Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug aims to cut sickle cell pain crises in half

NCT ID NCT03814746

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 15, 2026 · Updated 7 times

Summary

This phase 3 study tests two doses of crizanlizumab against a placebo in 255 adolescents and adults with sickle cell disease who have frequent pain crises. The goal is to see if the drug can reduce the number of severe pain episodes that require a hospital or clinic visit. Participants may also continue taking hydroxyurea or other standard treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
crizanlizumab (a drug given by IV infusion)
What this could lead to
If it works, this could provide a new treatment to reduce painful crises and hospital visits for people with sickle cell disease.
What could go wrong
This is an advanced trial, but results may not show a clear benefit over placebo. Side effects or lack of efficacy could limit its use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

254 people

The number who actually took part.

Started

Jul 2019

Expected to finish

Nov 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Written informed consent must be obtained prior to any screening procedures 2. Male or female patients aged 12 years and older on the day of signing informed consent. Adolescent include patients aged 12 to 17 years old and adults ≥ 18 years 3. Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) \[performed locally\]. All SCD genotypes are eligible, genotyping is not required for study entry 4. Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history. Prior VOC leading to healthcare visit must resolve at least 7 days prior to Week 1 Day 1 and must include: 1. Pain crisis defined as an acute onset of pain for which there is no other medically determined explanation other than vaso- occlusion - 2. which requires a visit to a medical facility and/or healthcare professional, 3. and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study 5. If receiving HU/HC or L-glutamine (local HA approved medicinal product), must have been receiving the drug for at least 6 months and at a stable dose for at least 3 months prior to Screening visit and plan to continue taking it at the same dose and schedule until the subject has reached one year of study treatment. Patients who have not been receiving such drug must not have received it for at least 6 months prior to Screening visit to be included. Patients must have evidence of insufficient control of acute pain, such as at least one VOC leading to healthcare visit while on HU/HC or L-Glutamine treatment. If receiving erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening visit and plan to continue taking the treatment to maintain stable Hb levels at least until the subject has reached one year of study treatment 6. Patients must meet the following central laboratory values prior to Week 1 Day 1: * Absolute Neutrophil Count ≥1.0 x 109/L * Platelet count ≥75 x 109/L * Hemoglobin: for adults (Hb) ≥4.0 g/dL and for adolescents (Hb) ≥5.5 g/dL * Glomerular filtration rate ≥ 45 mL/min/1.73 m2 using CKD-EPI formula in adults, and Shwartz formula in adolescents * Direct (conjugated) bilirubin \< 2.0 x ULN * Alanine transaminase (ALT) \< 3.0 x ULN 7. ECOG performance status ≤2.0 for adults and Karnofsky ≥ 50% for adolescents Key Exclusion Criteria: 1. History of stem cell transplant. 2. Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted. 3. Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction. 4. Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to Screening visit or plans to participate in another investigational drug trial. 5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment. 6. Concurrent severe and/or uncontrolled medical conditions which, in the opinion of the Investigator, could cause unacceptable safety risks or compromise participation in the study. 7. History or current diagnosis of ECG abnormalities indicating significant risk of safety such as: * Concomitant clinically significant cardiac arrhythmias (e.g ventricular tachycardia), and clinically significant second or third degree AV block without a pacemaker * History of familial long QT syndrome or know family history of Torsades de Pointes 8. Not able to understand and to comply with study instructions and requirements. 9. Received prior treatment with crizanlizumab or other selectin targeting agent

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Sickle cell disease (SCD) are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

hematologic disorder sickle cell disease Sickle Cell Trait Vaso-Occlusive Crises

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease (SCD)

Contacts and locations

Locations

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • Childrens Healthcare of Atlanta

    Atlanta, Georgia, 30342, United States

  • Levine Cancer Insitute Carolinas Healthcare System

    Charlotte, North Carolina, 28204, United States

  • Novartis Investigative Site

    Brussels, Brussels Capital, 1070, Belgium

  • Novartis Investigative Site

    Brussels, 1000, Belgium

  • Novartis Investigative Site

    Edegem, 2650, Belgium

  • Novartis Investigative Site

    Salvador, Estado de Bahia, 41253-190, Brazil

  • Novartis Investigative Site

    Belém, Pará, 66033-000, Brazil

  • Novartis Investigative Site

    Recife, Pernambuco, 50070-170, Brazil

  • Novartis Investigative Site

    Rio de Janeiro, Rio de Janeiro, 20211-030, Brazil

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-003, Brazil

  • Novartis Investigative Site

    Ribeirão Preto, São Paulo, 14048-900, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 01232-010, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 05403-000, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 08270-070, Brazil

  • Novartis Investigative Site

    Toronto, Ontario, M5G 2C4, Canada

  • Novartis Investigative Site

    Montreal, Quebec, H2X 1R9, Canada

  • Novartis Investigative Site

    Barranquilla, Atlántico, 080020, Colombia

  • Novartis Investigative Site

    Valledupar, Cesar Department, 200001, Colombia

  • Novartis Investigative Site

    Montería, 230004, Colombia

  • Novartis Investigative Site

    Helsinki, 00290, Finland

  • Novartis Investigative Site

    Créteil, 94010, France

  • Novartis Investigative Site

    Marseille, 13885, France

  • Novartis Investigative Site

    Paris, 75015, France

  • Novartis Investigative Site

    Stuttgart, Baden-Wurttemberg, 70376, Germany

  • Novartis Investigative Site

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Novartis Investigative Site

    Berlin, 13353, Germany

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Accra, GA-270-9830, Ghana

  • Novartis Investigative Site

    Athens, 115 27, Greece

  • Novartis Investigative Site

    Pátrai, 265 04, Greece

  • Novartis Investigative Site

    Thessaloniki, 54636, Greece

  • Novartis Investigative Site

    Bhubaneswar, Odisha, 751003, India

  • Novartis Investigative Site

    Hyderabad, Telangana, 500082, India

  • Novartis Investigative Site

    Genova, GE, 16128, Italy

  • Novartis Investigative Site

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    Verona, VR, 37134, Italy

  • Novartis Investigative Site

    Naples, 80138, Italy

  • Novartis Investigative Site

    Irbid, 22110, Jordan

  • Novartis Investigative Site

    Beirut, 113-0236, Lebanon

  • Novartis Investigative Site

    Tripoli, 1434, Lebanon

  • Novartis Investigative Site

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Novartis Investigative Site

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    The Hague, South Holland, 2545 AA, Netherlands

  • Novartis Investigative Site

    Khoudh, 123, Oman

  • Novartis Investigative Site

    Panama City, 0801, Panama

  • Novartis Investigative Site

    Soweto, Gauteng, 2013, South Africa

  • Novartis Investigative Site

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    Madrid, 28034, Spain

  • Novartis Investigative Site

    Madrid, 28046, Spain

  • Novartis Investigative Site

    Adana, Saricam, 01330, Turkey (Türkiye)

  • Novartis Investigative Site

    Sheffield, South Yorkshire, S10 2JF, United Kingdom

  • Novartis Investigative Site

    Cambridge, CB2 0QQ, United Kingdom

  • Novartis Investigative Site

    London, SE1 9RT, United Kingdom

  • Novartis Investigative Site

    London, SE5 9RS, United Kingdom

  • Novartis Investigative Site

    Sheffield, S10 2TH, United Kingdom

  • U of TX Health Science Ct

    Houston, Texas, 77030, United States

  • Univ of Tenn Health Sciences Ctr

    Memphis, Tennessee, 38163, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.