Amyotrophic Lateral SclerosisPhase 1NCT03651349

To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers

A Phase 1 study testing HK-001 in people with Amyotrophic Lateral Sclerosis, run by Everfront Biotech Co., Ltd. at 1 site in Taiwan.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

Who is it for?: Ages 20 years and olderHealthy volunteers can take part
Conditions the study is for: Amyotrophic Lateral Sclerosis
What is being tested?: HK-001
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 56 participants
Where: 1 siteTaiwan
Study dates: 1 Feb 2021to 30 Jun 2028
Phase: Phase 1
Are there results?: Not yet: the study is under way

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 20 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

Subject's age is no less than 20 years old.
Subjects whose body mass index (BMI) at screening is within a range of ≥18.5 kg/m2 and <25.0 kg/m2.
BMI = Body Weight (kg) / [Height (m)]2 And body weight is not less than 50 kg and 45 kg for males and females, respectively.
Subject's medical history shows no contraindication to the test medications [hypersensitivity to (Z)-BP or any component of test and reference products].
Subjects who are judged to be in good health by the investigator based upon the results of physical examinations (PEs) and chest X-ray (within 60 days prior to the first study dose), and all items of routine laboratory tests, including serum biochemistry, hematology and urinalysis, are within normal range as judged by the site. Assessment items of blood biochemistry include electrolytes (sodium, potassium, chloride, calcium, phosphorus), albumin, total cholesterol, total bilirubin, ALP, SGOT, SGPT, GGT, BUN, PT, APPT, serum creatinine, triglyceride, glucose, amylase, lipase, ACTH, aldosterone, cortisol, T3, free T4, TSH, and uric acid. Assessment items of hematology tests include red blood cell (RBC), white blood cell (WBC) and platelet count; differential WBC count including neutrophils, lymphocytes, monocytes, eosinophils, and basophils; hemoglobin, and hematocrit. Assessment items of urinalysis include appearance, gravity, pH, erythrocyte, leukocyte, epithelial cells, glucose, protein, ketones, and nitrite
Female subjects show negative pregnancy test results within 30 days prior to the first study dose.

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Subjects did not take any of the following medications in the specified durations:
Any medication (except contraceptives) within 14 days prior to the first dose of the study
Any enzyme inducer or inhibitor within 30 days prior to the first dose of the study
Subjects understood and have signed the written informed consent form.

Who cannot join

Subjects with any properly diagnosed disease within 30 days prior to the first dose of the study
Subjects with a clinically significant hematological, endocrinal, cardiovascular, hepatic, renal, gastrointestinal, and/or pulmonary disorders; subjects with any predisposing condition that might interfere with the absorption, distribution, metabolism and excretion of drugs; subjects who have had any previous gastrointestinal surgery, except appendectomy if performed >90 days prior to the first dose of the study
Subjects who have received any known hepatic or renal clearance-altering agents (e.g., erythromycin, cimetidine, barbiturates, phenothiazine, clarithromycin, trolearndomycin, ketoconazole, miconazolem fluconazole, itraconazole) for a period of up to 30 days prior to the first dose of the study
Subjects had participated in investigational drug trials and took any investigational drugs within 60 days prior to the first study dose.
Subjects had blood donation for more than 250 mL within 60 days prior to the first dose of the study.
Subjects had a history of drug abuse or alcohol abuse according to DSM IV criteria.

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Subjects who are smokers or have smoking history
Subjects who cannot stop caffeine-intakes for 48 hours prior to the first study dose and during the entire study period.
Subjects who are pregnant or lactating
For enrollment of female subjects with child-bearing potential, the subject must be practicing sexual abstinence or be using and willing to continue to use a medically acceptable form of birth control for at least 1 month prior to screening (that period will extend to 3 months for oral contraceptive use) and for at least 30 days after the last dose of study drug. For a subject to be considered not to be of child-bearing potential, she must have been amenorrheic for at least 2 years, or must have had a hysterectomy, a bilateral tubal ligation, and/or a bilateral oophorectomy (as determined by the medical history). The male partner of a female study subject with childbearing potential must use a condom and ensure that his partner uses a suitable method of contraception as outlined above.
Subjects who are inappropriate to participate in this study, as judged by the clinical investigator
Subjects who have been tested positive for the following tests:
Human immunodeficiency virus (HIV)
Hepatitis B virus (HBV)
Hepatitis C virus (HCV)

Trial locations and contacts

1 site in 1 country.

