AchondroplasiaPhase 1NCT01590446
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
A Phase 1 study testing BMN 111 and Normal Saline in people with Achondroplasia, run by BioMarin Pharmaceutical at 1 site in United States.
Trial summary
The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose. Information about any side effects that may occur will also be collected. Read more about this study
- Who is it for?
- Ages 22 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Achondroplasia
- What is being tested?
- BMN 111 and Normal Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 74 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2012to Jun 2012
- 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 22 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Is willing and able to provide written, signed informed consent (legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure.
- Is a male 22 to 45 years of age, inclusive
- Has a body weight between 63 and 100 kg, inclusive
- Has a body mass index (BMI) between 18 and 32 kg/m2, inclusive
- Is able and willing to abstain from nicotine, alcohol, methylxanthine-containing beverages or food (e.g., coffee, tea, colas, chocolate, energy drinks), poppy seeds, and grapefruit juice for 48 hours prior to admission and for the duration of the study
- Is in good health generally, as determined by medical history, physical examination, clinical laboratory evaluations, and 12-lead electrocardiogram (ECG) at Screening
- Is willing and able to perform all study procedures as physically possible
- If sexually active, is willing to use a condom during sexual intercourse with female partners and to have their female partners use an additional effective means of contraception (e.g., intrauterine device, coil, diaphragm plus spermicide, oral contraceptive) or to abstain from sexual intercourse if female partner is not surgically sterile by tubal occlusion (ligation or occluding device) or postmenopausal from time of initial admission to the research facility until their last study visit
Who cannot join
- Baseline systolic blood pressure < 100 mmHg
- Subjects with spontaneous orthostatic hypotension, including a systolic decline of > 20 or diastolic change of > 10 mmHg or heart rate increase of > 30 bpm
- Has renal insufficiency as determined by eGFR < 65 mL/min/1.73m2 using the revised Cockcroft-Gault calculation: (140 - age [y])
- body weight [kg] / 72 serum creatinine [mg/dL]
- Has anemia (Hb < 12.5 gm/dL)
- Has history of cardiac or vascular disease, including the following: Congenital heart disease; Hypertension or hypotension; Cerebrovascular disease; aortic insufficiency; Clinically significant atrial or ventricular arrhythmias; Cardiac valvular heart disease; Hypertrophic cardiomyopathy or other cardiomyopathy
Show all 26Show fewer
- Has a Screening ECG showing any of the following: Resting heart rate < 45 or > 100 bpm; PR interval > 210 msec; P wave duration > 120 msec; QRS interval < 70 or > 120 msec; Corrected QTc > 440 msec; QRS axis outside the range of -30 + 100 degrees; Right or left atrial enlargement or ventricular hypertrophy; Second- or third-degree atrioventricular block
- Heart block or intraventricular conduction defect
- Has diabetes mellitus Type I or Type II
- Is being treated with angiotensin-converting enzyme inhibitors, antihypertensive medications, diuretics, calcium-channel blockers, beta-blockers, cardiac glycosides, systemic anticholinergic agents, or drugs that may impair or enhance compensatory tachycardia
- Is being treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids.
- Has any acute illness associated with volume dehydration (e.g., nausea/vomiting/diarrhea).
- Uses of any prescription medications, over-the-counter medications, or nutritional supplements within 10 days prior to dosing.
- Uses any other investigational product or investigational medical device within 90 days prior to screening or requires any investigational agent prior to completion of all scheduled study assessments.
- Consumes at least 14 units/week of alcohol (1 unit approximates 360 mL beer, 100 mL wine, or 35 mL spirits) or has significant history of alcoholism or drug/chemical abuse as determined by the Investigator.
- Has donated > 50 mL of blood or plasma within 60 days prior to study treatment administration.
- Has a positive urine drug screen or alcohol breath test result during Screening or upon admission to the research facility.
- Has used nicotine or tobacco-containing products (snuff, chewing tobacco, cigarettes,cigars, pipes, and nicotine replacements) within 90 days of the first dose of study treatment as confirmed by urine cotinine screen.
- Has a positive cotinine test result during Screening or upon admission to the research facility.
