AchondroplasiaPhase 1NCT05813314
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
A Phase 1 study testing BMN 111 administration via Injector Pen and BMN 111 administration via vial and syringe in people with Achondroplasia, run by BioMarin Pharmaceutical at 1 site in United States.
Trial summary
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Achondroplasia
- What is being tested?
- BMN 111 administration via Injector Pen and BMN 111 administration via vial and syringe
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Mar 2023to 14 Jun 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Participants must be aged 18 to 55 years inclusive, at the time of signing the informed consent.
- Participants must be non-smoking (have never smoked, or stopped smoking > 3 months prior to dosing), and can be of any ethnicity.
- Participants must be healthy as determined by the absence of clinically relevant abnormalities identified by medical evaluation, including medical history, full physical examination, laboratory tests, cardiac monitoring, supine vital signs to include temperature, and assessment of prior and concomitant medications.
- Body weight ≥ 60 kg and < 110 kg and body mass index within the range 18.0 to 32.0 kg/m2 (inclusive).
- Males and/or females are eligible to participate in this clinical study.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Females will not be pregnant or lactating, and females of childbearing potential and males will agree to use contraception.
- Participants must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF).
Who cannot join
- Any condition considered to affect drug absorption (eg, conditions affecting SC administration such as reduced SC blood flow, lipohypertrophy, and significantly high/low skin temperature).
- Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder, as determined by the investigator (or designee).
- History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the investigator (or designee).
- History of migraines.
- Confirmed (eg, two consecutive measurements) systolic blood pressure > 140 or < 90 mmHg, diastolic blood pressure > 90 or < 50 mmHg, and pulse rate > 100 or < 40 beats per minute or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms e.g., dizziness and fainting) or recurrent symptomatic orthostatic hypotension.
- Positive hepatitis panel and/or positive human immunodeficiency virus test.
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- Exposure to mycobacterium tuberculosis, indicative of tuberculosis infection, as confirmed by a positive QuantiFERON-TB Gold test.
- A positive COVID-19 test at Screening or Check-in.
- Taking any of the prohibited medications.
- Administration of a coronavirus disease 2019 (COVID-19) vaccine within 30 days prior to dosing.
- Use or intend to use any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John's wort, within 30 days prior to dosing, unless deemed acceptable by the investigator (or designee).
- Use or intend to use any prescription medications/products other than hormone replacement therapy (HRT), oral, implantable, transdermal, injectable, or intrauterine contraceptives within 7 days (or 14 days if the drug is a potential enzyme inducer), or 5 half-lives (whichever is longer), prior to dosing, unless deemed acceptable by the investigator (or designee).
- Use or intend to use slow release medications/products considered to still be active within 14 days prior to Check-in, unless deemed acceptable by the investigator (or designee).
- Use or intend to use any nonprescription medications/products including vitamins, minerals, and phytotherapeutic/herbal/plant derived preparations within 7 days, or 5 half-lives (whichever is longer), prior to dosing, unless deemed acceptable by the investigator (or designee).
- Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the past 30 days or 5 half-lives prior to dosing (whichever is longer).
- Have previously completed or withdrawn from this study or any other study investigating BMN 111 and have previously received BMN 111.
- Aspartate aminotransferase, alanine aminotransferase, or bilirubin ≥ 1.5 × upper limit of normal or estimated glomerular filtration rate < 60 mL/min/1.73m2 (Chronic Kidney Disease Epidemiology Collaboration 2021).
- Receipt of blood products within 2 months prior to Check-in.
- Donation of blood from 3 months prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening.
- Poor peripheral venous access.
- Participants who, in the opinion of the investigator (or designee), should not participate in this study.
- Any skin condition, such as scarring or tattoos, which may preclude receipt of the study drug.
