Paroxysmal Nocturnal HemoglobinuriaPhase 1NCT05490017
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
A Phase 1 study testing KP104 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Kira Pharmacenticals (US), LLC. at 1 site in Australia.
Trial summary
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of KP104 and Part 2, multiple ascending dose… 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
- What is being tested?
- KP104
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 1 siteAustralia
- Study dates
- 30 Dec 2020to 2 Sep 2022
- 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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Weight of > 40 kilograms (kg) and < 120 kg at Screening.
- In good general health, determined by no clinically significant findings in the opinion of the Investigator from medical history, physical examination, 12-lead ECG, clinical laboratory findings, and vital signs at Screening and Check-in.
- Hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and platelet count results within the normal range at the Screening Visit; participants with Gilbert's disease with associated abnormalities of liver function tests are eligible for enrollment. Tests may be repeated at the discretion of the Investigator to confirm abnormalities.
- Creatinine clearance based on the Cockcroft-Gault equation of >= 80 milliliters per minute (ml/min).
- Females of childbearing potential and males must practice effective contraception from Screening until 28 days after the end of study (EOS) visit.
- Females of childbearing potential must have a negative pregnancy test at Screening and within 24 hours prior to dosing of study drug; for post-menopausal subjects, a blood sample will also be tested for follicle stimulating hormone to confirm post-menopausal status.
Who cannot join
- Any clinically significant underlying illness in the opinion of the Investigator.
- Any history or sign of significant chronic active or recurrent infection, or screening laboratory evidence consistent with a significant chronic active or recurrent infection requiring treatment with antibacterials, antivirals, or antifungals.
- Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibacterials, antivirals, or antifungals.
- History of clinically significant hematologic or bone marrow disease or blood dyscrasias.
- History of meningococcal infection.
- History of tuberculosis.
Show all 13Show fewer
- History of asplenia (functional or anatomical).
- Prior exposure to KP104.
- Known allergy to penicillin antibiotics or history of allergy or contraindication to required prophylactic antibiotic therapy to be used during the study.
- Known or suspected complement deficiency during screening.
- Positive serology for Hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) at Screening.
- History of drug or alcohol abuse within 1 year of Screening in the opinion of the investigator, or a positive test for drugs of abuse or alcohol at Screening or Check-in.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: SYNERGY-1: A Phase 1 First-in-human, Randomized, Double Blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 in Healthy Subjects
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of KP104 and Part 2, multiple ascending dose (MAD).
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- KP104, Placebo
- Keywords
- KP104, Paroxysmal Nocturnal Hemoglobinuria, Single ascending Dose, Multiple Ascending Dose, First in Human, Complement Inhibitor, Healthy participants
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KP104Drug
Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose.
PlaceboDrug
Participants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL).
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Single Ascending Dose Cohort 1 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 2 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 3 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 4 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 5 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 6 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 7 | Experimental | Not described Receives: Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 1 | Experimental | Not described Receives: Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 2 | Experimental | Not described Receives: Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 3 | Experimental | Not described Receives: Drug: KP104 |
| Part 1: Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| Part 2: Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
An Adverse event (AE) is defined as any unfavorable and unintended sign including an abnormal laboratory finding), symptom, or disease or any worsening of a pre-existing condition temporally associated with the use of a study drug, whether or not related to study drug. A TEAE is defined as any AE that started or worsened in severity on or after the first dose of study treatment.
Measured: Up to Day 85
Number of participants reporting Treatment Emergent Serious Adverse Events (TESAEs)
A TESAE is defined as any AE that started or worsened in severity on or after the first dose of study treatment.
Measured: Up to Day 85
Number of participants with Dose-limiting toxicities (DLT)
A DLT is defined as any adverse event considered by the investigator to be KP104-related with a severity greater than or equal to (>=) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 Grade 3 which also represents a shift from baseline clinical status of > 1 NCI CTCAE grade. A hypersensitivity/administration reaction occurring with a severity of Grade 2 despite the use of pre-medications will also be designated as a DLT.
Measured: Up to Day 85
Number of participants reporting AEs of Special interests (AESIs)
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease or any worsening of a pre-existing condition temporally associated with the use of a study drug, whether or not related to study drug. Number of participants with AESIs including infections and local or systemic administration reactions will be assessed.
Measured: Up to Day 85
Secondary outcomes (4)
Maximum concentration (Cmax) of KP104
Measured: Up to Day 29
Area under the concentration-time profile (AUC) of KP104
Measured: Up to Day 29
Change from baseline in total and free serum C5 levels
Measured: Baseline and up to Day 29
Change from baseline in rabbit red blood cell (RBC) assay
Measured: Baseline and up to Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2022
- Study start30 Dec 2020
- Primary completion4 May 2022
- Study completion2 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kira Pharmacenticals (US), LLC.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05490017
- Sponsor's protocol ID
- KP104-101
- Status last verified
- Feb 2023
- First submitted
- 4 Aug 2022
- Last updated
- 1 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SYNERGY-1: A Phase 1 First-in-human, Randomized, Double Blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT05490017
- Acronym / sponsor ID
- KP104-101
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- KP104, Paroxysmal Nocturnal Hemoglobinuria, Single ascending Dose, Multiple Ascending Dose, First in Human, Complement Inhibitor, Healthy participants
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Kira Pharmacenticals (US), LLC.Industry (pharma, biotech, devices)
- Study start
- 30 Dec 2020 (actual)
- Primary completion
- 4 May 2022 (actual)
- Study completion
- 2 Sep 2022 (actual)
- First posted
- 5 Aug 2022 (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 study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of KP104 and Part 2, multiple ascending dose (MAD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 12
| Arm | Type | Receives |
|---|---|---|
| Part 1: Single Ascending Dose Cohort 1 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 2 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 3 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 4 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 5 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 6 | Experimental | Drug: KP104 |
| Part 1: Single Ascending Dose Cohort 7 | Experimental | Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 1 | Experimental | Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 2 | Experimental | Drug: KP104 |
| Part 2: Multiple Ascending Dose Cohort 3 | Experimental | Drug: KP104 |
| Part 1: Placebo | Placebo comparator | Drug: Placebo |
| Part 2: Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Weight of > 40 kilograms (kg) and < 120 kg at Screening.
- In good general health, determined by no clinically significant findings in the opinion of the Investigator from medical history, physical examination, 12-lead ECG, clinical laboratory findings, and vital signs at Screening and Check-in.
- Hemoglobin, hematocrit, white blood cell count, absolute neutrophil count, and platelet count results within the normal range at the Screening Visit; participants with Gilbert's disease with associated abnormalities of liver function tests are eligible for enrollment. Tests may be repeated at the discretion of the Investigator to confirm abnormalities.
- Creatinine clearance based on the Cockcroft-Gault equation of >= 80 milliliters per minute (ml/min).
- Females of childbearing potential and males must practice effective contraception from Screening until 28 days after the end of study (EOS) visit.
Key exclusion criteria (13 listed)
- Any clinically significant underlying illness in the opinion of the Investigator.
- Any history or sign of significant chronic active or recurrent infection, or screening laboratory evidence consistent with a significant chronic active or recurrent infection requiring treatment with antibacterials, antivirals, or antifungals.
- Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibacterials, antivirals, or antifungals.
- History of clinically significant hematologic or bone marrow disease or blood dyscrasias.
- History of meningococcal infection.
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 |
|---|---|---|
| KP104Participants will receive KP104 intravenous (IV) dose approximately for 1 hour or subcutaneous (SC) dose. | Drug | Part 1: Single Ascending Dose Cohort 1, Part 1: Single Ascending Dose Cohort 2, Part 1: Single Ascending Dose Cohort 3, Part 1: Single Ascending Dose Cohort 4, Part 1: Single Ascending Dose Cohort 5, Part 1: Single Ascending Dose Cohort 6, Part 1: Single Ascending Dose Cohort 7, Part 2: Multiple Ascending Dose Cohort 1, Part 2: Multiple Ascending Dose Cohort 2, Part 2: Multiple Ascending Dose Cohort 3 |
| PlaceboParticipants will receive matching placebo which is KP104 vehicle containing sodium phosphate, sodium chloride, and L-Lysine Hydrochloride (L-Lys-HCL). | Drug | Part 1: Placebo, Part 2: Placebo |
Primary outcome
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to Day 85
An Adverse event (AE) is defined as any unfavorable and unintended sign including an abnormal laboratory finding), symptom, or disease or any worsening of a pre-existing condition temporally associated with the use of a study drug, whether or not related to study drug. A TEAE is defined as any AE that started or worsened in severity on or after the first dose of study treatment.
Number of participants reporting Treatment Emergent Serious Adverse Events (TESAEs)
Time frame: Up to Day 85
A TESAE is defined as any AE that started or worsened in severity on or after the first dose of study treatment.
Number of participants with Dose-limiting toxicities (DLT)
Time frame: Up to Day 85
A DLT is defined as any adverse event considered by the investigator to be KP104-related with a severity greater than or equal to (>=) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 Grade 3 which also represents a shift from baseline clinical status of > 1 NCI CTCAE grade. A hypersensitivity/administration reaction occurring with a severity of Grade 2 despite the use of pre-medications will also be designated as a DLT.
Number of participants reporting AEs of Special interests (AESIs)
Time frame: Up to Day 85
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease or any worsening of a pre-existing condition temporally associated with the use of a study drug, whether or not related to study drug. Number of participants with AESIs including infections and local or systemic administration reactions will be assessed.
4 secondary outcomes
Maximum concentration (Cmax) of KP104
Time frame: Up to Day 29
Area under the concentration-time profile (AUC) of KP104
Time frame: Up to Day 29
Change from baseline in total and free serum C5 levels
Time frame: Baseline and up to Day 29
Change from baseline in rabbit red blood cell (RBC) assay
Time frame: Baseline and up to Day 29
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 1 Mar 2023.
- Individual participant dataSharing planned: Yes
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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 KP104 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Kira Pharmacenticals (US), LLC. at 1 site in Australia.
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 6 inclusion and 13 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 1 Mar 2023. 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 Australia. 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?
Number of participants reporting Treatment Emergent Adverse Events (TEAEs) (time frame: Up to Day 85). The study lists 4 primary outcomes in all. 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 1 Mar 2023.