- Has a history of any clinically significant neurological, gastrointestinal, renal, hepatic, cardiovascular, psychiatric, respiratory, metabolic, endocrine, hematological, or other major disorders as determined by the Investigator.
- Has had a clinically significant illness within 4 weeks of administration of the first dose of study treatment as determined by the Investigator.
- Is being treated with a concomitant medication that prolongs the QT/QTc interval within 7 days or 3 half-lives, whichever is longer, prior to the Screening Visit.
- Has AST or ALT greater than 3xULN or total bilirubin greater than 2xULN.
- Has known hypersensitivity to BMN 111 or its excipients.
- Has partner planning to become pregnant at any time during the study.
- Has any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Two-Part, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 111 Administered to Healthy Adult Volunteers
The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose. Information about any side effects that may occur will also be collected.
- Conditions
- Achondroplasia
- Being tested
- BMN 111, Normal Saline
- Keywords
- Achondroplasia
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 111Drug
Also called Modified C-Natriuretic Peptide, ProCNP38
SC injection, Part 1 single dose and Part 2 multiple dose.
Normal SalineDrug
SC injection, Part 1 single dose and Part 2 multiple dose
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Normal Saline |
| BMN 111 | Active comparator | Not described Receives: Drug: BMN 111 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety based on vitals signs
Measured: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
Safety based on adverse events
Measured: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
Secondary outcomes (2)
Pharmacokinetics
Measured: Daily on dosing days Assessed during Part 1 for approximately 10 days and during Part 2 for approximately 24 days
Safety based on cardiovascular effects
Measured: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2012 (estimated)
- Study startFeb 2012
- Primary completionJun 2012
- Study completionJun 2012
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
- Factorial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 74 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01590446
- Sponsor's protocol ID
- 111-101
- Status last verified
- Jun 2012
- First submitted
- 14 Mar 2012
- Last updated
- 11 Jun 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Two-Part, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 111 Administered to Healthy Adult Volunteers
- ClinicalTrials.gov ID
- NCT01590446
- Acronym / sponsor ID
- 111-101
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 74 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Feb 2012
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 3 May 2012 (estimated)
- 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 measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose. Information about any side effects that may occur will also be collected.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Normal Saline |
| BMN 111 | Active comparator | Drug: BMN 111 |
Who may qualify
- Ages
- 22 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Is willing and able to provide written, signed informed consent (legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure.
- Is a male 22 to 45 years of age, inclusive
- Has a body weight between 63 and 100 kg, inclusive
- Has a body mass index (BMI) between 18 and 32 kg/m2, inclusive
- Is able and willing to abstain from nicotine, alcohol, methylxanthine-containing beverages or food (e.g., coffee, tea, colas, chocolate, energy drinks), poppy seeds, and grapefruit juice for 48 hours prior to admission and for the duration of the study
Key exclusion criteria (26 listed)
- Baseline systolic blood pressure < 100 mmHg
- Subjects with spontaneous orthostatic hypotension, including a systolic decline of > 20 or diastolic change of > 10 mmHg or heart rate increase of > 30 bpm
- Has renal insufficiency as determined by eGFR < 65 mL/min/1.73m2 using the revised Cockcroft-Gault calculation: (140 - age [y])
- body weight [kg] / 72 serum creatinine [mg/dL]
- Has anemia (Hb < 12.5 gm/dL)
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 111Also: Modified C-Natriuretic Peptide, ProCNP38SC injection, Part 1 single dose and Part 2 multiple dose. | Drug | BMN 111 |
| Normal SalineSC injection, Part 1 single dose and Part 2 multiple dose | Drug | Placebo |
Primary outcome
Safety based on vitals signs
Time frame: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
Safety based on adverse events
Time frame: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
2 secondary outcomes
Pharmacokinetics
Time frame: Daily on dosing days Assessed during Part 1 for approximately 10 days and during Part 2 for approximately 24 days
Safety based on cardiovascular effects
Time frame: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2012.
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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 and Normal Saline 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 26 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: finished, as of the sponsor's last update on 11 Jun 2012. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety based on vitals signs (time frame: Daily throughout the study Assessed for approximately 8 days following each single dose in Part 1, and for approximately 24 days following each daily dose in Part 2). The study lists 2 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 11 Jun 2012.