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Trial record 1 of 4 for:    KP104
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To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104

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ClinicalTrials.gov Identifier: NCT05476887
Recruitment Status : Not yet recruiting
First Posted : July 27, 2022
Last Update Posted : July 27, 2022
Sponsor:
Information provided by (Responsible Party):
Kira Pharmacenticals (US), LLC.

Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve subjects 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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: KP104 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Complement Inhibitor-naïve Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Estimated Study Start Date : February 2023
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : September 2024


Arm Intervention/treatment
Experimental: Part 1: Dose escalation Cohort 1
Participants will receive escalating and varying dose intervals of KP104 every week.
Drug: KP104
Participants will receive KP104 intravenously (IV) loading dose followed by maintenance dose every week or biweekly.

Experimental: Part 1: Dose escalation Cohort 2
Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.
Drug: KP104
Participants will receive KP104 intravenously (IV) loading dose followed by maintenance dose every week or biweekly.

Experimental: Part 1: Dose escalation Cohort 3
Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.
Drug: KP104
Participants will receive KP104 intravenously (IV) loading dose followed by maintenance dose every week or biweekly.

Experimental: Part 2: Proof-of-concept Cohort 1
Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly.
Drug: KP104
Participants will receive KP104 intravenously (IV) loading dose followed by maintenance dose every week or biweekly.




Primary Outcome Measures :
  1. Part 1: Number of participants with Dose-limiting toxicities (DLT) [ Time Frame: Up to Week 4 ]
    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.

  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 [ Time Frame: Baseline and at Week 12 ]
    Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion.

  3. Part 2: Percentage of participants with >= 2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion for biweekly dosing [ Time Frame: Baseline and at Week 13 ]
    Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion.


Secondary Outcome Measures :
  1. Part 2: Change from Baseline in serum lactate dehydrogenase (LDH) levels for weekly dosing [ Time Frame: Baseline and at Week 12 ]
    Blood samples will be collected for the analysis of serum LDH.

  2. Part 2: Change from Baseline in serum LDH levels for biweekly dosing [ Time Frame: Baseline and at Week 13 ]
    Blood samples will be collected for the analysis of serum LDH.

  3. Part 2: Change from Baseline in hemoglobin level for weekly dosing [ Time Frame: Baseline and at Week 12 ]
    Blood samples will be collected for the analysis of hemoglobin level

  4. Part 2: Change from Baseline in the hemoglobin level for biweekly dosing [ Time Frame: Baseline and at Week 13 ]
    Blood samples will be collected for the analysis of hemoglobin level.

  5. Part 2: Change from Baseline in red blood cell (RBC) transfusion dependence for weekly dosing [ Time Frame: Baseline and at Week 12 ]
    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.

  6. Part 2: Change in RBC transfusion dependence for biweekly dosing [ Time Frame: Baseline and at Week 13 ]
    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.

  7. Part 1 and 2: Number of participants reporting Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 21 ]
    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.

  8. Part 1 and 2: Number of participants reporting Treatment Emergent Serious Adverse Events (TESAEs) [ Time Frame: Up to Week 21 ]
    A TESAE is defined as any AE that started or worsened in severity on or after the first dose of study treatment.

  9. Part 1 and 2: Number of participants reporting AEs of Special interests (AESIs) [ Time Frame: Up to Week 21 ]
    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.

  10. Part 1 and 2: Maximum concentration (Cmax) of KP104 for weekly dosing [ Time Frame: Up to Week 20 ]
  11. Part 1 and 2: Cmax of KP104 for biweekly dosing [ Time Frame: Up to Week 21 ]
  12. Part 1 and 2: Trough concentration (Ctrough) of KP104 for weekly dosing [ Time Frame: Up to Week 20 ]
  13. Part 1 and 2: Ctrough of KP104 for biweekly dosing [ Time Frame: Up to Week 21 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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 × 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.

Exclusion Criteria:

  • 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 NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05476887


Contacts
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Contact: Study Director privacy@kirapharma.com

Locations
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China
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
Sponsors and Collaborators
Kira Pharmacenticals (US), LLC.
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Responsible Party: Kira Pharmacenticals (US), LLC.
ClinicalTrials.gov Identifier: NCT05476887    
Other Study ID Numbers: KP104-201
First Posted: July 27, 2022    Key Record Dates
Last Update Posted: July 27, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Kira Pharmacenticals (US), LLC.:
KP104
Paroxysmal nocturnal hemoglobinuria
Complement Inhibitor
Dose Selection
Proof of Concept
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases