MELAS SyndromePhase 1NCT03056209
Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
A Phase 1 study testing KL1333 25 mg, KL1333 50 mg and KL1333 100 mg in people with MELAS Syndrome and Mitochondrial Respiratory Chain Deficiencies, run by Yungjin Pharm. Co., Ltd. at 1 site in South Korea.
Trial summary
The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose. Read more about this study
- Who is it for?
- Ages 19 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- MELAS Syndrome, Mitochondrial Respiratory Chain Deficiencies
- What is being tested?
- KL1333 25 mg, KL1333 50 mg and KL1333 100 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteSouth Korea
- Study dates
- 26 Jun 2017to 16 Apr 2018
- 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 19 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- 19 - 45 years of age at the time of screening
- Subjects weighing ≥55 and ≤90 kg with BMI between 18 and 27 kg/m2
- Subjects who agreed to voluntarily participate in this study and comply with all the study requirements by signing informed consent form after being informed of the nature of this study and understanding all aspects of this study
Who cannot join
- History of clinically significant hepatic, renal, neurologic, immunologic, respiratory, endocrine disease or hematologic•oncologic disease, cardiovascular, psychiatric disease
- History of disease or surgery of the gastrointestinal tract that could interfere with kinetics of the study drug. Simple hernia repair or appendectomy are excepted.
- History of clinically significant or relevant allergy/hypersensitivity
- Blood AST (SGOT), ALT (SGPT) >1.5 of upper limit
- eGFR value of ≤90mL/min/1.73m2
- Systolic blood pressure of <100 mmHg or >160 mmHg
Show all 17Show fewer
- Diastolic blood pressure of <60 mmHg or >100 mmHg
- Any abnormalities in 12-lead ECG at screening visit
- Subjects who showed positive result in drug abuse tests, or who has history of drug abuse within 60 days prior to the time of screening
- Subjects who took prescribed medications or oriental medicine within 14 days or over-the-counter (OTC) medications or vitamins within 7 days prior to the dose of the study drug (however, the subject can be included if other criteria are met according to the discretion of the investigator)
- Subjects who were administered any investigational products within 3 months from the first dose of the study drug
- Subjects who have donated whole blood (60 days) or partial blood (30 days), or received blood transfusion
- Subjects who have had alcohol consistently (>21units/week, 1unit=10 g of pure alcohol) or who is not able to stop drinking alcohol throughout the study period
- Subjects who have smoked until 90 days prior to the study initiation or who are not able to stop smoking throughout the study period
- Subjects who are not able to stop taking grapefruit/caffeine from 3 days prior to the first dose of the study drug throughout the study period
- Subjects who plan for pregnancy during the study period or who are not able to use established contraceptive method
- Subject who judged not eligible for study participation by investigator
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Seoul National University Hospital
Seoul, Jongno-gu, 110744
About this study
Official title: A Dose Block-randomized, Double-blind, Placebo-controlled, Single-dose, Dose-escalation, Phase I Clinical Study
The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.
- Being tested
- KL1333 25 mg, KL1333 50 mg, KL1333 100 mg, KL1333 200 mg, KL1333 400 mg
- Keywords
- Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Medical subject headings
- MELAS Syndrome, Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KL1333 25 mgDrug
oral administration, single dose, 25 mg 1 tab
KL1333 50 mgDrug
oral administration, single dose, 25 mg 2 tabs
KL1333 100 mgDrug
oral administration, single dose, 100 mg 1 tab
KL1333 200 mgDrug
oral administration, single dose, 100 mg 2 tabs
KL1333 400 mgDrug
oral administration, single dose, 100 mg 4 tabs
KL1333 600 mgDrug
oral administration, single dose, 100 mg 6 tabs
KL1333 800 mgDrug
oral administration, single dose, 100 mg 8 tabs
PlaceboDrug
oral administration, placebo
| Study group | Role | What they receive |
|---|---|---|
| KL1333 25mg | Experimental | Group 1 Receives: Drug: KL1333 25 mg; Drug: Placebo |
| KL1333 50mg | Experimental | Group 2 Receives: Drug: KL1333 50 mg; Drug: Placebo |
| KL1333 100mg | Experimental | Group 3 Receives: Drug: KL1333 100 mg; Drug: Placebo |
| KL1333 200mg | Experimental | Group 4 Receives: Drug: KL1333 200 mg; Drug: Placebo |
| KL1333 400mg | Experimental | Group 5 Receives: Drug: KL1333 400 mg; Drug: Placebo |
| KL1333 600mg | Experimental | Group 6 Receives: Drug: KL1333 600 mg; Drug: Placebo |
| KL1333 800mg | Experimental | Group 7 Receives: Drug: KL1333 800 mg; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of reported adverse events
Measured: from day 1 to day 15
Secondary outcomes (3)
