AmyloidosisPhase 1NCT03610035

A Study of NPT189 in Healthy Subjects

A Phase 1 study testing NPT189 in people with Amyloidosis, run by Proclara Biosciences, Inc. at 1 site in Netherlands.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The purpose of the study is to evaluate the safety and tolerability of single dose of NPT189 in healthy volunteers. The study will also evaluate the pharmacokinetic characteristics of NPT189. Read more about this study

Who is it for?: Ages 18 to 65 yearsHealthy volunteers can take part
Conditions the study is for: Amyloidosis
What is being tested?: NPT189
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 48 participants
Where: 1 siteNetherlands
Study dates: 24 Jul 2018to 17 Feb 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

Male or female subjects between the ages of 18 and 65 years (inclusive);
Has provided written informed consent to participate in the study;
Weigh ≥ 45kg and ≤ 120kg with Body Mass Index of 18-32 inclusive;
Subject Rescreening: This study does not permit the re-enrollment of a subject who has discontinued from the study as a pre-treatment failure without approval from the sponsor unless the reason they were not included was because the required number of subjects needed at the time of randomization had already been met. Rescreened subjects must continue to meet all inclusion and exclusion criteria. All screening procedures must be performed within 28 days of dose administration
Female subjects of childbearing potential (defined as not surgically sterile or at least two years postmenopausal confirmed by a Screening Follicle Stimulating Hormone (FSH) level of > 40) must agree to use one of the following forms of contraception from 60 days prior through 90 days following the dose of Study Drug: hormonal (oral, transdermal, implant, or injection), barrier (condom, diaphragm with spermicide), intrauterine device (IUD), complete abstinence, or vasectomized partner (6 months minimum) or male subjects who are sexually active with women of child-bearing potential must agree to complete abstinence or to use a condom for 90 days following the dose of Study Drug;
No clinically significant abnormal findings on physical examination, vital signs, ECG, or clinical laboratory evaluation during screening; and systolic blood pressure between 140 mm and 90 mm Hg and diastolic blood pressure between 90 mm and 45 mm Hg.
Can understand and sign the informed consent document, can communicate with the investigator, and can understand and comply with the requirements of the protocol.

Who cannot join

Have a past or present serious medical illness or other medical or social reason that in the opinion of the Principal Investigator, would jeopardize the safety of the subject or impact the validity of the study results;
Participation in a clinical trial (other than a screening visit) within 60 days of study drug administration;
Use of any over-the-counter or prescription medication within 14 days prior to the dose of Study Drug or during the study, with the exception of hormonal contraceptives for women of childbearing potential, unless approved by the Principal Investigator or use of beta-blockers within 30 days prior to dose of Study Drug or during the study;
Donation of blood or plasma within 30 days of the dose of Study Drug and throughout the duration of the study and for male subjects, donation of sperm through the duration of the study;
Women who are nursing, pregnant, suspected of being pregnant, or trying to become pregnant, or are lactating, have a positive serum pregnancy test at screening or urine pregnancy test prior to administration of Study Drug regardless of childbearing potential;
Positive blood screen for HIV, hepatitis B surface antigen (HbSAg), or hepatitis C as Screening, or a positive urine screen for alcohol (Day -1 only) or, drugs of abuse, or cotinine at Screening and on Day -1;
Have clinically significant abnormalities in laboratory values as judged by the clinical investigator;
Have a history of alcoholism and/or drug abuse;

Trial locations and contacts

1 site in 1 country.

Netherlands1 site

PRA Health Sciences
Groningen, 9728 NZ

Other trials in Netherlands

About this study

Official title: A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of NPT189 in Healthy Subjects

The purpose of the study is to evaluate the safety and tolerability of single dose of NPT189 in healthy volunteers. The study will also evaluate the pharmacokinetic characteristics of NPT189.

Read the detailed description

This study is a Phase1, randomized, double-blind, placebo-controlled, single dose, dose escalation safety and tolerability study of NPT189 in health subjects. Six dose cohorts are planned each with a maximum of 8 participants per cohort. Participants will receive NPT189 by intravenous (IV) or a matching placebo by intravenous infusion. Safety, tolerability, and pharmacokinetics will be assessed.

Conditions: Amyloidosis
Being tested: NPT189, Placebo
Medical subject headings: Amyloidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NPT189Drug
Also called IgG1 (Immunoglobulin G), Fusion Protein, GAIM (General Amyloid Interaction Motif)
IgG1 Fc-GAIM fusion protein, a recombinant fusion protein
PlaceboDrug
Placebo

Study groups in NCT03610035
Study group	Role	What they receive
Experimental	Experimental	NPT189 Receives: Drug: NPT189
Placebo Comparator	Placebo comparator	Placebo Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Safety of a single intravenous (IV) infusion of NPT189 (subjects experiencing adverse events)
Summary of the frequency and percentage of
Measured: Day 1 through to Day 49

Secondary outcomes (7)

Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Cmax)
Observed maximum concentration (Cmax)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Tmax)
Time of the maximum measured concentration (Tmax)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUClast)
Area under the concentration-time curve from time zero to the last quantifiable concentration time-point (AUClast)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUCinf)
Area under the concentration-time curve from time zero extrapolated to infinity (AUCinf)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Terminal elimination half-life (t 1/2)
Terminal elimination half-life (t 1/2)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Clearance (CL)
Clearance (CL)
Measured: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Volume of distribution (Vd)
Apparent volume of distribution (Vd)
Measured: Day 1 through to Day 49

