ClinicalTrials.gg
Sign In
Active, not recruitingNCT05476887Updated Oct 28, 2024

To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104

A Phase 2 interventional study of KP104 in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Kira Pharmacenticals (US), LLC.. Active, not recruiting at 4 sites in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-28.

Sponsored by Kira Pharmacenticals (US), LLC. · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Nov 2024, 1 year 11 months ago, but the record still lists the study as active, not recruiting.
Phase
Phase 2
Study type
Interventional
Enrollment
35
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH. The study will be conducted in 2 parts. Part 1 is a dose-selection study to assess escalating doses and varying dose intervals of KP104. Part 2 is a proof-of-concept (POC) study assessing the efficacy of the optimal intravenous (IV) loading dose followed by the optimal maintenance dose and regimen of KP104. Participants who complete the Initial Treatment Period and demonstrate benefit from KP104 will be eligible for a 9-month open-label extension (OLE) treatment period.

02

Conditions studied

  • Paroxysmal Nocturnal Hemoglobinuria

Keywords

  • KP104
  • Paroxysmal nocturnal hemoglobinuria
  • Complement Inhibitor
  • Dose Selection
  • Proof of Concept
03

In context

Hemoglobinuria, Paroxysmal

192 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.

This study's enrollment of 35 is close to the median of 34 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.

Browse Hemoglobinuria, Paroxysmal studies →

Lead sponsor

Kira Pharmacenticals (US), LLC. is the lead sponsor of 4 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Initial Treatment Period:

  • Documented diagnosis of PNH confirmed by flow cytometry evaluation of white blood cells and red blood cells, with granulocyte or monocyte clone size of >= 10 percent (%) within 6 months of the Screening visit.
  • Presence of 1 or more PNH-related signs or symptoms within 3 months of initiation of Screening.
  • LDH >= 2.0 × upper limit of normal (ULN) at screening.
  • Hemoglobin \<= 10.0 g/dL at screening.
  • 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.

Extension Treatment Period (OLE):

  • Complete the 12-week (weekly dosing) or 13-week (biweekly dosing) Initial Treatment Period per the protocol.
  • Benefited from KP104 treatment and will continue benefiting from KP104 treatment per the investigator's judgement.
  • Willing to participate in Extension Treatment Period, able to comply with this protocol and be available for the entire duration of the study.

Exclusion criteria

Exclusion Criteria:

Initial Treatment Period:

  • Any clinically significant poorly controlled underlying illness other than PNH per discretion of investigators.
  • Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibiotics, antivirals, or antifungals.
  • History of meningococcal infection.
  • History of untreated tuberculosis.
  • History of splenectomy
  • Positive serology for Hepatitis C Virus (HCV) ribonucleic acid (RNA) or human immunodeficiency virus (HIV) at Screening
  • History of bone marrow or stem cell transplantation
  • Absolute neutrophil count (ANC) \<500 cells per microliter (cells/μL)
  • Reticulocyte count\< 100 x 10\^3 cells/μL
  • Platelet count\< 30,000 cells/μL
  • History of systemic autoimmune disease
  • Estimated glomerular filtration rate (eGFR) \<30 milliliters/minute/1.73 meter square (mL/min/1.73 m\^2) calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.

Extension Treatment Period (OLE):

  • Women who are pregnant.
  • Women of childbearing potential and men with sexual partners of childbearing potential who are not using adequate contraception and who are not willing to use adequate contraception.
  • Any medical condition that, in the opinion of the investigator, may pose a safety risk to a participant in this study, or may interfere with study participation.

Other protocol defined Inclusion/Exclusion criteria may apply.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
35 participants (actual)

Study arms

  • Experimental
    Part 1: Dose escalation Cohort 1

    Participants will receive escalating and varying dose intervals of KP104 every week.

    Drug: KP104

  • Experimental
    Part 1: Dose escalation Cohort 2

    Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.

    Drug: KP104

  • Experimental
    Part 1: Dose escalation Cohort 3

    Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.

    Drug: KP104

  • Experimental
    Part 2: Proof-of-concept Cohort 1

    Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.

    Drug: KP104

  • Experimental
    Open-label extension (OLE)

    Participants will receive KP104, who benefit from KP104 treatment in Part 1 and 2

    Drug: KP104

Interventions

  • DrugKP104

    KP104 intravenously (IV loading + subcutaneous \[SC\] maintenance every week \[QW\] or every 2 weeks \[Q2W\]) will be administered.

06

What researchers measure

Primary outcomes

  1. Part 1: 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.

    Time frame: Up to Week 4

  2. Part 2: Percentage of participants with >= 2 grams per deciliter (g/dL) increase in hemoglobin level from Baseline in the absence of transfusion for weekly dosing

    Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion.

    Time frame: Baseline and at Week 12

  3. Part 2: Percentage of participants with >= 2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion for biweekly dosing

    Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion.

    Time frame: Baseline and at Week 13

  4. Open-label Extension (OLE): Number of participants reporting Treatment Emergent Adverse Events (TEAEs), treatment-emergent serious adverse events (TESAEs) and AEs of special interest (AESIs)

    Time frame: Up to 9 months

Secondary outcomes

  1. Part 2: Change from Baseline in serum lactate dehydrogenase (LDH) levels for weekly dosing

    Blood samples will be collected for the analysis of serum LDH.

    Time frame: Baseline and at Week 12

  2. Part 2: Change from Baseline in serum LDH levels for biweekly dosing

    Blood samples will be collected for the analysis of serum LDH.

    Time frame: Baseline and at Week 13

  3. Part 2: Change from Baseline in hemoglobin level for weekly dosing

    Blood samples will be collected for the analysis of hemoglobin level

    Time frame: Baseline and at Week 12

  4. Part 2: Change from Baseline in the hemoglobin level for biweekly dosing

    Blood samples will be collected for the analysis of hemoglobin level.

    Time frame: Baseline and at Week 13

  5. Part 2: Change from Baseline in red blood cell (RBC) transfusion dependence for weekly dosing

    The RBC transfusion dependence is the difference in the volume of RBC transfusions per month for the 3 months prior to initiation of investigational product versus the volume of RBC transfusions per month for each month on study.

    Time frame: Baseline and at Week 12

  6. Part 2: Change in RBC transfusion dependence for biweekly dosing

    The RBC transfusion difference is the difference in the volume of RBC transfusions per month for the 3 months prior to initiation of investigational product versus the volume of RBC transfusions per month for each month on study.

    Time frame: Baseline and at Week 13

  7. Part 1 and 2: Number of participants reporting TEAEs, TESAEs and AESIs

    Time frame: Up to Week 13

  8. Part 1 and 2: Concentration within one hour of end of infusion (CEOI) of KP104

    Time frame: Up to Week 13

  9. Part 1 and 2: Trough concentration (Ctrough) of KP104

    Time frame: Up to Week 13

07

Study locations

4 sites
  • Peking Union Medical College Hospital
    Beijing, China
  • Jiangsu Province Hospital
    Nanjing, China
  • Chinese Academy of Medical Sciences Peking Union Medical College - Institute of Hematology Blood Diseases Hospital
    Tianjin, China
  • Henan Cancer Hospital
    Zhengzhou, China
08

References and documents

Individual participant data

Plan to share: Yes

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 28, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05476887
Lead sponsor
Kira Pharmacenticals (US), LLC.
Responsible party
Sponsor
First posted
Jul 27, 2022
Start date
Nov 25, 2022
Primary completion
Nov 2024 (estimated)
Completion
Feb 2025 (estimated)
Last update
Oct 28, 2024

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Vensobafusp alfa

Other studies in this condition

Hemoglobinuria, Paroxysmal →

Other studies from this sponsor

Kira Pharmacenticals (US), LLC.

Start the discussion