Amyotrophic Lateral SclerosisPhase 1NCT01449578
Dexpramipexole SAD/MAD Study
A Phase 1 study testing Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 1 site in United States.
Trial summary
This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Dexpramipexole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2011to Jan 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 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Must give written informed consent.
- Adult males/females aged 18 to 55 years inclusive and between 19 and 30 kg/m2 body mass index (BMI), inclusive at screening.
- Subjects who are healthy as determined by prestudy medical history, physical examination and 12-lead ECG.
- Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.
- Normal systemic blood pressure defined as a systolic blood pressure of 90 to 140 mmHg and a diastolic blood pressure of 50 to 90 mmHg.
Who cannot join
- History of cardiovascular disease (e.g., hypertension, arrhythmia, heart failure, Long QT Syndrome, or other conditions/diseases causing prolongation of the QT/QTc interval).
- A prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval >450 ms before study treatment administration) at screening, admission or pre-dose on Day 1.
- Any clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening, admission or pre-dose on Day 1.
- Prior exposure to dexpramipexole.
- Treatment with pramipexole or any dopamine agonist within 1 year.
- Treatment with another investigational drug or approved therapy for investigational use within 30 days, or 5 half-lives (whichever is longer), or in follow up for any other drug, biologic, or device study.
- Currently active infection or serious infection (e.g., pneumonia, septicemia) within the 2 months prior to Day -2 as determined by the Investigator.
- Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Blinded, Placebo-Controlled Ascending Dose Study of the Safety and Pharmacokinetics of Dexpramipexole in Healthy Volunteers
This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
Read the detailed description
Preclinical and clinical data to date support the exploration of doses of dexpramipexole higher than 150 mg twice daily for their effectiveness in slowing the progression of ALS. Exploration of doses of dexpramipexole higher than 150 mg twice daily is justified by preclinical and clinical data that suggest that higher doses could potentially be more effective in slowing the progression of ALS than the dose of dexpramipexole currently being explored in Phase 3 studies (150 mg twice daily).
This is a Phase 1, single-center, blinded, randomized, placebo controlled, ascending-dose study consisting of 2 parts; Part A (single-ascending dose [SAD]) and Part B (multiple ascending dose [MAD]). The study will explore safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Dexpramipexole, Dexpramipexole Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexpramipexoleDrug
Also called BIIB050
Oral Tablet at varying doses
Dexpramipexole PlaceboDrug
Oral tablet at varying doses
| Study group | Role | What they receive |
|---|---|---|
| Part A, Treatment 1 | Experimental | Dexpramipexole single dose (SAD Dose 1) Receives: Drug: Dexpramipexole |
| Part A, Treatment 1 placebo | Placebo comparator | Dexpramipexole single dose placebo (SAD Dose 1) Receives: Drug: Dexpramipexole Placebo |
| Part A, Treatment 2 | Experimental | Dexpramipexole single dose (SAD Dose 2) Receives: Drug: Dexpramipexole |
| Part A, Treatment 2 placebo | Placebo comparator | Dexpramipexole single dose placebo (SAD Dose 2) Receives: Drug: Dexpramipexole Placebo |
| Part A, Treatment 3 | Experimental | Dexpramipexole single dose (SAD Dose 3) Receives: Drug: Dexpramipexole |
| Part A, Treatment 3 placebo | Placebo comparator | Dexpramipexole single dose placebo (SAD Dose 3) Receives: Drug: Dexpramipexole Placebo |
| Part B, Treatment 1 | Experimental | Dexpramipexole multiple dose (MAD Dose 1) Receives: Drug: Dexpramipexole |
| Part B, Treatment 1 placebo | Placebo comparator | Dexpramipexole multiple dose placebo (MAD Dose 1) Receives: Drug: Dexpramipexole Placebo |
| Part B, Treatment 2 | Experimental | Dexpramipexole multiple dose (MAD Dose 2) Receives: Drug: Dexpramipexole |
| Part B, Treatment 2 placebo | Placebo comparator | Dexpramipexole multiple dose placebo (MAD Dose 2) Receives: Drug: Dexpramipexole Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability based on medical review of adverse events & results of vital sign measurements, electrocardiogram (ECGs), physical examinations and clinical laboratory tests.
Measured: Change from baseline to 11 Days.
Secondary outcomes (1)
Dexpramipexole pharmacokinetics time frame in plasma
Measured: pre-dose and 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2011 (estimated)
- Study startNov 2011
- Primary completionJan 2012
- Study completionJan 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Knopp Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01449578
- Sponsor's protocol ID
- 223HV103
- Status last verified
- Nov 2014
- First submitted
- 22 Sep 2011
- Last updated
- 25 Nov 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Blinded, Placebo-Controlled Ascending Dose Study of the Safety and Pharmacokinetics of Dexpramipexole in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT01449578
- Acronym / sponsor ID
- 223HV103
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Knopp BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Nov 2011
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 10 Oct 2011 (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.
