Paroxysmal Nocturnal Hemoglobinuria, PNHPhase 1NCT07462780
A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
A Phase 1 study testing CMS-D017 Capsule in people with Paroxysmal Nocturnal Hemoglobinuria, PNH and Complement-mediated Kidney Disease, run by Shenzhen Kangzhe Biotechnology Co., Ltd. at 1 site in China.
Trial summary
This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple ascending dose (MAD) study (referred to as Part 2 MAD). Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Complement-mediated Kidney Disease
- What is being tested?
- CMS-D017 Capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 1 siteChina
- Study dates
- 5 Mar 2026to Dec 2026
- 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 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Voluntarily participates in this study and signs the informed consent form.
- Able to communicate well with the investigator and understands and complies with all requirements and restrictions of this study, and is able to complete the study in accordance with the protocol.
- Aged 18-55 years (inclusive, as of the day of signing the informed consent form), male or female.
- Body Mass Index (BMI) between 19.0 and 26.0 kg/m² (inclusive) at screening, with females weighing ≥ 45.0 kg and males weighing ≥ 50.0 kg.
- Participants (and their partners) with reproductive capacity must have no plans for pregnancy, egg donation, or sperm donation from the date of signing the informed consent form until 3 months after the last dose of the study drug, and must comply with contraceptive requirements (see Appendix 1), agreeing to use at least one highly effective non-hormonal contraceptive method.
Who cannot join
- Allergy History
- History of severe allergies, including food allergies, or allergy to the study drug or its components.
- Medical History/Conditions
- Significant history or clinical manifestations of cardiovascular, respiratory, digestive, urogenital, hematologic, endocrine and metabolic, rheumatic, immunologic, neuropsychiatric, or musculoskeletal diseases requiring medication and/or other treatments (including dietary restrictions and physical therapy), as deemed unsuitable for participation in this study by the investigator.
- Any condition that may affect drug absorption, including but not limited to: malabsorption syndrome, inflammatory bowel disease, celiac disease, gastrectomy, cholecystectomy, bowel resection (except appendectomy).
- History of meningococcal infection or first-degree relatives with history of meningococcal infection.
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- Active infection or acute disease state (e.g., fever, nausea, vomiting, or diarrhea) within 2 weeks prior to screening.
- Current history of tuberculosis infection; or positive tuberculosis (TB) test result. Note: If the TB test result is indeterminate, one repeat test is allowed.
- History of severe trauma or surgery within 8 weeks prior to screening, or planned surgery during the study period.
- History or current presence of the following cardiac risk factors:
- Torsades de pointes or risk factors (e.g., hypokalemia, hypomagnesemia, use of drugs causing delayed cardiac repolarization) History of cardiac arrest, syncope, heart failure; myocardial infarction, angina; valvular heart disease; cardiomyopathy or family history; clinically significant arrhythmias (e.g., sick sinus syndrome, atrioventricular conduction block, Adams-Stokes syndrome, Brugada syndrome or family history, long QT syndrome, atrial flutter, atrial fibrillation, supraventricular tachycardia, ventricular tachycardia).
- Prior/Concomitant Treatments
- Participation in any other clinical study involving drugs or medical devices within 3 months prior to screening, or planned participation in such studies during this study, or within 5 half-lives of that drug (whichever is longer).
- Vaccination within 4 weeks prior to screening or planned vaccination during the study or within 1 month after last dose (participants vaccinated against meningococcal and pneumococcal infections may be included if vaccination was completed at least 2 weeks before dosing).
- Use of known CYP2C8 or CYP3A inducers or inhibitors, or P-gp inhibitors within 4 weeks prior to dosing (see Appendix 2).
- Use of any prescription or over-the-counter drugs (including herbal medicines, vitamins, minerals, and dietary supplements) within 2 weeks or at least 5 half-lives prior to dosing, whichever is longer.
- Substance Use, Alcohol, Tobacco, or Nicotine, Dietary/Exercise Restrictions
- History of drug abuse within 6 months prior to screening, or positive result for any drug abuse test.
- Alcohol consumption exceeding 14 units per week within 3 months prior to screening (1 unit = 360 mL beer, 150 mL wine, or 45 mL spirits), or positive breath alcohol test, or inability to abstain from alcohol during the study.
- Average smoking of more than 5 cigarettes per day within 3 months prior to screening, or inability to stop using any tobacco products during the study.
- Inability to abstain from grapefruit or grapefruit-related citrus fruits or juices (e.g., pomelo) within 7 days prior to dosing and during the study.
- Consumption of caffeine-containing products (e.g., coffee, tea, cola, other caffeinated beverages, or chocolate) within 3 days prior to dosing, or refusal to avoid such products throughout the study.
