Safety and Tolerability in Healthy VolunteersPhase 1NLY01-H1NCT03672604
A Phase 1 Study to Assess the Safety of NLY01 in Healthy Subjects
A Phase 1 study testing NLY01 in people with Safety and Tolerability in Healthy Volunteers, run by Neuraly, Inc. at 1 site in United States.
Trial summary
This is a Phase 1, first-in-human study designed to assess the safety, tolerability, and pharmacokinetics of NLY01, a PEGylated form of exenatide, in healthy volunteers. NLY01 is being developed as a potential treatment for neurodegenerative disorders including Parkinson's disease. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Safety and Tolerability in Healthy Volunteers
- What is being tested?
- NLY01
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Sep 2018to 30 Jul 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Men or women, 18 to 65 years of age, inclusive.
- BMI ≥ 18.5 and ≤ 32.0 kg/m2 at screening and check-in. BMI = weight (kg)/(height [m])2.
- Women of child-bearing potential must agree to use a medically acceptable method of contraception from screening through 30 days after the final dose of study drug.
- Non-childbearing potential.
- Men who are sexually active and whose partners are women of child-bearing potential must agree to use condoms from screening through 90 days after administration of study drug, and their partners must be willing to use a medically acceptable method of contraception (a barrier method, intrauterine device, or hormonal contraception) from screening through 90 days after administration of the last dose of study drug.
- Men must agree to not donate sperm from screening through 90 days after study drug administration.
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- Subjects must be able to communicate effectively with the study personnel.
- Subjects must be healthy and without clinically significant abnormalities as assessed by review of medical and surgical history, physical examination, vital signs measurement, ECG, and laboratory evaluations conducted at screening and on Day -1 Check-in. A single repeat measurement/test may be performed to confirm vital signs, ECG, and clinical laboratory tests abnormalities (ie, to confirm that a subject is eligible).
- Subjects must be nonsmokers, defined as having abstained from tobacco- or nicotine containing products (eg, cigarettes, chewing tobacco, snuff, nicotine patches, and electronic cigarettes) in the 6 months prior to screening.
- Subjects must be informed of the nature and risks of the study and give written informed consent prior to screening.
Who cannot join
- Positive pregnancy test or is lactating/breastfeeding.
- History or presence of conditions which, in the judgment of the investigator, are known to interfere with the distribution, metabolism, or excretion of drugs.
- History or presence of conditions that may place the subject at increased risk as determined by the investigator.
- History of surgery or major trauma within 12 weeks of screening, or surgery planned during the study.
- History of alcohol abuse, illicit drug use, significant mental illness, physical dependence to any opioid, or any history of drug abuse or addiction within 12 months of screening.
- Use of prescription, OTC drugs (including herbal preparations) within 7 days or 5 half lives (if known), whichever is longer, prior to administration of the first dose of study drug.
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- Has received a vaccination within 14 days prior to administration of the first dose of study drug.
- Has taken other investigational drugs or participated in any clinical study within 60 days or 5 half-lives (if known) of the investigational drug's PK, PD, or biological activity (if known), whichever is longer, prior to administration of the first dose of study drug in this study or is currently participating in another clinical study.
- Prior exposure to exenatide (Byetta® or Bydureon®).
- Significant blood loss (> 450 mL) or has donated 1 or more units of blood or plasma within 6 weeks prior to study randomization.
- History of hypoglycemia.
- History of gastroparesis.
- History of pancreatitis.
- Positive urine results for drugs of abuse, alcohol, or cotinine screen.
- Positive screen for HIV-1 and HIV-2 antibodies, HBsAg, or HCV antibody.
- Clinically significant cardiac changes demonstrated by ECG at screening or Day-1 including:
- QTcF interval > 450 msec
- PR interval ≤ 110 msec or > 240 msec
- Evidence of second- or third-degree atrioventricular block
- Pathological Q-waves (defined as Q-wave > 40 msec or depth greater than 0.5 mV)
- Evidence of ventricular pre-excitation
- Evidence of complete left BBB, incomplete left BBB, complete right BBB
- Intraventricular conduction delay with QRS duration > 120 msec
- Bradycardia (defined as sinus rate < 50 bpm) or tachycardia (defined as sinus rate > 100 bpm)
- Has any of the following abnormal vital signs at screening or Day-1:
- Pulse < 40 or > 100 bpm
- Respiratory rate < 8 or > 20 breaths per minute
- Systolic blood pressure < 95 or > 145 mmHg
- Diastolic blood pressure < 45 or > 90 mmHg
- Serum potassium, chloride, calcium, or sodium outside the normal reference range at screening
- Hepatic transaminases (ALT or AST) > 100 IU/mL at screening.