This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
Detailed description
Preclinical and clinical data to date support the exploration of doses of dexpramipexole higher than 150 mg twice daily for their effectiveness in slowing the progression of ALS. Exploration of doses of dexpramipexole higher than 150 mg twice daily is justified by preclinical and clinical data that suggest that higher doses could potentially be more effective in slowing the progression of ALS than the dose of dexpramipexole currently being explored in Phase 3 studies (150 mg twice daily).
This is a Phase 1, single-center, blinded, randomized, placebo controlled, ascending-dose study consisting of 2 parts; Part A (single-ascending dose [SAD]) and Part B (multiple ascending dose [MAD]). The study will explore safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 10
| Arm | Type | Receives |
|---|---|---|
| Part A, Treatment 1 | Experimental | Drug: DexpramipexoleDexpramipexole single dose (SAD Dose 1) |
| Part A, Treatment 1 placebo | Placebo comparator | Drug: Dexpramipexole PlaceboDexpramipexole single dose placebo (SAD Dose 1) |
| Part A, Treatment 2 | Experimental | Drug: DexpramipexoleDexpramipexole single dose (SAD Dose 2) |
| Part A, Treatment 2 placebo | Placebo comparator | Drug: Dexpramipexole PlaceboDexpramipexole single dose placebo (SAD Dose 2) |
| Part A, Treatment 3 | Experimental | Drug: DexpramipexoleDexpramipexole single dose (SAD Dose 3) |
| Part A, Treatment 3 placebo | Placebo comparator | Drug: Dexpramipexole PlaceboDexpramipexole single dose placebo (SAD Dose 3) |
| Part B, Treatment 1 | Experimental | Drug: DexpramipexoleDexpramipexole multiple dose (MAD Dose 1) |
| Part B, Treatment 1 placebo | Placebo comparator | Drug: Dexpramipexole PlaceboDexpramipexole multiple dose placebo (MAD Dose 1) |
| Part B, Treatment 2 | Experimental | Drug: DexpramipexoleDexpramipexole multiple dose (MAD Dose 2) |
| Part B, Treatment 2 placebo | Placebo comparator | Drug: Dexpramipexole PlaceboDexpramipexole multiple dose placebo (MAD Dose 2) |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Must give written informed consent.
- Adult males/females aged 18 to 55 years inclusive and between 19 and 30 kg/m2 body mass index (BMI), inclusive at screening.
- Subjects who are healthy as determined by prestudy medical history, physical examination and 12-lead ECG.
- Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.
- Normal systemic blood pressure defined as a systolic blood pressure of 90 to 140 mmHg and a diastolic blood pressure of 50 to 90 mmHg.
Key exclusion criteria (8 listed)
- History of cardiovascular disease (e.g., hypertension, arrhythmia, heart failure, Long QT Syndrome, or other conditions/diseases causing prolongation of the QT/QTc interval).
- A prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval >450 ms before study treatment administration) at screening, admission or pre-dose on Day 1.
- Any clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening, admission or pre-dose on Day 1.
- Prior exposure to dexpramipexole.
- Treatment with pramipexole or any dopamine agonist within 1 year.
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 |
|---|---|---|
| DexpramipexoleAlso: BIIB050Oral Tablet at varying doses | Drug | Part A, Treatment 1, Part A, Treatment 2, Part A, Treatment 3, Part B, Treatment 1, Part B, Treatment 2 |
| Dexpramipexole PlaceboOral tablet at varying doses | Drug | Part A, Treatment 1 placebo, Part A, Treatment 2 placebo, Part A, Treatment 3 placebo, Part B, Treatment 1 placebo, Part B, Treatment 2 placebo |
Primary outcome
Safety and tolerability based on medical review of adverse events & results of vital sign measurements, electrocardiogram (ECGs), physical examinations and clinical laboratory tests.
Time frame: Change from baseline to 11 Days.
1 secondary outcome
Dexpramipexole pharmacokinetics time frame in plasma
Time frame: pre-dose and 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose
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 25 Nov 2014.
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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 Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences 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 5 inclusion and 8 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 25 Nov 2014. 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 and tolerability based on medical review of adverse events & results of vital sign measurements, electrocardiogram (ECGs), physical examinations and clinical laboratory tests. (time frame: Change from baseline to 11 Days.). 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 25 Nov 2014.