- Unwilling or unable to comply with scheduled visits, the treatment plan, laboratory tests and other study procedures or lifestyle guidance.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Open-Label, Sponsor-Open, Two-Sequence, Two-Period Crossover Study to Investigate the Bioequivalence of a Single Subcutaneous Dose of BMN 111 Administered Using Either a Vial and Syringe or Injector Pen in Healthy Adult Participants
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
- Conditions
- Achondroplasia
- Being tested
- BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 111 administration via Injector PenCombination product
BMN 111 Injection will be given using an injector pen
BMN 111 administration via vial and syringeDrug
BMN 111 Injection will be given using vial and syringe
| Study group | Role | What they receive |
|---|---|---|
| BMN 111 injection with vial and syringe | Active comparator | Study treatment will be provided in glass vials. Each glass vial will be labeled as required per country requirement. Pre-filled Diluent Transfer Syringes will be provided for reconstitution. Receives: Drug: BMN 111 administration via vial and syringe |
| BMN 111 injection with injector pen | Experimental | Study treatment will be provided in a prefilled injector pen containing a dual chamber drug cartridge, for reconstitution and injection, after setting of the specified dose with the 2.0 mg/mL formulation. Receives: Combination Product: BMN 111 administration via Injector Pen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum observed drug concentration (Cmax)
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Area under the concentration versus time curve from time 0 to infinity (AUC0-∞)
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Area under the concentration versus time curve from time 0 to t, where t is the last timepoint with a measurable concentration (AUC0-t)
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Secondary outcomes (4)
Time of Cmax (tmax)
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Time of t1/2
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Time of terminal rate constant (λZ)
Measured: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Incidence and severity of adverse events
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2023
- Study start15 Mar 2023
- Primary completion14 Jun 2023
- Study completion14 Jun 2023
Why it stopped: The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05813314
- Sponsor's protocol ID
- 111-104
- Status last verified
- Sep 2023
- First submitted
- 16 Mar 2023
- Last updated
- 12 Oct 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Open-Label, Sponsor-Open, Two-Sequence, Two-Period Crossover Study to Investigate the Bioequivalence of a Single Subcutaneous Dose of BMN 111 Administered Using Either a Vial and Syringe or Injector Pen in Healthy Adult Participants
- ClinicalTrials.gov ID
- NCT05813314
- Acronym / sponsor ID
- 111-104
- Conditions
- Achondroplasia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns.
- Enrolment
- 36 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 15 Mar 2023 (actual)
- Primary completion
- 14 Jun 2023 (actual)
- Study completion
- 14 Jun 2023 (actual)
- First posted
- 14 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BMN 111 injection with vial and syringe | Active comparator | Drug: BMN 111 administration via vial and syringeStudy treatment will be provided in glass vials. Each glass vial will be labeled as required per country requirement. Pre-filled Diluent Transfer Syringes will be provided for reconstitution. |
| BMN 111 injection with injector pen | Experimental | Combination Product: BMN 111 administration via Injector PenStudy treatment will be provided in a prefilled injector pen containing a dual chamber drug cartridge, for reconstitution and injection, after setting of the specified dose with the 2.0 mg/mL formulation. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Participants must be aged 18 to 55 years inclusive, at the time of signing the informed consent.
- Participants must be non-smoking (have never smoked, or stopped smoking > 3 months prior to dosing), and can be of any ethnicity.
- Participants must be healthy as determined by the absence of clinically relevant abnormalities identified by medical evaluation, including medical history, full physical examination, laboratory tests, cardiac monitoring, supine vital signs to include temperature, and assessment of prior and concomitant medications.
- Body weight ≥ 60 kg and < 110 kg and body mass index within the range 18.0 to 32.0 kg/m2 (inclusive).
- Males and/or females are eligible to participate in this clinical study.
Key exclusion criteria (23 listed)
- Any condition considered to affect drug absorption (eg, conditions affecting SC administration such as reduced SC blood flow, lipohypertrophy, and significantly high/low skin temperature).
- Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder, as determined by the investigator (or designee).
- History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the investigator (or designee).
- History of migraines.
- Confirmed (eg, two consecutive measurements) systolic blood pressure > 140 or < 90 mmHg, diastolic blood pressure > 90 or < 50 mmHg, and pulse rate > 100 or < 40 beats per minute or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms e.g., dizziness and fainting) or recurrent symptomatic orthostatic hypotension.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMN 111 administration via Injector PenBMN 111 Injection will be given using an injector pen | Combination product | BMN 111 injection with injector pen |
| BMN 111 administration via vial and syringeBMN 111 Injection will be given using vial and syringe | Drug | BMN 111 injection with vial and syringe |
Primary outcome
Maximum observed drug concentration (Cmax)
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Area under the concentration versus time curve from time 0 to infinity (AUC0-∞)
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Area under the concentration versus time curve from time 0 to t, where t is the last timepoint with a measurable concentration (AUC0-t)
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
4 secondary outcomes
Time of Cmax (tmax)
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Time of t1/2
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Time of terminal rate constant (λZ)
Time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose
Incidence and severity of adverse events
Time frame: 2 weeks
Results and safety
The study was stopped early (reason given: The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BMN 111 administration via Injector Pen and BMN 111 administration via vial and syringe in people with Achondroplasia, run by BioMarin Pharmaceutical at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum observed drug concentration (Cmax) (time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2023.