Amyotrophic Lateral SclerosisPhase 1NCT01424163
Dexpramipexole Japanese PK Study
A Phase 1 study testing Single dose reduced, Single dose standard and Multiple Dose in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 1 site in United Kingdom.
Trial summary
This is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Single dose reduced, Single dose standard and Multiple Dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Aug 2011to Feb 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 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subjects who are able and willing to give written informed consent.
- Adult Japanese and Caucasian males/females aged 18 to 60 years inclusive and between 18 and 30 kg/m2 body mass index (BMI), inclusive.
- Male and female subjects will be enrolled on the study. Male subjects and female 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
- Japanese subjects must be born in Japan and have both parents and four grandparents of Japanese descent.
- Japanese subjects must have lived outside of Japan for no more than 5 years.
- Japanese subjects must not have significant changes with regard to diet; i.e., their diet must not have significantly changed since leaving Japan.
- Caucasian subjects will be matched individually (on a 1:1 basis) to Japanese subjects with respect to gender and age, and if possible BMI.
Who cannot join
- Subjects who do not conform to the above inclusion criteria.
- Female subjects who are pregnant, trying to become pregnant or lactating.
- Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
- Subjects who have a clinically relevant surgical history.
- Subjects who have previously received dexpramipexole or pramipexole.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single and Multiple Dose, Open-Label Study of the Pharmacokinetics, Safety, and Tolerability of Dexpramipexole (BIIB050) in Healthy Japanese and Caucasian Subjects
This is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects.
Read the detailed description
The study is designed to evaluate the influence of ethnic factors on dexpramipexole safety, tolerability, and PK. Subjects will be admitted to the clinical unit on Day -1 (the day prior to first dosing) and will remain in the clinical unit under observation until discharge. All subjects will have a final follow up visit. Whilst resident in the clinic, subjects will receive 4 treatments: The first 3 treatments comprise Part A of the study and the final treatment group comprises Part B.
Part A:
Treatment 1: Dose 1 (reduced) of dexpramipexole; a single dose Treatment 2: Dose 2 (standard) dexpramipexole; a single dose Treatment 3: Dose 3 (standard) dexpramipexole; 5 doses administered at 12 hour intervals Part B: Dose 4 (standard) dexpramipexole; 5 doses administered at 12 hour intervals There will be a minimum washout of 3 days between treatments.
For all subjects, prior to proceeding to the next treatment group, safety and tolerability data will be reviewed.
Caucasian subjects will be matched individually (on a 1:1 basis) to Japanese subjects with respect to gender and age and if possible BMI.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Single dose reduced, Single dose standard, Multiple Dose
- 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.
Single dose reducedDrug
Treatment 1 (Part A)
Single dose standardDrug
Treatment 2 (Part A)
Multiple DoseDrug
Treatment 3 (Part A)
Multiple DoseDrug
Part B
| Study group | Role | What they receive |
|---|---|---|
| Treatment 1 (Part A) | Experimental | Dexpramipexole single dose (reduced dose) Receives: Drug: Single dose reduced |
| Treatment 2 (Part A) | Experimental | Dexpramipexole single dose (Standard dose) Receives: Drug: Single dose standard |
| Treatment 3 (Part A) | Experimental | Dexpramipexole multiple dosing Receives: Drug: Multiple Dose |
| Treatment for Part B | Experimental | Dexpramipexole multiple dosing Receives: Drug: Multiple Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cmax of dexpramipexole
Measured: pre-dose and at 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing on Days 1 and 5 in Treatments 1 and 2, and after the fifth dose on Day 11 in Treatment 3 for Parts A & B.
AUC of dexpramipexole
Measured: pre-dose and at 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing on Days 1 and 5 in Treatments 1 and 2, and after the fifth dose on Day 11 in Treatment 3 for Parts A & B.
Secondary outcomes (4)
Changes in clinical laboratory tests
Measured: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
ECG changes
Measured: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
Vital Sign changes
Measured: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
Adverse Event monitoring
Measured: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2011 (estimated)
- Study startAug 2011
- Primary completionFeb 2012
- Study completionFeb 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
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Knopp Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Biogen
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01424163
- Sponsor's protocol ID
- 223HV101
- Status last verified
- Nov 2014
- First submitted
- 28 Jul 2011
- Last updated
- 22 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single and Multiple Dose, Open-Label Study of the Pharmacokinetics, Safety, and Tolerability of Dexpramipexole (BIIB050) in Healthy Japanese and Caucasian Subjects
- ClinicalTrials.gov ID
- NCT01424163
- Acronym / sponsor ID
- 223HV101
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Knopp BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Biogen
- Study start
- Aug 2011
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 26 Aug 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 is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects.