Taiwan1 site

Hualien Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
Hualien City, 97002

Other trials in Taiwan

About this study

Official title: A Phase I Study to Determine the Maximum Tolerated Dose (MTD) of HK-001 and to Evaluate Its Pharmacokinetic Profile in Healthy Volunteers

Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts. After single dose administration, followed by an independent Data and Safety Monitoring Board (DSMB) meeting for safety assessments (including the available plasma pharmacokinetic profile), the subjects will be allowed to receive (Z)-BP or placebo twice a day orally at the study site for 14 consecutive days and follow up on the 28th day after the last dose administration by a site visit. The study drugs (including placebo) will be administered at the study site by following the investigator's instructions to either perform blood sampling for pharmacokinetic evaluation or maximize the treatment compliance.

There will be 7 cohorts and subjects will be randomized into cohorts consisting of 8 subjects each (6 active and 2 placebo controls per cohort). Dose cohorts will be escalated sequentially from low to high dose (50 mg, BID; 100 mg, BID; 150 mg, BID; 225 mg, BID; 300 mg, BID; 400 mg, BID; 525 mg, BID) by following a modified Fibonacci sequence, and based on the decision of an independent DSMB at a set time point. Following all subjects of a cohort complete the safety and PK evaluation after receiving the last dose administration, a cohort at the next dose level will be launched if the DSMB does not identify significant safety concerns after reviewing safety data and PK profiles.

Conditions: Amyotrophic Lateral Sclerosis
Being tested: HK-001, Placebo control
Medical subject headings: Amyotrophic Lateral Sclerosis

What participants receive

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

HK-001Drug
Also called (Z)-n-butylidenephthalide [(Z)-BP]
HK-001 is synthesized by PharmaCore Biotech Co., Ltd. Synthetic (Z)-BP with high purity will be used in this proposed study.
Placebo controlDrug
Also called Placebo
In order to evaluate the safety and tolerability of HK-001 more accurately, a drug-free placebo control is planned to be utilized for the same administration route in this proposed trial. The placebo control that is identical in appearance, smell, weight, and excipients will be supplied by Everfront Biotech Inc.

Study groups in NCT03651349
Study group	Role	What they receive
Placebo control	Placebo comparator	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: Placebo control
50 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
100 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
150 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
225 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
300 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
400 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001
525 mg, BID	Experimental	Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each. Receives: Drug: HK-001

What is being measured

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

Maximum Tolerated Dose (MTD)
The MTD will be determined by study definition as the highest dose level without significant safety and tolerability concern.
Measured: 48 days
Dose Limiting Toxicities (DLT)
In this study, the DLTs are defined as below:
1. Any Grade ≥2 AE found during study period that is considered at least possibly drug related as judged by the investigator. The NCI-CTCAE 4.03 will be used to grade serum chemistry, hematology, and other abnormalities in the current study.
2. Any drug-related (including probably or possibly drug-related) serious adverse event (SAE) found during study period.
Measured: 48 days

Timeline

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

1 Feb 202130 Jun 2028

First posted29 Aug 2018
Study start1 Feb 2021
Primary completion31 Dec 2027 (estimated)
Study completion30 Jun 2028 (estimated)

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: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 56 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03651349
Sponsor's protocol ID: EFBORAZ20141120
Status last verified: Sep 2026
First submitted: 26 Aug 2018
Last updated: 11 Sep 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

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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Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
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All Amyotrophic Lateral Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I Study to Determine the Maximum Tolerated Dose (MTD) of HK-001 and to Evaluate Its Pharmacokinetic Profile in Healthy Volunteers
ClinicalTrials.gov ID: NCT03651349
Acronym / sponsor ID: EFBORAZ20141120
Conditions: Amyotrophic Lateral Sclerosis
Phase: Phase 1
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 56 participants (estimated)
Lead sponsor: Everfront Biotech Co., Ltd.Industry (pharma, biotech, devices)
Study start: 1 Feb 2021 (actual)
Primary completion: 31 Dec 2027 (estimated)
Study completion: 30 Jun 2028 (estimated)
First posted: 29 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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 8

Arm	Type	Receives
Placebo control	Placebo comparator	Drug: Placebo controlEligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
50 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
100 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
150 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
225 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
300 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
400 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.
525 mg, BID	Experimental	Drug: HK-001Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts consisting of 8 subjects each.

