NCT00130000 is a Phase 3 clinical trial of Eculizumab in Paroxysmal Hemoglobinuria, Nocturnal, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

Who is sponsoring NCT00130000?

NCT00130000 is sponsored by National Heart, Lung, and Blood Institute (NHLBI).

What drugs are being tested in NCT00130000?

Interventions in NCT00130000: Eculizumab.

What condition does NCT00130000 study?

NCT00130000 studies Paroxysmal Hemoglobinuria, Nocturnal.

Is NCT00130000 still recruiting?

NCT00130000 is currently completed. Last updated 2 July 2017.

Last reviewed 2017-07-02 · How we verify

NCT00130000

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria

Completed Phase 3 Last updated 2 July 2017

What this trial tests

Phase 3 trial testing Eculizumab in Paroxysmal Hemoglobinuria, Nocturnal in 87 participants. Completed in 25 June 2007.

Timeline

9 August 2005

25 June 2007

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Primary purpose	treatment
Enrollment	87
Start date	9 August 2005
Estimated completion	25 June 2007
Sites	1 location across United States

Drugs / interventions tested

Eculizumab (ECULIZUMAB) — full drug profile →

Conditions studied

Paroxysmal Hemoglobinuria, Nocturnal — all drugs for Paroxysmal Hemoglobinuria, Nocturnal →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

18 and older, any sex, with Paroxysmal Hemoglobinuria, Nocturnal. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study will evaluate the safety and effectiveness of the experimental drug eculizumab for treating paroxysmal nocturnal hemoglobinuria (PNH), a disorder that can cause premature destruction of red blood cells. PNH may result in anemia requiring blood transfusions. Patients may be at high risk of life-threatening blood clots in their veins and may have urine discoloration, stomach pain, difficulty swallowing, tiredness, and poor quality of life. Men may have problems getting or maintaining an erection. Eculizumab is a monoclonal antibody that may improve the survival of red blood cells in patients with PNH. Patients 18 years of age and older who have been diagnosed with PNH for more than 6 months, who have active disease, and who require blood transfusions may be eligible for this study. Each candidate is screened with a physical examination, electrocardiogram, blood and urine tests, and a questionnaire for information on how PNH affects the patient physically, socially, emotionally, and functionally. Participants receive infusions of eculizumab through a needle in a vein once a week for five doses and then every two weeks for another 24 doses. All patients are vaccinated against Neisseria meningitides, a bacteria that can cause symptoms, possibly including life-threatening meningitis, in susceptible people, including people who take eculizumab. At every treatment visit, patients update their health status, transfusion record, and medication use; review their laboratory results from the preceding visit; have vital signs measured; and provide a blood sample for laboratory tests. At selected visits, they also provide a urine sample, have a repeat electrocardiogram, and complete a questionnaire. At the final treatment visit, participants have a complete physical examination, in addition to the routine procedures.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria.
Brodsky RA, Young NS, Antonioli E, Risitano AM, et al · · 2008 · cited 459× · PMID 18055865 · DOI 10.1182/blood-2007-06-094136
Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria.
Hillmen P, Muus P, Röth A, Elebute MO, et al · · 2013 · cited 290× · PMID 23617322 · DOI 10.1111/bjh.12347
Pharmacokinetic and pharmacodynamic effects of ravulizumab and eculizumab on complement component 5 in adults with paroxysmal nocturnal haemoglobinuria: results of two phase 3 randomised, multicentre studies.
Peffault de Latour R, Brodsky RA, Ortiz S, Risitano AM, et al · · 2020 · cited 51× · PMID 32449174 · DOI 10.1111/bjh.16711
The importance of terminal complement inhibition in paroxysmal nocturnal hemoglobinuria.
Kulasekararaj AG, Brodsky RA, Nishimura JI, Patriquin CJ, et al · · 2022 · cited 24× · PMID 35663504 · DOI 10.1177/20406207221091046
Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations.
Bodó I, Amine I, Boban A, Bumbea H, et al · · 2023 · cited 15× · PMID 37072660 · DOI 10.1007/s12325-023-02510-4
Halting targeted and collateral damage to red blood cells by the complement system.
Jalink M, de Boer ECW, Evers D, Havinga MQ, et al · · 2021 · cited 13× · PMID 34191092 · DOI 10.1007/s00281-021-00859-8
Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.
Schrezenmeier H, Kulasekararaj A, Mitchell L, de Latour RP, et al · · 2024 · cited 8× · PMID 37804344 · DOI 10.1007/s00277-023-05483-0
Paroxysmal Nocturnal Hemoglobinuria: Biology and Treatment.
Bravo-Perez C, Guarnera L, Williams ND, Visconte V. · · 2023 · cited 7× · PMID 37763731 · DOI 10.3390/medicina59091612

Verify or expand the search:

Other trials of Eculizumab

Trials testing the same drug.

NCT06764160 — Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG · Phase 3 · completed
NCT06513338 — Complement C5 mAb in the Treatment of Anti-GBM Disease · Phase 2 · completed
NCT05876351 — Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China · Phase 3 · completed
NCT05886244 — Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China · Phase 3 · completed
NCT05131204 — Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment · Phase 3 · terminated

Other recruiting trials for Paroxysmal Hemoglobinuria, Nocturnal

Currently open trials in the same condition.

NCT03157635 — Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Partici · Phase 1, PHASE2 · active not recruiting

Other National Heart, Lung, and Blood Institute (NHLBI) trials

Trials by the same sponsor.

NCT07566494 — Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease · Phase 1 · not yet recruiting
NCT07137455 — EDEN Intracardiac Electrogram Recording and Classifying System · NA · enrolling by invitation
NCT05372627 — NHLBI-Emory Advanced Cardiac CT Reconstruction · not yet recruiting
NCT07516379 — GRAfT 2.0. A Multimodal Prospective Approach to Define the Mechanisms and Clinical Features of Acute and Chronic Rejecti · not yet recruiting
NCT06948097 — Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fost · Phase 1 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT00130000 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
Last refreshed: 2 July 2017

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00130000.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing