Who is sponsoring NCT03030183?

NCT03030183 is sponsored by Ra Pharmaceuticals.

What drugs are being tested in NCT03030183?

Interventions in NCT03030183: Zilucoplan (RA101495).

What condition does NCT03030183 study?

NCT03030183 studies Paroxysmal Nocturnal Hemoglobinuria (PNH).

Is NCT03030183 still recruiting?

NCT03030183 is currently completed. Last updated 27 July 2022.

Are results published for NCT03030183?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-07-27 · How we verify

NCT03030183

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

Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

Completed Phase 2 Results posted Last updated 27 July 2022

What this trial tests

Phase 2 trial testing Zilucoplan (RA101495) in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 3 participants. Completed in 28 March 2018.

Timeline

17 April 2017

Primary endpoint
28 March 2018

28 March 2018

Quick facts

Lead sponsor	Ra Pharmaceuticals
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	3
Start date	17 April 2017
Primary completion	28 March 2018
Estimated completion	28 March 2018
Sites	6 locations across United States

Drugs / interventions tested

Zilucoplan (RA101495) — full drug profile →

Conditions studied

Paroxysmal Nocturnal Hemoglobinuria (PNH) — all drugs for Paroxysmal Nocturnal Hemoglobinuria (PNH) →

Sponsor

Ra Pharmaceuticals — full company profile →

Who can join

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

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. Primary · Through Week 12 of the study

Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels

Group	Value	95% CI
Zilucoplan (RA101495)	100.3	± 128.0

Change-from-baseline Bilirubin Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	-3.3	± 5.1

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	-4.7	± 12.0

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	-4.3	± 7.5

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	-0.3	± 2.9

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	-6.7	± 17.5

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	-2.3	± 12.5

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	-7.7	± 14.8

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	-2.0	± 6.2

Change-from-baseline Total Hemoglobin Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	5.7	± 4.2

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	3.3	± 3.8

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	0.3	± 4.0

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	-0.3	± 7.2

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	0.7	± 5.8

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	-3.3	± 4.2

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	2.3	± 3.1

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	0.3	± 2.5

Change-from-baseline Free Hemoglobin Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	-145.90

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	-140.60

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	-147.70

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	-138.40

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	-147.30

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	-148.10

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	-143.50

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	-144.30

Change-from-baseline Haptoglobin Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	0.133	± 0.231

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	0.000	± 0.000

Change-from-baseline Reticulocyte Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	-0.0190	± 0.0382

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	0.0090	± 0.0052

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	0.0430	± 0.0327

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	0.0477	± 0.0499

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	0.0310	± 0.0157

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	0.0253	± 0.0248

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	0.0350	± 0.0350

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	0.0190	± 0.0092

Change-from-baseline Hemoglobinuria Values Secondary · Through week 12

Changes from baseline at each of the scheduled postbaseline time-points Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10. Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.

Week 1

Group	Value	95% CI
Zilucoplan (RA101495)	0.0

Week 2

Group	Value	95% CI
Zilucoplan (RA101495)	0.5	± 0.7

Week 3

Group	Value	95% CI
Zilucoplan (RA101495)	0.5	± 0.7

Week 4

Group	Value	95% CI
Zilucoplan (RA101495)	1.0	± 1.4

Week 6

Group	Value	95% CI
Zilucoplan (RA101495)	0.5	± 0.7

Week 8

Group	Value	95% CI
Zilucoplan (RA101495)	2.0

Week 10

Group	Value	95% CI
Zilucoplan (RA101495)	2.3	± 2.5

Week 12

Group	Value	95% CI
Zilucoplan (RA101495)	-0.3	± 2.1

Adverse events — posted to ClinicalTrials.gov

Time frame: Through week 12 of the Study. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Zilucoplan (RA101495)

Serious: 0/3 (0%)

Deaths: 0/3

Other adverse events (8 terms — click to expand)

Reaction	System	Zilucoplan (RA101495)
Nausea	Gastrointestinal disorders	—
Influenza like illness	General disorders	—
Lipase increased	Investigations	—
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—
Headache	Nervous system disorders	—
Haemoglobinuria	Renal and urinary disorders	—
Nephrolithiasis	Renal and urinary disorders	—

Data from ClinicalTrials.gov NCT03030183 adverse events section.

Sponsor's own description

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.

Publications & conference data

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

New insights into the immune functions of complement.
Reis ES, Mastellos DC, Hajishengallis G, Lambris JD. · · 2019 · cited 357× · PMID 31048789 · DOI 10.1038/s41577-019-0168-x
The renaissance of complement therapeutics.
Ricklin D, Mastellos DC, Reis ES, Lambris JD. · · 2018 · cited 305× · PMID 29199277 · DOI 10.1038/nrneph.2017.156
Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT.
Risitano AM, Marotta S, Ricci P, Marano L, et al · · 2019 · cited 155× · PMID 31258525 · DOI 10.3389/fimmu.2019.01157
Diseases of complement dysregulation-an overview.
Wong EKS, Kavanagh D. · · 2018 · cited 80× · PMID 29327071 · DOI 10.1007/s00281-017-0663-8
Cyclic Peptides in Pipeline: What Future for These Great Molecules?
Costa L, Sousa E, Fernandes C. · · 2023 · cited 50× · PMID 37513908 · DOI 10.3390/ph16070996
Expanding horizons in complement drug discovery: challenges and emerging strategies.
Harris CL. · · 2018 · cited 47× · PMID 28986638 · DOI 10.1007/s00281-017-0655-8
Treatment of Rare Inflammatory Kidney Diseases: Drugs Targeting the Terminal Complement Pathway.
Anliker-Ort M, Dingemanse J, van den Anker J, Kaufmann P. · · 2020 · cited 33× · PMID 33362783 · DOI 10.3389/fimmu.2020.599417
Emerging Role of C5 Complement Pathway in Peripheral Neuropathies: Current Treatments and Future Perspectives.
Giorgio C, Zippoli M, Cocchiaro P, Castelli V, et al · · 2021 · cited 29× · PMID 33917266 · DOI 10.3390/biomedicines9040399

Verify or expand the search:

Other trials of Zilucoplan (RA101495)

Trials testing the same drug.

NCT03225287 — Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study · Phase 2 · terminated
NCT03078582 — Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients · Phase 2 · completed

Other recruiting trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Currently open trials in the same condition.

NCT07187401 — A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in · Phase 1, PHASE2 · recruiting
NCT07229235 — REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria · recruiting
NCT06934967 — Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients · Phase 3 · recruiting
NCT06312644 — Study of Ultomiris® (Ravulizumab) Safety in Pregnancy · recruiting
NCT06933914 — Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria · Phase 2, PHASE3 · recruiting

Other Ra Pharmaceuticals trials

Trials by the same sponsor.

NCT04025632 — Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy · Phase 2 · terminated
NCT03315130 — Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis · Phase 2 · completed
NCT03078582 — Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients · Phase 2 · completed

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 NCT03030183 (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 Ra Pharmaceuticals
Last refreshed: 27 July 2022

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/NCT03030183.

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

NCT03030183

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Sponsor's own description

Publications & conference data

Related trials

Other trials of Zilucoplan (RA101495)

Other recruiting trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Other Ra Pharmaceuticals trials

Verify against primary sources