Who may qualify

Ages: 20 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (10 listed)

Subject's age is no less than 20 years old.
Subjects whose body mass index (BMI) at screening is within a range of ≥18.5 kg/m2 and <25.0 kg/m2.
BMI = Body Weight (kg) / [Height (m)]2 And body weight is not less than 50 kg and 45 kg for males and females, respectively.
Subject's medical history shows no contraindication to the test medications [hypersensitivity to (Z)-BP or any component of test and reference products].
Subjects who are judged to be in good health by the investigator based upon the results of physical examinations (PEs) and chest X-ray (within 60 days prior to the first study dose), and all items of routine laboratory tests, including serum biochemistry, hematology and urinalysis, are within normal range as judged by the site. Assessment items of blood biochemistry include electrolytes (sodium, potassium, chloride, calcium, phosphorus), albumin, total cholesterol, total bilirubin, ALP, SGOT, SGPT, GGT, BUN, PT, APPT, serum creatinine, triglyceride, glucose, amylase, lipase, ACTH, aldosterone, cortisol, T3, free T4, TSH, and uric acid. Assessment items of hematology tests include red blood cell (RBC), white blood cell (WBC) and platelet count; differential WBC count including neutrophils, lymphocytes, monocytes, eosinophils, and basophils; hemoglobin, and hematocrit. Assessment items of urinalysis include appearance, gravity, pH, erythrocyte, leukocyte, epithelial cells, glucose, protein, ketones, and nitrite

Key exclusion criteria (15 listed)

Subjects with any properly diagnosed disease within 30 days prior to the first dose of the study
Subjects with a clinically significant hematological, endocrinal, cardiovascular, hepatic, renal, gastrointestinal, and/or pulmonary disorders; subjects with any predisposing condition that might interfere with the absorption, distribution, metabolism and excretion of drugs; subjects who have had any previous gastrointestinal surgery, except appendectomy if performed >90 days prior to the first dose of the study
Subjects who have received any known hepatic or renal clearance-altering agents (e.g., erythromycin, cimetidine, barbiturates, phenothiazine, clarithromycin, trolearndomycin, ketoconazole, miconazolem fluconazole, itraconazole) for a period of up to 30 days prior to the first dose of the study
Subjects had participated in investigational drug trials and took any investigational drugs within 60 days prior to the first study dose.
Subjects had blood donation for more than 250 mL within 60 days prior to the first dose of the study.

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
HK-001Also: (Z)-n-butylidenephthalide [(Z)-BP]HK-001 is synthesized by PharmaCore Biotech Co., Ltd. Synthetic (Z)-BP with high purity will be used in this proposed study.	Drug	100 mg, BID, 150 mg, BID, 225 mg, BID, 300 mg, BID, 400 mg, BID, 50 mg, BID, 525 mg, BID
Placebo controlAlso: PlaceboIn order to evaluate the safety and tolerability of HK-001 more accurately, a drug-free placebo control is planned to be utilized for the same administration route in this proposed trial. The placebo control that is identical in appearance, smell, weight, and excipients will be supplied by Everfront Biotech Inc.	Drug	Placebo control

Primary outcome

Maximum Tolerated Dose (MTD)
Time frame: 48 days
The MTD will be determined by study definition as the highest dose level without significant safety and tolerability concern.
Dose Limiting Toxicities (DLT)
Time frame: 48 days
In this study, the DLTs are defined as below: 1. Any Grade ≥2 AE found during study period that is considered at least possibly drug related as judged by the investigator. The NCI-CTCAE 4.03 will be used to grade serum chemistry, hematology, and other abnormalities in the current study. 2. Any drug-related (including probably or possibly drug-related) serious adverse event (SAE) found during study period.

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.

Related trials for Amyotrophic Lateral Sclerosis

RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
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Functional Outcomes and Control Using Synchron BCI - Australia
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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.

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 HK-001 in people with Amyotrophic Lateral Sclerosis, run by Everfront Biotech Co., Ltd. at 1 site in Taiwan.

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 15 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: under way, not looking for participants, as of the sponsor's last update on 11 Sep 2026. 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 Taiwan. 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?

This study is still under way. Results are not available in the source record yet.

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?

Maximum Tolerated Dose (MTD) (time frame: 48 days). 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?

On the ClinicalTrials.gov record for NCT03651349.

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 11 Sep 2026.