HealthyPhase 1NCT03636204
A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
A Phase 1 study testing AL001 in people with Healthy and Frontotemporal Dementia, run by Alector Inc. at 8 sites in 3 countries.
Trial summary
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy, Frontotemporal Dementia
- What is being tested?
- AL001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 8 sitesin 3 countries
- Study dates
- 14 Sep 2018to 31 Dec 2019
- 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 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- BMI 18.0-35.0 kg/m2
- 45-120 kg, inclusive
- At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up.
- Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit.
- In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator.
- Willingness and able to comply with the study protocol, in the investigator's judgement.
Who cannot join
- Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- Positive drug or alcohol at screening and prior to first dose
- History of alcohol abuse or substance abuse
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United States5 sites
University of Alabama
Birmingham, Alabama, 35294
Study site
Orlando, Florida, 32806
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
UCSF
San Francisco, California, 94158
Canada2 sites
Lawson Health Research Institute, St. Joseph's
London, Ontario
Sunnybrook Health Sciences Centre
Toronto
About this study
Official title: A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
Read the detailed description
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.
- Conditions
- Healthy, Frontotemporal Dementia
- Being tested
- AL001, Placebo
- Medical subject headings
- Frontotemporal Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AL001Biological
Active dose of AL001
PlaceboOther
Saline solution administered as a single infusion as palcebo.
| Study group | Role | What they receive |
|---|---|---|
| AL001 | Experimental | Up to six single ascending doses of AL001 Receives: Biological: AL001 |
| Saline Solution | Placebo comparator | Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
Measured: 85 days
Secondary outcomes (3)
Pharmacokinetics (PK) of AL001
Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points
Measured: 85 days
Maximum plasma concentration (Cmax) for AL001
Evaluate Cmax for serum and CSF concentration of AL001 at specified time points
Measured: 85 days
Area under the curve concentration (AUC) for AL001
Evaluate AUC for serum and CSF concentration of AL001 at specified time points
Measured: 85 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2018
- Study start14 Sep 2018
- Primary completion31 Dec 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HealthyStudies 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
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alector Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- George Stoica, Principal investigator, Bioclinica Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03636204
- Sponsor's protocol ID
- AL001-1
- Status last verified
- Jul 2020
- First submitted
- 12 Jul 2018
- Last updated
- 27 Jul 2020
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Kurnellas M, Mitra A, Schwabe T, Paul R, Arrant AE, Roberson ED, Ward M, Yeh F, Long H, Rosenthal A. Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia. J Transl Med. 2023 Jun 15;21(1):387. doi: 10.1186/s12967-023-04251-y.
PubMed 37322482
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
- ClinicalTrials.gov ID
- NCT03636204
- Acronym / sponsor ID
- AL001-1
- Conditions
- Healthy, Frontotemporal Dementia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Alector Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Sep 2018 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 17 Aug 2018 (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.
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
Detailed description
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AL001 | Experimental | Biological: AL001Up to six single ascending doses of AL001 |
| Saline Solution | Placebo comparator | Other: PlaceboSaline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- BMI 18.0-35.0 kg/m2
- 45-120 kg, inclusive
- At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up.
- Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit.
- In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator.
Key exclusion criteria (3 listed)
- Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- Positive drug or alcohol at screening and prior to first dose
- History of alcohol abuse or substance abuse
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 |
|---|---|---|
| AL001Active dose of AL001 | Biological | AL001 |
| PlaceboSaline solution administered as a single infusion as palcebo. | Other | Saline Solution |
Primary outcome
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
Time frame: 85 days
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
3 secondary outcomes
Pharmacokinetics (PK) of AL001
Time frame: 85 days
Maximum plasma concentration (Cmax) for AL001
Time frame: 85 days
Area under the curve concentration (AUC) for AL001
Time frame: 85 days
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 27 Jul 2020.
- Derived from this study: PubMed 37322482Kurnellas M, Mitra A, Schwabe T, Paul R, Arrant AE, Roberson ED, Ward M, Yeh F, Long H, Rosenthal A. Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia. J Transl Med. 2023 Jun 15;21(1):387. doi: 10.1186/s12967-023-04251-y.
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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.
George Stoica principal investigator
Bioclinica Research
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AL001 in people with Healthy and Frontotemporal Dementia, run by Alector Inc. at 8 sites in 3 countries.
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 6 inclusion and 3 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 27 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada, 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. 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?
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs) (time frame: 85 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03636204, which also lists 1 linked publication.
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 27 Jul 2020.