Amyotrophic Lateral SclerosisPhase 1NCT01424176
Dexpramipexole Renal PK Study
A Phase 1 study testing Dexpramipexole (dose 1) and Dexpramipexole (dose 2) in people with Amyotrophic Lateral Sclerosis and Renal Insufficiency, run by Knopp Biosciences at 2 sites in United States.
Trial summary
This is a multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Renal Insufficiency
- What is being tested?
- Dexpramipexole (dose 1) and Dexpramipexole (dose 2)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult males/females aged 18 to 75 years inclusive and between 19 and 36 kg/m2 inclusive BMI.
- Subjects with renal impairment must have stable renal disease (i.e., no change in disease status within the 28 days prior to dosing) as determined by the Investigator with laboratory and clinical findings that support the diagnosis of renal impairment.
- Subjects with renal impairment (excluding ESRD subjects), must have 2 separate estimates of creatinine clearance that are within 25% of each other, obtained >5 days apart, but not >6 months apart
- Subjects must have a GFR (estimated GFR; as defined by estimation of creatinine clearance using the MDRD formula) of ≥80 (healthy volunteers), between 50 and 79 (mild renal impairment), between 30 and 49 (moderate renal impairment), or <30 (severe renal impairment), or must require dialysis ≤3 times a week (ESRD).
- Healthy volunteers must be matched to renally impaired subjects for age (± 10 years), gender, and if possible BMI (± 20%).
Who cannot join
- Healthy volunteers who have received prescription medication within the 14 days prior to dosing (except for birth control).
- Renally impaired subjects who have received prescription medication within the 14 days prior to dosing (except for birth control and medications taken at a stable dose for underlying conditions, as determined by the Investigator).
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
Brooklyn Center, Minnesota, 55430
Research Site
Orlando, Florida, 32809
About this study
Official title: A Multicenter, Open-Label, Single-dose, Pharmacokinetic and Safety Study of Dexpramipexole (BIIB050) in Healthy Subjects and Subjects With Renal Impairment
This is a multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment.
Read the detailed description
Dexpramipexole (BIIB050, KNS-760704; (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydrochloride monohydrate) is a synthetic amino-benzothiazole. Data from multiple in vitro and in vivo assays have suggested that dexpramipexole is neuroprotective. It is being investigated for the treatment ALS.
As dexpramipexole is principally eliminated from the body by the kidneys a single oral dose of dexpramipexole will be administered to subjects with various stages of renal impariment comprising the following categories: mild, moderate, severe and ESRD subjects. Healthy volunteers will be matched to each catergory of renal impaired subjects.
- Conditions
- Amyotrophic Lateral Sclerosis, Renal Insufficiency
- Being tested
- Dexpramipexole (dose 1), Dexpramipexole (dose 2)
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Renal Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexpramipexole (dose 1)Drug
Also called BIIB050
Dexpramipexole (dose 2)Drug
Also called BIIB050
| Study group | Role | What they receive |
|---|---|---|
| Dexpramipexole (dose 1) | Experimental | Subjects with mild or moderate renal impairment. Receives: Drug: Dexpramipexole (dose 1) |
| Dexpramipexole (dose 2) | Experimental | Subjects with severe renal impairment and end stage renal disease (ESRD). Receives: Drug: Dexpramipexole (dose 2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
AUC after single dose of dexpramipexole
Measured: pre dose and at 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing in all subjects, and in addition at 96, 120, and 144 hours after dosing in subjects with severe renal impairment and ESRD.
Cmax after single dose of dexpramipexole
Measured: pre dose and at 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing in all subjects, and in addition at 96, 120, and 144 hours after dosing in subjects with severe renal impairment and ESRD.
Secondary outcomes (4)
Mointoring of Clinical Laboratory tests
Measured: pre-144 hours post dose
ECG Monitoring
Measured: pre-144 hrs post dose
Vital Sign monitoring
Measured: pre-144 hrs post dose
AE monitoring
Measured: pre-144 hrs post 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 startJul 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
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 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
- NCT01424176
- Sponsor's protocol ID
- 223RI101
- Status last verified
- Nov 2014
- First submitted
- 23 Jun 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 Multicenter, Open-Label, Single-dose, Pharmacokinetic and Safety Study of Dexpramipexole (BIIB050) in Healthy Subjects and Subjects With Renal Impairment
- ClinicalTrials.gov ID
- NCT01424176
- Acronym / sponsor ID
- 223RI101
- Conditions
- Amyotrophic Lateral Sclerosis, Renal Insufficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Knopp BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Jul 2011
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 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 multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment.
Detailed description
Dexpramipexole (BIIB050, KNS-760704; (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydrochloride monohydrate) is a synthetic amino-benzothiazole. Data from multiple in vitro and in vivo assays have suggested that dexpramipexole is neuroprotective. It is being investigated for the treatment ALS.
As dexpramipexole is principally eliminated from the body by the kidneys a single oral dose of dexpramipexole will be administered to subjects with various stages of renal impariment comprising the following categories: mild, moderate, severe and ESRD subjects. Healthy volunteers will be matched to each catergory of renal impaired subjects.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dexpramipexole (dose 1) | Experimental | Drug: Dexpramipexole (dose 1)Subjects with mild or moderate renal impairment. |
| Dexpramipexole (dose 2) | Experimental | Drug: Dexpramipexole (dose 2)Subjects with severe renal impairment and end stage renal disease (ESRD). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adult males/females aged 18 to 75 years inclusive and between 19 and 36 kg/m2 inclusive BMI.
- Subjects with renal impairment must have stable renal disease (i.e., no change in disease status within the 28 days prior to dosing) as determined by the Investigator with laboratory and clinical findings that support the diagnosis of renal impairment.
- Subjects with renal impairment (excluding ESRD subjects), must have 2 separate estimates of creatinine clearance that are within 25% of each other, obtained >5 days apart, but not >6 months apart
- Subjects must have a GFR (estimated GFR; as defined by estimation of creatinine clearance using the MDRD formula) of ≥80 (healthy volunteers), between 50 and 79 (mild renal impairment), between 30 and 49 (moderate renal impairment), or <30 (severe renal impairment), or must require dialysis ≤3 times a week (ESRD).
- Healthy volunteers must be matched to renally impaired subjects for age (± 10 years), gender, and if possible BMI (± 20%).
Key exclusion criteria (2 listed)
- Healthy volunteers who have received prescription medication within the 14 days prior to dosing (except for birth control).
- Renally impaired subjects who have received prescription medication within the 14 days prior to dosing (except for birth control and medications taken at a stable dose for underlying conditions, as determined by the Investigator).
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 |
|---|---|---|
| Dexpramipexole (dose 1)Also: BIIB050 | Drug | Dexpramipexole (dose 1) |
| Dexpramipexole (dose 2)Also: BIIB050 | Drug | Dexpramipexole (dose 2) |
Primary outcome
AUC after single dose of dexpramipexole
Time frame: pre dose and at 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing in all subjects, and in addition at 96, 120, and 144 hours after dosing in subjects with severe renal impairment and ESRD.
Cmax after single dose of dexpramipexole
Time frame: pre dose and at 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing in all subjects, and in addition at 96, 120, and 144 hours after dosing in subjects with severe renal impairment and ESRD.
4 secondary outcomes
Mointoring of Clinical Laboratory tests
Time frame: pre-144 hours post dose
ECG Monitoring
Time frame: pre-144 hrs post dose
Vital Sign monitoring
Time frame: pre-144 hrs post dose
AE monitoring
Time frame: pre-144 hrs 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.
2 sites 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 (dose 1) and Dexpramipexole (dose 2) in people with Amyotrophic Lateral Sclerosis and Renal Insufficiency, run by Knopp Biosciences at 2 sites 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 2 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 2 sites 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 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?
AUC after single dose of dexpramipexole (time frame: pre dose and at 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours after dosing in all subjects, and in addition at 96, 120, and 144 hours after dosing in subjects with severe renal impairment and ESRD.). 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 25 Nov 2014.