 2016, BMN 111 has been the subject of clinical research. In that year, the first trial was conducted with 30 patients and was sponsored by BioMarin Pharmaceutical. After this initial Phase 1 study, BMN 111 received approval for Phase 2 drug trials. Currently, there are 5 active trials being conducted in 16 cities across 8 countries."

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

C-type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia

Savarirayan, Ravi, Melita Irving, Carlos A. Bacino, Bret Bostwick, Joel Charrow, Valerie Cormier-Daire, Kim-Hanh Le Quan Sang, et al.. 2019. “C-type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1813446.

clinicaltrials.govStudy

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

2016. "A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02724228.

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