Paroxysmal Nocturnal Hemoglobinuria

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14 Paroxysmal Nocturnal Hemoglobinuria Trials Near You

Power is an online platform that helps thousands of Paroxysmal Nocturnal Hemoglobinuria patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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No Placebo
Highly Paid
Stay on Current Meds
Pivotal Trials (Near Approval)
Breakthrough Medication

Crovalimab vs Eculizumab for Paroxysmal Nocturnal Hemoglobinuria

Columbus, Ohio
This trial is testing the safety of crovalimab and eculizumab in patients with PNH who are already on medications. Both drugs work by stopping the immune system from destroying red blood cells. Eculizumab, a medication that was first approved for PNH in 2007, has revolutionized the treatment of this disease.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+

190 Participants Needed

Treosulfan-Based Conditioning for Bone Marrow Failure

Columbus, Ohio
This trial tests if a combination of three drugs can reduce complications for patients with bone marrow failure diseases. The drugs work by killing harmful cells, stopping their growth, and reducing immune reactions.
No Placebo Group

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 49

40 Participants Needed

Danicopan Add-On Therapy for Paroxysmal Nocturnal Hemoglobinuria

Cleveland, Ohio
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
No Placebo Group
Prior Safety Data
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3

80 Participants Needed

Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Cleveland, Ohio
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
No Placebo Group
Prior Safety Data
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3

208 Participants Needed

Long-Term Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Cleveland, Ohio
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3

160 Participants Needed

Pozelimab + Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria

Toronto
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

202 Participants Needed

Pozelimab + Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria

Toronto, Ontario
This trial is testing a new combination of two drugs, pozelimab and cemdisiran, for patients with a rare blood disorder called PNH. The goal is to see if this new combination is safe and works better than current treatments. Researchers will also check for side effects and how the body reacts to the drugs. Eculizumab is the first approved treatment for PNH and has significantly changed the treatment landscape by inhibiting terminal complement activation.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 3

190 Participants Needed

Stem Cell Transplant for Severe Aplastic Anemia

Bethesda, Maryland
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy. Eligibility: Recipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75 Design: Recipients will have: * Blood, urine, heart, and lung tests * Scans * Bone marrow sample Recipients will need a caregiver for several months. They may make fertility plans and a power of attorney. Donors will have blood and tissue tests, then injections to boost stem cells for 5-7 days. Donors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg. In the hospital for about 1 month, recipients will have: * Central line inserted in the neck or chest * Medicines for side effects * Chemotherapy over 8 days and radiation 1 time * Stem cell transplant over 4 hours Up to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests. At day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 75

56 Participants Needed

Stem Cell Transplant for Severe Aplastic Anemia

Bethesda, Maryland
Background: * Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications. * By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications. Objectives: - To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient. Eligibility: * Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants. * Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program. Design: * All participants will be screened with a physical examination and medical history. * DONORS: * Donors will undergo an initial apheresis procedure to donate white blood cells. * After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood. * After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood. * RECIPIENTS: * Recipients will provide an initial donation of white blood cells to be used for research purposes only. * From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant. * After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion. * After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant. * Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:4 - 80

120 Participants Needed

Crovalimab vs Eculizumab for Paroxysmal Nocturnal Hemoglobinuria

Charlotte, North Carolina
This trial is testing if crovalimab is as effective as eculizumab in treating people with PNH who haven't received similar treatments before. Both medications help prevent the immune system from attacking red blood cells. Eculizumab is a treatment for paroxysmal nocturnal hemoglobinuria (PNH) that decreases intravascular hemolysis and thrombosis and improves survival.
No Placebo Group
Pivotal Trial (Near Approval)

Trial Details

Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+

204 Participants Needed

Stem Cell Transplant for Bone Marrow Failure Syndrome

Philadelphia, Pennsylvania
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Unphased
Age:< 25

50 Participants Needed

Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria

Atlanta, Georgia
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:12 - 17

12 Participants Needed

Stem Cell Transplant for Blood Disorders

Minneapolis, Minnesota
This trial is studying how well stem cell transplants work for patients with blood diseases that are not cancer. The treatment involves giving patients healthy stem cells from a donor to replace their damaged or diseased cells. This method is the oldest and most widely used technique of stem cell transplant, primarily used to treat blood disorders.
No Placebo Group

Trial Details

Trial Status:Recruiting
Age:< 55

25 Participants Needed

Hematopoietic Cell Transplant for Aplastic Anemia

Minneapolis, Minnesota
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.
No Placebo Group

Trial Details

Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 75

60 Participants Needed

Why Other Patients Applied

"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

FF
ADHD PatientAge: 31

"As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

IZ
Healthy Volunteer PatientAge: 38

"I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

ID
Pancreatic Cancer PatientAge: 40

"I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

ZS
Depression PatientAge: 51

"I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

WR
Obesity PatientAge: 58

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We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

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Bask GillCEO at Power
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How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

Frequently Asked Questions

How much do Paroxysmal Nocturnal Hemoglobinuria clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Paroxysmal Nocturnal Hemoglobinuria clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Paroxysmal Nocturnal Hemoglobinuria trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Paroxysmal Nocturnal Hemoglobinuria is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Paroxysmal Nocturnal Hemoglobinuria medical study?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Paroxysmal Nocturnal Hemoglobinuria clinical trials?

Most recently, we added Hematopoietic Cell Transplant for Aplastic Anemia, Pozelimab + Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria and Danicopan Add-On Therapy for Paroxysmal Nocturnal Hemoglobinuria to the Power online platform.

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