- Engagement in strenuous exercise or physical activity within 3 days prior to dosing, or refusal to avoid such activities throughout the study.
- Examinations and Assessments
- Corrected QT interval (using Fridericia's formula, QTcF = QT/(RR^0.33)) > 450 msec in males or females.
- Abnormal findings in physical examination, vital signs, safety laboratory tests, 12-lead ECG, or other auxiliary tests (chest X-ray, abdominal ultrasound) deemed clinically significant by the investigator.
- Positive test results for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCVAb), syphilis antibody (TPAb), or human immunodeficiency virus antibody (HIV Ab).
- Other
- Pregnant or lactating females.
- Special dietary requirements or inability to comply with standardized diet.
- Difficulty with venous blood sampling (e.g., history of needle or blood phobia), or poor venous condition as deemed unsuitable for enrollment by the investigator.
- Donation or loss of ≥ 400 mL blood within 3 months prior to screening, or receipt of blood transfusion or blood products; or planned blood or blood component donation during the study.
- Other conditions deemed unsuitable for participation in this study by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University Third Hospital
Beijing
Haiyan Li, Professor, +86 10 8226 6226, haiyanli1027@hotmail.com
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Dose-escalation Phase I Study to Evaluate the Safety, Tolerability, PK and PD Characteristics of CMS-D017 Following Single and Multiple Administrations in Healthy Participants
This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple ascending dose (MAD) study (referred to as Part 2 MAD). The study aims to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) characteristics of CMS-D017 capsules following single and multiple oral administrations in healthy Chinese adult participants.
Both parts of the study are designed as randomized, double-blind, placebo-controlled, sequential cohort trials. Part 1 SAD plans to include 6 dose cohorts, with 8 participants per cohort (6 receiving CMS-D017 and 2 receiving placebo), for a total of 48 participants. Part 2 MAD plans to include 4 dose cohorts, with 10 participants per cohort (8 receiving CMS-D017 and 2 receiving placebo), for a total of 40 participants.
- Being tested
- CMS-D017 Capsule, CMS-D017 Placebo Capsule
- Keywords
- CMS-D017, Phase I trial in healthy participants, single and multiple ascending dose studies, pharmacokinetics, pharmacodynamics
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CMS-D017 CapsuleDrug
healthy participant
CMS-D017 Placebo CapsuleDrug
healthy participant
CMS-D017 CapsuleDrug
healthy subject
CMS-D017 Placebo CapsuleDrug
healthy subject
| Study group | Role | What they receive |
|---|---|---|
| Single Dose Escalation of CMS-D017 | Experimental | 6 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo Receives: Drug: CMS-D017 Capsule |
| Single Dose Escalation- placebo | Placebo comparator | 6 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo Receives: Drug: CMS-D017 Placebo Capsule |
| Multiple Dose Escalation of CMS-D017 | Experimental | 4 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo Receives: Drug: CMS-D017 Capsule |
| Multiple Dose Escalation of placebo | Experimental | 4 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo Receives: Drug: CMS-D017 Placebo Capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of CMS-D017 -Adverse events
Monitoring of adverse events
Measured: Up to 23 days
Secondary outcomes (13)
Tmax
Time to reach maximum observed plasma concentration
Measured: Up to 16 days
Rac_Cmax
Accumulation ratio based on Cmax
Measured: Up to 16 days
Cmax
Maximum observed concentration
Measured: Up to 16 days
AUC
Area under the curve
Measured: Up to 16 days
λz
Terminal phase elimination rate constant
Measured: Up to 16 days
t1/2
Terminal half-life
Measured: Up to 16 days
CL/F
Clearance divided by Bioavailability
Measured: Up to 16 days
Vz/F
Volume of distribution during the terminal phase divided by Bioavailability
Measured: Up to 16 days
Cavg,ss
Average plasma concentration at steady state
Measured: Up to 16 days
Rac_AUC0-tau
Accumulation ratio based on AUC0-tau
Measured: Up to 16 days
CLss/F
Clearance at steady state divided by bioavailability
Measured: Up to 16 days
Vss/F
Volume of distribution at steady state divided by bioavailability
Measured: Up to 16 days
Inhibition rate of the AP
Inhibition rate of complement system alternative pathway
Measured: Up to 16 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2026
- Study start5 Mar 2026
- Primary completionNov 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria, PNHStudies 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
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Kangzhe Biotechnology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07462780
- Sponsor's protocol ID
- D017-01-001
- Status last verified
- Feb 2026
- First submitted
- 1 Mar 2026
- Last updated
- 8 Apr 2026
- 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 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Dose-escalation Phase I Study to Evaluate the Safety, Tolerability, PK and PD Characteristics of CMS-D017 Following Single and Multiple Administrations in Healthy Participants
- ClinicalTrials.gov ID
- NCT07462780
- Acronym / sponsor ID
- D017-01-001
- Keywords
- CMS-D017, Phase I trial in healthy participants, single and multiple ascending dose studies, pharmacokinetics, pharmacodynamics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- Shenzhen Kangzhe Biotechnology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 5 Mar 2026 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 10 Mar 2026 (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 study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple ascending dose (MAD) study (referred to as Part 2 MAD). The study aims to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) characteristics of CMS-D017 capsules following single and multiple oral administrations in healthy Chinese adult participants.
Both parts of the study are designed as randomized, double-blind, placebo-controlled, sequential cohort trials. Part 1 SAD plans to include 6 dose cohorts, with 8 participants per cohort (6 receiving CMS-D017 and 2 receiving placebo), for a total of 48 participants. Part 2 MAD plans to include 4 dose cohorts, with 10 participants per cohort (8 receiving CMS-D017 and 2 receiving placebo), for a total of 40 participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single Dose Escalation of CMS-D017 | Experimental | Drug: CMS-D017 Capsule6 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo |
| Single Dose Escalation- placebo | Placebo comparator | Drug: CMS-D017 Placebo Capsule6 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo |
| Multiple Dose Escalation of CMS-D017 | Experimental | Drug: CMS-D017 Capsule4 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo |
| Multiple Dose Escalation of placebo | Experimental | Drug: CMS-D017 Placebo Capsule4 sequential dose escalation cohorts - participants are randomized either to investigational drug or matching placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Voluntarily participates in this study and signs the informed consent form.
- Able to communicate well with the investigator and understands and complies with all requirements and restrictions of this study, and is able to complete the study in accordance with the protocol.
- Aged 18-55 years (inclusive, as of the day of signing the informed consent form), male or female.
- Body Mass Index (BMI) between 19.0 and 26.0 kg/m² (inclusive) at screening, with females weighing ≥ 45.0 kg and males weighing ≥ 50.0 kg.
- Participants (and their partners) with reproductive capacity must have no plans for pregnancy, egg donation, or sperm donation from the date of signing the informed consent form until 3 months after the last dose of the study drug, and must comply with contraceptive requirements (see Appendix 1), agreeing to use at least one highly effective non-hormonal contraceptive method.
Key exclusion criteria (33 listed)
- Allergy History
- History of severe allergies, including food allergies, or allergy to the study drug or its components.
- Medical History/Conditions
- Significant history or clinical manifestations of cardiovascular, respiratory, digestive, urogenital, hematologic, endocrine and metabolic, rheumatic, immunologic, neuropsychiatric, or musculoskeletal diseases requiring medication and/or other treatments (including dietary restrictions and physical therapy), as deemed unsuitable for participation in this study by the investigator.
- Any condition that may affect drug absorption, including but not limited to: malabsorption syndrome, inflammatory bowel disease, celiac disease, gastrectomy, cholecystectomy, bowel resection (except appendectomy).
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 |
|---|---|---|
| CMS-D017 Capsulehealthy participant | Drug | Single Dose Escalation of CMS-D017 |
| CMS-D017 Placebo Capsulehealthy participant | Drug | Single Dose Escalation- placebo |
| CMS-D017 Capsulehealthy subject | Drug | Multiple Dose Escalation of CMS-D017 |
| CMS-D017 Placebo Capsulehealthy subject | Drug | Multiple Dose Escalation of placebo |
Primary outcome
Safety and tolerability of CMS-D017 -Adverse events
Time frame: Up to 23 days
Monitoring of adverse events
13 secondary outcomes
Tmax
Time frame: Up to 16 days
Rac_Cmax
Time frame: Up to 16 days
Cmax
Time frame: Up to 16 days
AUC
Time frame: Up to 16 days
λz
Time frame: Up to 16 days
t1/2
Time frame: Up to 16 days
CL/F
Time frame: Up to 16 days
Vz/F
Time frame: Up to 16 days
Cavg,ss
Time frame: Up to 16 days
Rac_AUC0-tau
Time frame: Up to 16 days
CLss/F
Time frame: Up to 16 days
Vss/F
Time frame: Up to 16 days
Inhibition rate of the AP
Time frame: Up to 16 days
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 8 Apr 2026.
- 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.
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 CMS-D017 Capsule in people with Paroxysmal Nocturnal Hemoglobinuria, PNH and Complement-mediated Kidney Disease, run by Shenzhen Kangzhe Biotechnology Co., Ltd. at 1 site in China.
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 33 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: looking for participants now, as of the sponsor's last update on 8 Apr 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 China. 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?
Contact Yulan Chen (+86 10 6400 9673, chenyulan@cms.net.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of CMS-D017 -Adverse events (time frame: Up to 23 days). 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 8 Apr 2026.