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Jul 201817 Feb 2019

First posted1 Aug 2018
Study start24 Jul 2018
Primary completion17 Feb 2019
Study completion17 Feb 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 AmyloidosisStudies 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: 48 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03610035
Sponsor's protocol ID: NPT189-CL001
Status last verified: Dec 2018
First submitted: 16 Jul 2018
Last updated: 15 Aug 2019
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Publications

Krishnan R, Tsubery H, Proschitsky MY, Asp E, Lulu M, Gilead S, Gartner M, Waltho JP, Davis PJ, Hounslow AM, Kirschner DA, Inouye H, Myszka DG, Wright J, Solomon B, Fisher RA. A bacteriophage capsid protein provides a general amyloid interaction motif (GAIM) that binds and remodels misfolded protein assemblies. J Mol Biol. 2014 Jun 26;426(13):2500-19. doi: 10.1016/j.jmb.2014.04.015. Epub 2014 Apr 22.
PubMed 24768993

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 15 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of NPT189 in Healthy Subjects
ClinicalTrials.gov ID: NCT03610035
Acronym / sponsor ID: NPT189-CL001
Conditions: Amyloidosis
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 48 participants (actual)
Lead sponsor: Proclara Biosciences, Inc.Industry (pharma, biotech, devices)
Study start: 24 Jul 2018 (actual)
Primary completion: 17 Feb 2019 (actual)
Study completion: 17 Feb 2019 (actual)
First posted: 1 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.

The purpose of the study is to evaluate the safety and tolerability of single dose of NPT189 in healthy volunteers. The study will also evaluate the pharmacokinetic characteristics of NPT189.

Detailed description

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
Experimental	Experimental	Drug: NPT189NPT189
Placebo Comparator	Placebo comparator	Drug: PlaceboPlacebo

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Male or female subjects between the ages of 18 and 65 years (inclusive);
Has provided written informed consent to participate in the study;
Weigh ≥ 45kg and ≤ 120kg with Body Mass Index of 18-32 inclusive;
Subject Rescreening: This study does not permit the re-enrollment of a subject who has discontinued from the study as a pre-treatment failure without approval from the sponsor unless the reason they were not included was because the required number of subjects needed at the time of randomization had already been met. Rescreened subjects must continue to meet all inclusion and exclusion criteria. All screening procedures must be performed within 28 days of dose administration
Female subjects of childbearing potential (defined as not surgically sterile or at least two years postmenopausal confirmed by a Screening Follicle Stimulating Hormone (FSH) level of > 40) must agree to use one of the following forms of contraception from 60 days prior through 90 days following the dose of Study Drug: hormonal (oral, transdermal, implant, or injection), barrier (condom, diaphragm with spermicide), intrauterine device (IUD), complete abstinence, or vasectomized partner (6 months minimum) or male subjects who are sexually active with women of child-bearing potential must agree to complete abstinence or to use a condom for 90 days following the dose of Study Drug;

Key exclusion criteria (8 listed)

Have a past or present serious medical illness or other medical or social reason that in the opinion of the Principal Investigator, would jeopardize the safety of the subject or impact the validity of the study results;
Participation in a clinical trial (other than a screening visit) within 60 days of study drug administration;
Use of any over-the-counter or prescription medication within 14 days prior to the dose of Study Drug or during the study, with the exception of hormonal contraceptives for women of childbearing potential, unless approved by the Principal Investigator or use of beta-blockers within 30 days prior to dose of Study Drug or during the study;
Donation of blood or plasma within 30 days of the dose of Study Drug and throughout the duration of the study and for male subjects, donation of sperm through the duration of the study;
Women who are nursing, pregnant, suspected of being pregnant, or trying to become pregnant, or are lactating, have a positive serum pregnancy test at screening or urine pregnancy test prior to administration of Study Drug regardless of childbearing potential;

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
NPT189Also: IgG1 (Immunoglobulin G), Fusion Protein, GAIM (General Amyloid Interaction Motif)IgG1 Fc-GAIM fusion protein, a recombinant fusion protein	Drug	Experimental
PlaceboPlacebo	Drug	Placebo Comparator

Primary outcome

Safety of a single intravenous (IV) infusion of NPT189 (subjects experiencing adverse events)
Time frame: Day 1 through to Day 49
Summary of the frequency and percentage of

7 secondary outcomes

Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Cmax)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Tmax)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUClast)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUCinf)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Terminal elimination half-life (t 1/2)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Clearance (CL)
Time frame: Day 1 through to Day 49
Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Volume of distribution (Vd)
Time frame: Day 1 through to Day 49

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 15 Aug 2019.
Background: PubMed 24768993Krishnan R, Tsubery H, Proschitsky MY, Asp E, Lulu M, Gilead S, Gartner M, Waltho JP, Davis PJ, Hounslow AM, Kirschner DA, Inouye H, Myszka DG, Wright J, Solomon B, Fisher RA. A bacteriophage capsid protein provides a general amyloid interaction motif (GAIM) that binds and remodels misfolded protein assemblies. J Mol Biol. 2014 Jun 26;426(13):2500-19. doi: 10.1016/j.jmb.2014.04.015. Epub 2014 Apr 22.
Proschitsky, M et al., GAIM Fusions are Therapeutic Candidates for Peripheral Amyloidosis (2018).Link provided by the sponsor.
Individual participant dataSharing planned: Undecided

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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.

Proclara Biosciences study director

Proclara

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 NPT189 in people with Amyloidosis, run by Proclara Biosciences, Inc. at 1 site in Netherlands.

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 8 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 15 Aug 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 Netherlands. 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?

Safety of a single intravenous (IV) infusion of NPT189 (subjects experiencing adverse events) (time frame: Day 1 through to Day 49). 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 NCT03610035, 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 15 Aug 2019.