Maximum plasma concentration (Cmax) of KL1333
Measured: from day 1 to day 15
Area Under the Curve (AUC) of KL1333
Measured: from day 1 to day 15
Half-life (T1/2) of KL1333
Measured: from day 1 to day 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2017
- Study start26 Jun 2017
- Primary completion16 Apr 2018
- Study completion16 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MELAS SyndromeStudies 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
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yungjin Pharm. Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kyung-Sang Yu, MD., MBA, Study chair, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03056209
- Sponsor's protocol ID
- KL1333_101
- Status last verified
- Apr 2018
- First submitted
- 5 Feb 2017
- Last updated
- 27 Apr 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose Block-randomized, Double-blind, Placebo-controlled, Single-dose, Dose-escalation, Phase I Clinical Study
- ClinicalTrials.gov ID
- NCT03056209
- Acronym / sponsor ID
- KL1333_101
- Keywords
- Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Yungjin Pharm. Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Jun 2017 (actual)
- Primary completion
- 16 Apr 2018 (actual)
- Study completion
- 16 Apr 2018 (actual)
- First posted
- 17 Feb 2017 (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 First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| KL1333 25mg | Experimental | Drug: KL1333 25 mg, Drug: PlaceboGroup 1 |
| KL1333 50mg | Experimental | Drug: KL1333 50 mg, Drug: PlaceboGroup 2 |
| KL1333 100mg | Experimental | Drug: KL1333 100 mg, Drug: PlaceboGroup 3 |
| KL1333 200mg | Experimental | Drug: KL1333 200 mg, Drug: PlaceboGroup 4 |
| KL1333 400mg | Experimental | Drug: KL1333 400 mg, Drug: PlaceboGroup 5 |
| KL1333 600mg | Experimental | Drug: KL1333 600 mg, Drug: PlaceboGroup 6 |
| KL1333 800mg | Experimental | Drug: KL1333 800 mg, Drug: PlaceboGroup 7 |
Who may qualify
- Ages
- 19 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 19 - 45 years of age at the time of screening
- Subjects weighing ≥55 and ≤90 kg with BMI between 18 and 27 kg/m2
- Subjects who agreed to voluntarily participate in this study and comply with all the study requirements by signing informed consent form after being informed of the nature of this study and understanding all aspects of this study
Key exclusion criteria (17 listed)
- History of clinically significant hepatic, renal, neurologic, immunologic, respiratory, endocrine disease or hematologic•oncologic disease, cardiovascular, psychiatric disease
- History of disease or surgery of the gastrointestinal tract that could interfere with kinetics of the study drug. Simple hernia repair or appendectomy are excepted.
- History of clinically significant or relevant allergy/hypersensitivity
- Blood AST (SGOT), ALT (SGPT) >1.5 of upper limit
- eGFR value of ≤90mL/min/1.73m2
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 |
|---|---|---|
| KL1333 25 mgoral administration, single dose, 25 mg 1 tab | Drug | KL1333 25mg |
| KL1333 50 mgoral administration, single dose, 25 mg 2 tabs | Drug | KL1333 50mg |
| KL1333 100 mgoral administration, single dose, 100 mg 1 tab | Drug | KL1333 100mg |
| KL1333 200 mgoral administration, single dose, 100 mg 2 tabs | Drug | KL1333 200mg |
| KL1333 400 mgoral administration, single dose, 100 mg 4 tabs | Drug | KL1333 400mg |
| KL1333 600 mgoral administration, single dose, 100 mg 6 tabs | Drug | KL1333 600mg |
| KL1333 800 mgoral administration, single dose, 100 mg 8 tabs | Drug | KL1333 800mg |
| Placebooral administration, placebo | Drug | KL1333 100mg, KL1333 200mg, KL1333 25mg, KL1333 400mg, KL1333 50mg, KL1333 600mg, KL1333 800mg |
Primary outcome
Number of reported adverse events
Time frame: from day 1 to day 15
3 secondary outcomes
Maximum plasma concentration (Cmax) of KL1333
Time frame: from day 1 to day 15
Area Under the Curve (AUC) of KL1333
Time frame: from day 1 to day 15
Half-life (T1/2) of KL1333
Time frame: from day 1 to day 15
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 Apr 2018.
- Individual participant dataSharing planned: No
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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.
Kyung-Sang Yu, MD., MBA study chair
Seoul National University Hospital
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 KL1333 25 mg, KL1333 50 mg and KL1333 100 mg in people with MELAS Syndrome and Mitochondrial Respiratory Chain Deficiencies, run by Yungjin Pharm. Co., Ltd. at 1 site in South Korea.
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 3 inclusion and 17 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 Apr 2018. 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 South Korea. 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. 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 reported adverse events (time frame: from day 1 to day 15). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Apr 2018.