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Lifeline for sickle cell patients: drug access extended in new study

NCT ID NCT04657822

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study offers continued access to crizanlizumab, a drug that helps reduce painful crises in sickle cell disease, for 130 patients who benefited from it in earlier Novartis trials. Participants must have completed a prior study and lack other access to the drug. The main goal is to keep providing treatment and monitor for side effects.

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 to reduce pain crises)
What this could lead to
If successful, this study ensures patients with sickle cell disease can keep receiving a treatment that helps control their condition while longer-term access is arranged.
What could go wrong
This is not a test of whether the drug works—it's a continuation study for people already benefiting. Side effects are still possible, and the drug may not be available to everyone long-term.

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 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 130 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2021

Expected to finish

Jun 2031

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

6 months 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.

Inclusion Criteria: 1. Written informed consent/assent, according to local guidelines, signed by the adult patients. In the population under 18 years, it will be signed by the patient and/or by the parents or legal guardian prior to enrolling in the rollover study and receiving study medication 2. SCD patient currently enrolled in a Novartis-sponsored study receiving crizanlizumab and has fulfilled all the requirements in the parent study. Patient is currently benefiting from the treatment with crizanlizumab as determined by the investigator and has completed the treatment schedule as planned in the parent study 3. Patient has demonstrated compliance to the planned visit schedule in the parent study, and in the opinion of the investigator has shown willingness and ability to comply with future visit schedules Exclusion Criteria: 1. Patient had permanently discontinued from crizanlizumab study treatment in the parent study before the parent study completion 2. Ongoing/unresolved treatment-related Grade 3 or higher AEs, and/or any ongoing AE requiring dose interruption. Patients meeting all other eligibility criteria may be enrolled once toxicities have resolved unless those toxicities were grade 4 3. Concurrent participation in any other investigational clinical trial other than the parent study or plan to participate in any other investigational clinical trial 4. Pregnant or nursing women 5. Women of childbearing potential who are unwilling to be on highly effective contraceptives during dosing and until 15 weeks after stopping treatment with crizanlizumab 6. SCD patients who do not meet parent study protocol criteria to continue with crizanlizumab

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Conditions

The condition(s) this trial relates to.

sickle cell disease Vaso-Occlusive Crises

As listed by the trial registrant

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

Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    7 sites in 4 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Augusta University Georgia

    RECRUITING

    Augusta, Georgia, 30912, United States

  • Childrens Hospital of Philadelphia

    ACTIVE_NOT_RECRUITING

    Philadelphia, Pennsylvania, 19104-4399, United States

  • Childrens National Hospital

    ACTIVE_NOT_RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Cook Childrens Medical Center

    COMPLETED

    Fort Worth, Texas, 76104, United States

  • East Carolina University

    COMPLETED

    Greenville, North Carolina, 27834, United States

  • East Carolina University

    COMPLETED

    Greenville, North Carolina, 27858, United States

  • Novartis Investigative Site

    COMPLETED

    Brussels, 1000, Belgium

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Laken, 1020, Belgium

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Liège, 4000, Belgium

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Salvador, Estado de Bahia, 41253-190, Brazil

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    São Paulo, São Paulo, 01232-010, Brazil

  • Novartis Investigative Site

    RECRUITING

    Barranquilla, Atlántico, 080020, Colombia

  • Novartis Investigative Site

    RECRUITING

    Valledupar, Cesar Department, 200001, Colombia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Cali, Valle del Cauca Department, 760032, Colombia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Montería, 230004, Colombia

  • Novartis Investigative Site

    COMPLETED

    Créteil, 94010, France

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Paris, 75015, France

  • Novartis Investigative Site

    COMPLETED

    Paris, 75015, France

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Heidelberg, 69120, Germany

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Padova, PD, 35128, Italy

  • Novartis Investigative Site

    COMPLETED

    Orbassano, TO, 10043, Italy

  • Novartis Investigative Site

    RECRUITING

    Beirut, 113-0236, Lebanon

  • Novartis Investigative Site

    RECRUITING

    Tripoli, 1434, Lebanon

  • Novartis Investigative Site

    RECRUITING

    Khoudh, 123, Oman

  • Novartis Investigative Site

    COMPLETED

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Madrid, 28009, Spain

  • Novartis Investigative Site

    WITHDRAWN

    Hatay, Antakya, 31101, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Adana, Saricam, 01330, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Adana, Yuregir, 01250, Turkey (Türkiye)

  • University Of Alabama

    RECRUITING

    Birmingham, Alabama, 35233, United States

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