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Phase 2 N=1 Treatment

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Enrolled (actual)
1
Serious AEs
100.0%
Results posted
Jul 2023
Primary outcome: Primary: Measurement of Ratio of LDH to the Upper Limit of Normal (ULN) — 5.6; 1.9 Ratio of LDH:ULN (250 U/L)

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Coversin (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
AKARI Therapeutics
Primary completion
Mar 2018

Outcome Measures

OutcomeResultp-value
PRIMARY
Measurement of Ratio of LDH to the Upper Limit of Normal (ULN)
5.6; 1.9
PRIMARY
Number and Type of Adverse Events (AE)
2; 20; 13; 1; 1; 18
SECONDARY
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
8.2; 7.8; -0.7; 7.8; -0.4; 8.0
SECONDARY
Measurement of Haptoglobin (Hp) at Days 28, 90 and 180, Absolute and Change From Baseline
NA; NA; NA; NA
SECONDARY
Measurement of Lactate Dehydrogenase (LDH) at Baseline, Day 90 and Day 180
5.6; 1.6; 1.5
SECONDARY
Change in Functional Assessment of Chronic Illness Therapy (FACIT) Score at Days 0, 28, 90 and 180
13.0; 13.3; 11.0
SECONDARY
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
16.7; 16.7; 16.7; 6.6; 0.0; 6.6
SECONDARY
Dependency on Blood Transfusion
0; 0

Summary

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.

Eligibility Criteria

Inclusion Criteria

  • Patients with known Paroxysmal Nocturnal Haemoglobinuria (PNH)
  • LDH >=1.5 Upper Limit of Normal (ULN)
  • Resistance to Eculizumab proven by both a recognised C5 polymorphism on genetic screening and complement inhibition on CH50 ELISA of 100kg
  • Pregnancy (females)
  • Failure to satisfy the PI of fitness to participate for any other reason
  • Known allergy to ticks or severe reaction to arthropod venom (e.g., bee or wasp venom)
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02591862). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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