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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

Must not have

Current chronic therapy with restricted medications

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

No Placebo-Only Group

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.

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).

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.

Eligibility Criteria

Inclusion Criteria

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am on long-term medication that is restricted.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

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%

Oropharyngeal pain

10%

Ear pain

10%

Otitis media

10%

Diarrhoea

10%

Ear infection

Viral infection

Injection site bruising

Pain in extremity

Influenza

Fatigue

Injection site mass

Gastroenteritis viral

Gastroenteritis

Seasonal allergy

Dizziness

Fall

Tonsillitis

Injection site rash

Nausea

Arthropod bite

Otorrhoea

Abdominal pain

Nasal congestion

Dry skin

Vitamin D deficiency

Injection site induration

Injection site inflammation

Injection site vesicles

Injection site pain

Injection site haemorrhage

Otitis externa

Bronchitis

Enterobiasis

Abdominal pain upper

Neck pain

Bone contusion

Ligament sprain

Procedural anxiety

Body temperature increased

Paraesthesia

Presyncope

Rhinorrhoea

Tympanic membrane perforation

Gait disturbance

Radius fracture

Adenoidal hypertrophy

Sleep apnoea syndrome

Cellulitis

Lower respiratory tract infection

Influenza like illness

Impetigo

Otitis media acute

Pharyngitis

Sinusitis

Tooth abscess

Varicella

Viral pharyngitis

Viral upper respiratory tract infection

Localised infection

Scarlet fever

Streptococcal infection

Lethargy

Migraine

Constipation

Gingival pain

Arthropathy

Back pain

Arthropod sting

Back injury

Contusion

Procedural dizziness

Skin abrasion

Thermal burn

Nasal obstruction

Pulmonary congestion

Dermatitis allergic

Drug eruption

Hyperhidrosis

Pruritus

Rash pruritic

Pallor

Ocular hyperaemia

Enuresis

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 2Eligibility criteria met