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No longer recruiting (closed or complete)Last updated: 30 January 2024

This phase II trial is evaluating the drug RA101495 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People18+

Clinical summary

Summary

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.

Conditions

This trial is treating people with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility

Inclusion

  • Diagnosis of PNH by flow cytometry
  • For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level ≥2 times the upper limit of normal (xULN) during Screening
  • For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening

Exclusion

  • History of meningococcal disease
  • Current systemic infection or suspicion of active bacterial infection

Inclusion

  • You have had a certain type of treatment or surgical procedure.

Exclusion

  • You have certain types of non-cancer medical conditions.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

Ra Pharmaceuticals

Scientific Title

Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

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