Parkinson DiseasePhase 1NCT03710707
Study to Evaluate DNL201 in Subjects With Parkinson's Disease
A Phase 1 study testing DNL201 in people with Parkinson Disease, run by Denali Therapeutics Inc. at 7 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL201 in subjects with Parkinson's disease. Read more about this study
- Who is it for?
- Ages 30 to 80 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- DNL201
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 29 participants
- Where
- 7 sitesUnited States
- Study dates
- 4 Dec 2018to 6 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 30 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Body mass index (BMI) between 18 and 35.0 kg/m2, inclusive
- Clinical diagnosis of Parkinson's disease meeting UK Brain Bank criteria and H\&Y Stage I, II, or III.
- sPD subgroup without a LRRK2 mutation; PD LRRK2 subgroup with LRRK2 mutation
- Screening dopamine transporter (DAT) SPECT scan with a DAT deficit consistent with Parkinson's disease
- Able to hold Parkinson's disease medications 8 hours (overnight) prior to specific study assessments
Who cannot join
- Any history of clinically significant asthma, chronic obstructive pulmonary disease, or emphysema within 5 years of screening, or other clinically significant pulmonary disease within 6 months of screening
- Abnormal Vitals including Respiratory Rate, Body Temperature, and Blood Pressure
- Pulmonary Function Tests (PFTs) (FVC <60% predicted, FEV1 <50% predicted, FEV1:FVC ratio <0.6, DLCO <70% predicted)
- Clinically significant neurologic disorder other than Parkinson's disease, including history of stroke, cognitive impairment, seizure within 5 years of screening, or head trauma with loss of consciousness within 6 months of screening
- Montreal Cognitive Assessment (MoCA) score of <24 at screening
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Clinical Site(s)
Aurora, Colorado, 80045
Clinical Site(s)
Farmington Hills, Michigan, 48334
Clinical Site(s)
Long Beach, California, 90808
Clinical Site(s)
Miami, Florida, 33143
Clinical Site(s)
Orlando, Florida, 32806
Clinical Site(s)
Philadelphia, Pennsylvania, 19104
Clinical Site(s)
Spokane, Washington, 99202
About this study
Official title: A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Determine the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL201 in Subjects With Parkinson's Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL201 in subjects with Parkinson's disease.
- Conditions
- Parkinson Disease
- Being tested
- DNL201, Placebo
- Keywords
- LRRK2, Parkinson's Disease, Movement Disorders
- Medical subject headings
- Parkinson Disease, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DNL201Drug
Oral repeating dose
PlaceboDrug
Oral repeating dose
| Study group | Role | What they receive |
|---|---|---|
| DNL201 low dose | Experimental | Not described Receives: Drug: DNL201 |
| DNL201 high dose | Experimental | Not described Receives: Drug: DNL201 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Randomization to Day 42
Number of Subjects with laboratory test abnormalities
Measured: Randomization to Day 42
Number of Subjects with vital sign abnormalities
Measured: Randomization to Day 42
Number of Subjects with electrocardiogram (ECG) abnormalities
Measured: Randomization to Day 42
Number of Subjects with clinically significant neurological examination abnormalities
Measured: Randomization to Day 42
Secondary outcomes (7)
Pharmacokinetic measure of maximum observed plasma concentration (Cmax) of DNL201
Measured: Randomization to Day 28
Pharmacokinetic measure of time to reach maximum observed plasma concentration (Tmax) of DNL201
Measured: Randomization to Day 28
Pharmacokinetic measure of trough plasma observed concentration (Ctrough) of DNL201
Measured: Randomization to Day 28
Pharmacokinetic measure of area under the plasma drug concentration-time curve (AUC) of DNL201
Measured: Randomization to Day 28
Pharmacokinetic measure of CSF concentrations of DNL201
Measured: Randomization to Day 28
Pharmacodynamic measure of pS935 in whole blood and/or PBMCs
Measured: Randomization to Day 28
Pharmacodynamic measure of pRab10 in PBMCs
Measured: Randomization to Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2018
- Study start4 Dec 2018
- Primary completion6 Dec 2019
- Study completion6 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Parkinson DiseaseStudies 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Denali Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03710707
- Sponsor's protocol ID
- DNLI-B-0002
- Status last verified
- Jan 2020
- First submitted
- 15 Oct 2018
- Last updated
- 13 Jan 2020
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Maloney MT, Wang X, Ghosh R, Andrews SV, Maciuca R, Masoud ST, Agam M, Caprioli RM, Astarita G, Bondar VV, Chen J, Chiu CL, Davis SS, Ho AC, Nguyen HN, Propson NE, Reyzer ML, Davis OB, Deen MC, Zhu S, Di Paolo G, Vocadlo DJ, Estrada AA, de Vicente J, Lewcock JW, Arguello A, Suh JH, Huntwork-Rodriguez S, Henry AG. LRRK2 kinase activity regulates Parkinson's disease-relevant lipids at the lysosome. Mol Neurodegener. 2025 Aug 6;20(1):89. doi: 10.1186/s13024-025-00880-7.
PubMed 40770658Joshi D, Kulkarni M, Parekh P, Shah S, Greig NH, Acharya S. Targeting protein kinases in Parkinson's disease: the emerging role of phytoconstituents. Nutr Neurosci. 2025 Dec;28(12):1532-1563. doi: 10.1080/1028415X.2025.2531356. Epub 2025 Jul 18.
PubMed 40680102
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Determine the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL201 in Subjects With Parkinson's Disease
- ClinicalTrials.gov ID
- NCT03710707
- Acronym / sponsor ID
- DNLI-B-0002
- Conditions
- Parkinson Disease
- Keywords
- LRRK2, Parkinson's Disease, Movement Disorders
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Denali Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Dec 2018 (actual)
- Primary completion
- 6 Dec 2019 (actual)
- Study completion
- 6 Dec 2019 (actual)
- First posted
- 18 Oct 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.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL201 in subjects with Parkinson's disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| DNL201 low dose | Experimental | Drug: DNL201 |
| DNL201 high dose | Experimental | Drug: DNL201 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 30 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Body mass index (BMI) between 18 and 35.0 kg/m2, inclusive
- Clinical diagnosis of Parkinson's disease meeting UK Brain Bank criteria and H\&Y Stage I, II, or III.
- sPD subgroup without a LRRK2 mutation; PD LRRK2 subgroup with LRRK2 mutation
- Screening dopamine transporter (DAT) SPECT scan with a DAT deficit consistent with Parkinson's disease
- Able to hold Parkinson's disease medications 8 hours (overnight) prior to specific study assessments
Key exclusion criteria (5 listed)
- Any history of clinically significant asthma, chronic obstructive pulmonary disease, or emphysema within 5 years of screening, or other clinically significant pulmonary disease within 6 months of screening
- Abnormal Vitals including Respiratory Rate, Body Temperature, and Blood Pressure
- Pulmonary Function Tests (PFTs) (FVC <60% predicted, FEV1 <50% predicted, FEV1:FVC ratio <0.6, DLCO <70% predicted)
- Clinically significant neurologic disorder other than Parkinson's disease, including history of stroke, cognitive impairment, seizure within 5 years of screening, or head trauma with loss of consciousness within 6 months of screening
- Montreal Cognitive Assessment (MoCA) score of <24 at screening
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 |
|---|---|---|
| DNL201Oral repeating dose | Drug | DNL201 high dose, DNL201 low dose |
| PlaceboOral repeating dose | Drug | Placebo |
Primary outcome
Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Randomization to Day 42
Number of Subjects with laboratory test abnormalities
Time frame: Randomization to Day 42
Number of Subjects with vital sign abnormalities
Time frame: Randomization to Day 42
Number of Subjects with electrocardiogram (ECG) abnormalities
Time frame: Randomization to Day 42
Number of Subjects with clinically significant neurological examination abnormalities
Time frame: Randomization to Day 42
7 secondary outcomes
Pharmacokinetic measure of maximum observed plasma concentration (Cmax) of DNL201
Time frame: Randomization to Day 28
Pharmacokinetic measure of time to reach maximum observed plasma concentration (Tmax) of DNL201
Time frame: Randomization to Day 28
Pharmacokinetic measure of trough plasma observed concentration (Ctrough) of DNL201
Time frame: Randomization to Day 28
Pharmacokinetic measure of area under the plasma drug concentration-time curve (AUC) of DNL201
Time frame: Randomization to Day 28
Pharmacokinetic measure of CSF concentrations of DNL201
Time frame: Randomization to Day 28
Pharmacodynamic measure of pS935 in whole blood and/or PBMCs
Time frame: Randomization to Day 28
Pharmacodynamic measure of pRab10 in PBMCs
Time frame: Randomization to Day 28
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 13 Jan 2020.
- Derived from this study: PubMed 40770658Maloney MT, Wang X, Ghosh R, Andrews SV, Maciuca R, Masoud ST, Agam M, Caprioli RM, Astarita G, Bondar VV, Chen J, Chiu CL, Davis SS, Ho AC, Nguyen HN, Propson NE, Reyzer ML, Davis OB, Deen MC, Zhu S, Di Paolo G, Vocadlo DJ, Estrada AA, de Vicente J, Lewcock JW, Arguello A, Suh JH, Huntwork-Rodriguez S, Henry AG. LRRK2 kinase activity regulates Parkinson's disease-relevant lipids at the lysosome. Mol Neurodegener. 2025 Aug 6;20(1):89. doi: 10.1186/s13024-025-00880-7.
- Derived from this study: PubMed 40680102Joshi D, Kulkarni M, Parekh P, Shah S, Greig NH, Acharya S. Targeting protein kinases in Parkinson's disease: the emerging role of phytoconstituents. Nutr Neurosci. 2025 Dec;28(12):1532-1563. doi: 10.1080/1028415X.2025.2531356. Epub 2025 Jul 18.
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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.
7 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 DNL201 in people with Parkinson Disease, run by Denali Therapeutics Inc. at 7 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 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 13 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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. 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?
Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Randomization to Day 42). The study lists 5 primary outcomes in all. 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 NCT03710707, which also lists 2 linked publications.
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 13 Jan 2020.