- Any hematology, chemistry, or urinalysis test results that are clinically significant.
- Any other condition or prior therapy that, in the investigator's opinion, would make the subject unsuitable for the study, or unable or unwilling to comply with the study procedures.
- Unwilling or unlikely to comply with the requirements of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
ICON Early Phase Services/CRU
San Antonio, Texas, 78209
About this study
Official title: A Phase 1, Single- and Multiple-Dose, Double-Blind, Randomized, Placebo-Controlled, Dose-Escalating Study to Assess the Safety, Tolerability, and Pharmacokinetics of NLY01 in Healthy Subjects
This is a Phase 1, first-in-human study designed to assess the safety, tolerability, and pharmacokinetics of NLY01, a PEGylated form of exenatide, in healthy volunteers. NLY01 is being developed as a potential treatment for neurodegenerative disorders including Parkinson's disease. This study is intended to identify the appropriate dose-range for evaluation in Parkinson's disease patients.
Read the detailed description
This is a Phase 1, first-in-human, double-blind, randomized, placebo-controlled, single and multiple-dose study to assess the safety, tolerability, and PK of NLY01, a PEGylated form of the anti-diabetic peptide exenatide, when administered by SC injection in healthy subjects.
In Part A of the study, 5 ascending-dose cohorts will be sequentially enrolled with an evaluation of safety and tolerability prior to each dose-escalation. Subjects in each cohort will be randomized to receive NLY01 or placebo. Each dose escalation and selection of doses for Part B will be conducted with oversight by an independently-chaired Safety Review Committee.
In Part B, subjects will receive once-weekly subcutaneous doses of NLY01 or placebo for 4 weeks. Three ascending-dose cohorts will be sequentially-enrolled with a safety review prior to each dose-escalation. Subjects in all Part B cohorts will receive fixed doses of NLY01 (or placebo) once-weekly for 4 weeks.
- Being tested
- NLY01
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NLY01Drug
Also called Placebo, normal saline
NLY01, a PEGylated form of the anti-diabetic peptide exenatide
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Dose | Placebo comparator | Cohort 1 = 0.25 mg NLY01 Cohort 2 = 0.8 mg NLY01 Cohort 3 = 2.5 mg NLY01 Cohort 4 = 5 mg NLY01 Cohort 5 = 10 mg NLY01
All cohorts include 8 subjects randomized to receive a single dose of NLY01 or placebo (6 active, 2 placebo). Receives: Drug: NLY01 |
| Part B: Multiple Dose | Placebo comparator | In Part B, NLY01 or placebo will be administered once-weekly for 4 doses. There will be 3 sequentially-enrolled, ascending-dose cohorts of 8 subjects (6 active, 2 placebo). Doses in Part B will be a fraction of the maximum tolerated dose (MTD) established in Part A.
Cohort 6 = 15% of the single-dose MTD Cohort 7 = 35% of the single-dose MTD Cohort 8 = 70% of the single-dose MTD Receives: Drug: NLY01 |
| Part C:Multiple Dose | Placebo comparator | In Part C, NLY01 or placebo will be administered once-weekly for 6 doses.
Cohort 10 = 2.5 mg NLY01 Cohort 11 = 5 mg NLY01 Receives: Drug: NLY01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Related Adverse Events
Frequency and intensity of treatment-related adverse events as assessed using the CTCAE v4.03 criteria and the DMID Adult Toxicity Table for GI-related events.