Detailed description
The study is designed to evaluate the influence of ethnic factors on dexpramipexole safety, tolerability, and PK. Subjects will be admitted to the clinical unit on Day -1 (the day prior to first dosing) and will remain in the clinical unit under observation until discharge. All subjects will have a final follow up visit. Whilst resident in the clinic, subjects will receive 4 treatments: The first 3 treatments comprise Part A of the study and the final treatment group comprises Part B.
Part A:
Treatment 1: Dose 1 (reduced) of dexpramipexole; a single dose Treatment 2: Dose 2 (standard) dexpramipexole; a single dose Treatment 3: Dose 3 (standard) dexpramipexole; 5 doses administered at 12 hour intervals Part B: Dose 4 (standard) dexpramipexole; 5 doses administered at 12 hour intervals There will be a minimum washout of 3 days between treatments.
For all subjects, prior to proceeding to the next treatment group, safety and tolerability data will be reviewed.
Caucasian subjects will be matched individually (on a 1:1 basis) to Japanese subjects with respect to gender and age and if possible BMI.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Treatment 1 (Part A) | Experimental | Drug: Single dose reducedDexpramipexole single dose (reduced dose) |
| Treatment 2 (Part A) | Experimental | Drug: Single dose standardDexpramipexole single dose (Standard dose) |
| Treatment 3 (Part A) | Experimental | Drug: Multiple DoseDexpramipexole multiple dosing |
| Treatment for Part B | Experimental | Drug: Multiple DoseDexpramipexole multiple dosing |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Subjects who are able and willing to give written informed consent.
- Adult Japanese and Caucasian males/females aged 18 to 60 years inclusive and between 18 and 30 kg/m2 body mass index (BMI), inclusive.
- Male and female subjects will be enrolled on the study. Male subjects and female 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
- Japanese subjects must be born in Japan and have both parents and four grandparents of Japanese descent.
- Japanese subjects must have lived outside of Japan for no more than 5 years.
Key exclusion criteria (5 listed)
- Subjects who do not conform to the above inclusion criteria.
- Female subjects who are pregnant, trying to become pregnant or lactating.
- Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
- Subjects who have a clinically relevant surgical history.
- Subjects who have previously received dexpramipexole or pramipexole.
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 |
|---|---|---|
| Single dose reducedTreatment 1 (Part A) | Drug | Treatment 1 (Part A) |
| Single dose standardTreatment 2 (Part A) | Drug | Treatment 2 (Part A) |
| Multiple DoseTreatment 3 (Part A) | Drug | Treatment 3 (Part A) |
| Multiple DosePart B | Drug | Treatment for Part B |
Primary outcome
Cmax of dexpramipexole
Time frame: pre-dose and at 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing on Days 1 and 5 in Treatments 1 and 2, and after the fifth dose on Day 11 in Treatment 3 for Parts A & B.
AUC of dexpramipexole
Time frame: pre-dose and at 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing on Days 1 and 5 in Treatments 1 and 2, and after the fifth dose on Day 11 in Treatment 3 for Parts A & B.
4 secondary outcomes
Changes in clinical laboratory tests
Time frame: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
ECG changes
Time frame: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
Vital Sign changes
Time frame: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last dose)
Adverse Event monitoring
Time frame: pre-dose to 72 hrs post-dose in each dose group and again at follow-up (14 hrs post last 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 22 Jun 2021.
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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 Single dose reduced, Single dose standard and Multiple Dose in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 1 site in United Kingdom.
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 7 inclusion and 5 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 22 Jun 2021. 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 Kingdom. 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 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?
Cmax of dexpramipexole (time frame: pre-dose and at 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing on Days 1 and 5 in Treatments 1 and 2, and after the fifth dose on Day 11 in Treatment 3 for Parts A & B.). 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 22 Jun 2021.