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ClinicalTrials.gov 4 recruiting now full pipeline small index

Paroxysmal Nocturnal Hemoglobinuria (PNH): full-pipeline trial registry

4 of 6 indexed Paroxysmal Nocturnal Hemoglobinuria (PNH) trials are currently recruiting.

Corpus placement: #2,866 of 28,707 conditions by registered trial count.

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6 US clinical trials · 4 currently recruiting · #2,866 of 28,707 conditions by registered trial count

Paroxysmal Nocturnal Hemoglobinuria (PNH): 6 US clinical trials tracked, 4 recruiting.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is the subject of 6 registered US clinical trials on ClinicalTrials.gov, 4 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is Alexion Pharmaceuticals, running 3 of these trials.

Enrollment posture brief

Registry condition paroxysmal-nocturnal-hemoglobinuria-pnh

OPEN 4 | RECR 67% | LATE 3 | EARLY 2 | RANK #2866 | SPON 3

Volume-matched condition peers

Nearest open-share peer: Adenocarcinoma of Lung (67% recruiting · 6 trials)

Paroxysmal Nocturnal Hem (th…67%Acute Hypoxemic Respiratory 66.7%Abdominal Aortic Aneurysms50%Acupuncture50%Acute Lymphoblastic Leukemia50%
Open-enrollment share among volume-matched conditions near Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH) lists 4 open slots (67% of 6 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →

Key findings

What ClinicalTrials.gov does not surface for Paroxysmal Nocturnal Hemoglobinuria (PNH) on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.

  1. Paroxysmal Nocturnal Hemoglobinuria (PNH) is recruiting above its size-band peers.

    4 of 6 indexed trials (66.7%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. One sponsor concentrates Paroxysmal Nocturnal Hemoglobinuria (PNH) research.

    Alexion Pharmaceuticals accounts for 3 of 6 trials (50%). 4 distinct sponsors appear on at least one study for this condition label.

  3. Most Paroxysmal Nocturnal Hemoglobinuria (PNH) trials plan to enrol fewer than 100 people.

    Median target enrolment is 75 among the 6 trials that report a genuine target (sentinel values excluded). 4 of those (66.7%) plan for fewer than 100 participants.

  4. Paroxysmal Nocturnal Hemoglobinuria (PNH) research is mostly interventional.

    5 interventional and 1 observational studies make up this index (83.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 22Phase 33

Phase Distribution

PhaseTrial count
Phase 2 2
Phase 3 3

Top Sponsors

Alexion Pharmaceuticals 3 trials
Apellis Pharmaceuticals 1 trials
National Heart, Lung, and Blood Institute (NHLBI) 1 trials
Novartis Pharmaceuticals 1 trials

Named interventions

Most-linked intervention names on Paroxysmal Nocturnal Hemoglobinuria (PNH) records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Eculizumab Biological
2 trials
Ravulizumab Biological
2 trials
Peripheral Blood Stem Cells Other
1 trials
LNP023 Drug
1 trials
Ultomiris Drug
1 trials
Pegcetacoplan Drug
1 trials
Cyclophosphamide Drug
1 trials

US sites by state

Facility locations filed on Paroxysmal Nocturnal Hemoglobinuria (PNH) trials. A trial with sites in several states counts in each.

Maryland 2 trials
California 2 trials
Texas 1 trials
Pennsylvania 1 trials
New York 1 trials
New Jersey 1 trials
Michigan 1 trials
Massachusetts 1 trials

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Related

Read our methodology - how this data is sourced, computed, and verified.

What to do with this Paroxysmal Nocturnal Hemoglobinuria (PNH) page

6 registered trials, 4 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

  • 4 Paroxysmal Nocturnal Hemoglobinuria (PNH) trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
  • Alexion Pharmaceuticals sponsors the most Paroxysmal Nocturnal Hemoglobinuria (PNH) trials on record, review their full trial history before evaluating a specific study. See Alexion Pharmaceuticals's trials
  • Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Paroxysmal Nocturnal Hemoglobinuria (PNH) condition card totals 6 registered trials; most-linked intervention Eculizumab; top US site state Maryland. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.