NCT06051357

Brief Summary

This is a multicenter, randomized, open-label phase II clinical trial. A total of 24 patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HRS-5965 for 12 weeks.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
26

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2023

Shorter than P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 5, 2023

Completed
17 days until next milestone

First Posted

Study publicly available on registry

September 22, 2023

Completed
2 months until next milestone

Study Start

First participant enrolled

November 15, 2023

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 25, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 25, 2024

Completed
Last Updated

October 15, 2024

Status Verified

October 1, 2024

Enrollment Period

4 months

First QC Date

September 5, 2023

Last Update Submit

October 10, 2024

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

  • Changes in hemoglobin.

    12 weeks

Secondary Outcomes (10)

  • Changes in LDH.

    12 weeks

  • Changes in haptoglobin.

    12 weeks

  • Changes in bilirubin.

    12 weeks

  • Changes in reticulocyte counts.

    12weeks

  • Changes in C3 complement fragment deposition.

    12 week

  • +5 more secondary outcomes

Study Arms (2)

Treatment group A

EXPERIMENTAL
Drug: HRS-5965 tablets

Treatment group B

EXPERIMENTAL
Drug: HRS-5965 tablets

Interventions

HRS-5965 tabletsDRUG

HRS-5965 tablets for 12 weeks

Treatment group ATreatment group B

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of PNH confirmed by flow cytometry with clone size ≥ 10%.
  • Have not received complement inhibitor therapy ;
  • LDH \> 1.5×ULN;
  • Hemoglobin level \< 10 g/dL.

You may not qualify if:

  • Known or suspected hereditary or acquired complement deficiency;
  • Patients with laboratory evidence of bone marrow failure (reticulocytes \<100x109/L; platelets \<30x109/L; neutrophils \<0.5x109/L);
  • Presence or suspicion of a systemic active bacterial, viral, or fungal infection (based on judgment of the investigator) within 2 weeks prior to the first dose of HRS-5965;
  • History of infection with capsular bacteria (e.g., meningococcus, pneumococcus, etc.)
  • Positive of HIV, HBsAg or HCVAb.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, 100730, China

Location

The Blood Disease Hospital of the Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 5, 2023

First Posted

September 22, 2023

Study Start

November 15, 2023

Primary Completion

March 25, 2024

Study Completion

March 25, 2024

Last Updated

October 15, 2024

Record last verified: 2024-10

Locations

CN(2)