Measured: Day 1 through Day 29 (Part A) or Day 57 (Part B)
Secondary outcomes (2)
Pharmacokinetics of NLY01
Serum concentration-time profiles for NLY01
Measured: Periodic, predose through Day 29 (Part A) or Day 57 (Part B)
Immunogenicity
Serum anti-drug antibody assessment
Measured: Periodic, predose through Day 29 (Part A) or Day 57 (Part B)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2018
- Study start19 Sep 2018
- Primary completion30 Jul 2019
- Study completion30 Jul 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 Safety and Tolerability in Healthy VolunteersStudies 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
- Sequential
- Model description
- A Phase 1, first-in-human, double-blind, randomized, placebo-controlled, single-ascending followed by multiple-dose study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neuraly, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Emanuel DeNoia, MD, Principal investigator, ICON Early Phase Services/CRU
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03672604
- Sponsor's protocol ID
- C-NRALY-PHAS1-M-0000
- Acronym
- NLY01-H1
- Status last verified
- Sep 2019
- First submitted
- 13 Sep 2018
- Last updated
- 30 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: As a Phase 1 safety study, data will be limited to adverse events and PK. This data will be provided in summary form and/or with descriptive statistics. Individual listings are not expected to provide further insight.
Publications
Yun SP, Kam TI, Panicker N, Kim S, Oh Y, Park JS, Kwon SH, Park YJ, Karuppagounder SS, Park H, Kim S, Oh N, Kim NA, Lee S, Brahmachari S, Mao X, Lee JH, Kumar M, An D, Kang SU, Lee Y, Lee KC, Na DH, Kim D, Lee SH, Roschke VV, Liddelow SA, Mari Z, Barres BA, Dawson VL, Lee S, Dawson TM, Ko HS. Block of A1 astrocyte conversion by microglia is neuroprotective in models of Parkinson's disease. Nat Med. 2018 Jul;24(7):931-938. doi: 10.1038/s41591-018-0051-5. Epub 2018 Jun 11.
PubMed 29892066Park EJ, Choi J, Lee KC, Na DH. Emerging PEGylated non-biologic drugs. Expert Opin Emerg Drugs. 2019 Jun;24(2):107-119. doi: 10.1080/14728214.2019.1604684. Epub 2019 Apr 19.
PubMed 30957581
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Single- and Multiple-Dose, Double-Blind, Randomized, Placebo-Controlled, Dose-Escalating Study to Assess the Safety, Tolerability, and Pharmacokinetics of NLY01 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT03672604
- Acronym / sponsor ID
- NLY01-H1 / C-NRALY-PHAS1-M-0000
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Neuraly, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Sep 2018 (actual)
- Primary completion
- 30 Jul 2019 (actual)
- Study completion
- 30 Jul 2019 (actual)
- First posted
- 14 Sep 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.
This is a Phase 1, first-in-human study designed to assess the safety, tolerability, and pharmacokinetics of NLY01, a PEGylated form of exenatide, in healthy volunteers. NLY01 is being developed as a potential treatment for neurodegenerative disorders including Parkinson's disease. This study is intended to identify the appropriate dose-range for evaluation in Parkinson's disease patients.
Detailed description
This is a Phase 1, first-in-human, double-blind, randomized, placebo-controlled, single and multiple-dose study to assess the safety, tolerability, and PK of NLY01, a PEGylated form of the anti-diabetic peptide exenatide, when administered by SC injection in healthy subjects.
In Part A of the study, 5 ascending-dose cohorts will be sequentially enrolled with an evaluation of safety and tolerability prior to each dose-escalation. Subjects in each cohort will be randomized to receive NLY01 or placebo. Each dose escalation and selection of doses for Part B will be conducted with oversight by an independently-chaired Safety Review Committee.
In Part B, subjects will receive once-weekly subcutaneous doses of NLY01 or placebo for 4 weeks. Three ascending-dose cohorts will be sequentially-enrolled with a safety review prior to each dose-escalation. Subjects in all Part B cohorts will receive fixed doses of NLY01 (or placebo) once-weekly for 4 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- A Phase 1, first-in-human, double-blind, randomized, placebo-controlled, single-ascending followed by multiple-dose study
| Arm | Type | Receives |
|---|---|---|
| Part A: Single Dose | Placebo comparator | Drug: NLY01Cohort 1 = 0.25 mg NLY01 Cohort 2 = 0.8 mg NLY01 Cohort 3 = 2.5 mg NLY01 Cohort 4 = 5 mg NLY01 Cohort 5 = 10 mg NLY01 All cohorts include 8 subjects randomized to receive a single dose of NLY01 or placebo (6 active, 2 placebo). |
| Part B: Multiple Dose | Placebo comparator | Drug: NLY01In Part B, NLY01 or placebo will be administered once-weekly for 4 doses. There will be 3 sequentially-enrolled, ascending-dose cohorts of 8 subjects (6 active, 2 placebo). Doses in Part B will be a fraction of the maximum tolerated dose (MTD) established in Part A. Cohort 6 = 15% of the single-dose MTD Cohort 7 = 35% of the single-dose MTD Cohort 8 = 70% of the single-dose MTD |
| Part C:Multiple Dose | Placebo comparator | Drug: NLY01In Part C, NLY01 or placebo will be administered once-weekly for 6 doses. Cohort 10 = 2.5 mg NLY01 Cohort 11 = 5 mg NLY01 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Men or women, 18 to 65 years of age, inclusive.
- BMI ≥ 18.5 and ≤ 32.0 kg/m2 at screening and check-in. BMI = weight (kg)/(height [m])2.
- Women of child-bearing potential must agree to use a medically acceptable method of contraception from screening through 30 days after the final dose of study drug.
- Non-childbearing potential.
- Men who are sexually active and whose partners are women of child-bearing potential must agree to use condoms from screening through 90 days after administration of study drug, and their partners must be willing to use a medically acceptable method of contraception (a barrier method, intrauterine device, or hormonal contraception) from screening through 90 days after administration of the last dose of study drug.
Key exclusion criteria (34 listed)
- Positive pregnancy test or is lactating/breastfeeding.
- History or presence of conditions which, in the judgment of the investigator, are known to interfere with the distribution, metabolism, or excretion of drugs.
- History or presence of conditions that may place the subject at increased risk as determined by the investigator.
- History of surgery or major trauma within 12 weeks of screening, or surgery planned during the study.
- History of alcohol abuse, illicit drug use, significant mental illness, physical dependence to any opioid, or any history of drug abuse or addiction within 12 months of 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 |
|---|---|---|
| NLY01Also: Placebo, normal salineNLY01, a PEGylated form of the anti-diabetic peptide exenatide | Drug | Part A: Single Dose, Part B: Multiple Dose, Part C:Multiple Dose |
Primary outcome
Treatment-Related Adverse Events
Time frame: Day 1 through Day 29 (Part A) or Day 57 (Part B)
Frequency and intensity of treatment-related adverse events as assessed using the CTCAE v4.03 criteria and the DMID Adult Toxicity Table for GI-related events.
2 secondary outcomes
Pharmacokinetics of NLY01
Time frame: Periodic, predose through Day 29 (Part A) or Day 57 (Part B)
Immunogenicity
Time frame: Periodic, predose through Day 29 (Part A) or Day 57 (Part B)
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 30 Sep 2019.
- Background: PubMed 29892066Yun SP, Kam TI, Panicker N, Kim S, Oh Y, Park JS, Kwon SH, Park YJ, Karuppagounder SS, Park H, Kim S, Oh N, Kim NA, Lee S, Brahmachari S, Mao X, Lee JH, Kumar M, An D, Kang SU, Lee Y, Lee KC, Na DH, Kim D, Lee SH, Roschke VV, Liddelow SA, Mari Z, Barres BA, Dawson VL, Lee S, Dawson TM, Ko HS. Block of A1 astrocyte conversion by microglia is neuroprotective in models of Parkinson's disease. Nat Med. 2018 Jul;24(7):931-938. doi: 10.1038/s41591-018-0051-5. Epub 2018 Jun 11.
- Derived from this study: PubMed 30957581Park EJ, Choi J, Lee KC, Na DH. Emerging PEGylated non-biologic drugs. Expert Opin Emerg Drugs. 2019 Jun;24(2):107-119. doi: 10.1080/14728214.2019.1604684. Epub 2019 Apr 19.
- Neuraly, Inc websiteLink provided by the sponsor.
- ICON Early Phase Services - Clinical Trial SiteLink provided by the sponsor.
- Individual participant dataSharing planned: No. As a Phase 1 safety study, data will be limited to adverse events and PK. This data will be provided in summary form and/or with descriptive statistics. Individual listings are not expected to provide further insight.
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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.
Emanuel DeNoia, MD principal investigator
ICON Early Phase Services/CRU
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 NLY01 in people with Safety and Tolerability in Healthy Volunteers, run by Neuraly, Inc. 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 10 inclusion and 34 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 30 Sep 2019. 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?
Treatment-Related Adverse Events (time frame: Day 1 through Day 29 (Part A) or Day 57 (Part B)). 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 NCT03672604, 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 30 Sep 2019.