A Study of Dose Confirmation and Safety of Experimental Crizanlizumab for Pediatric Sickle Cell Disease

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Age: Up to 17 years old
Gender:
Any
Healthy Volunteers: No
Keywords: sickle cell disease, sickle cell anemia, SCD, red blood cells, blood disorder, pediatric blood disorder, pediatric sickle cell disease
Type: Drug study, Phase 2
Target:
3 Participants
Investigator:
Description
The purpose of this study is to find out if a drug, previously approved for adults, is safe and effective for children with Sickle Cell Disease (SCD).

SCD is a rare blood disorder that causes red blood cells (RBC) to have a “sickle” shape. They are destroyed more quickly than normal cells, so patients with SCD have fewer red blood cells. They also have painful episodes, called Vaso-occlusive Crises (VOC). These episodes are caused by cells in the blood and blood vessels sticking together and slowing down the flow of blood.

The name of the experimental treatment is Crizanlizumab (SEG101). Part of determining whether this study treatment is safe and effective for children is to determine how much of the drug should be given (dosage). The participants in the study will be given various amounts based on age and other medical factors as determined by the study doctors.

This drug is currently approved by the FDA for use in adults (16 years and older) and has been proven effective is reducing the complications of SCD. This drug is not currently approved for children. Because it is not approved for children, it is called an experimental drug for the purposes of the study.

This is the first time that this study treatment is being given to children in a study. In a previous study for adults, the study treatment decreased the number of VOC episodes. The study treatment also resulted in longer periods between episodes of VOC in adults.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  • Male or female patients aged 2 to <18 years (Group 3 will be expanded to allow enrolment of patients aged 6 to <24 months (and at least 6 kg) in Part B once the appropriate dose is confirmed in 2 to <6 year old patients)
  • Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbSβ0-thalassemia, HbSβ+-thalassemia, and others) by hemoglobin electrophoresis or high-performance liquid chromatography (HPLC) performed locally.
  • Experienced at least 1 VOC within the preceding 12 months, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and should include all the following:
  • the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1)
  • either a visit to a medical facility or healthcare professional,
  • receipt of oral/parenteral opioid or other non-opioid parenteral analgesia.
  • If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening and plan to continue taking at the same dose and schedule during the trial. Dose alterations of HU/HC during Part A are not allowed, and if this occurs, the patient will enter directly to the Part B.
  • Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education
  • Transcranial Doppler (TCD) considered low risk within the past 6 months (for 2 to 16 years).

Exclusion criteria:

  • History of stem cell transplant.
  • Received any blood products within 30 days of Day 1 dosing.
  • Participating in a chronic transfusion program (preplanned series of transfusions for prophylactic purposes).
  • Patients with bleeding disorders
  • Planning on undergoing an exchange transfusion during the duration of the study. Patients requiring episodic transfusion in response to worsened anemia or VOC are permitted.
  • Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation.
  • Received a monoclonal antibody or immunoglobulin-based therapy within 6 months of Screening, or has documented immunogenicity to a prior monoclonal antibody.
  • Received active treatment on another investigational trial within 30 days (or 5 half lives of that agent, whichever is greater) prior to Screening or plans to participate in another investigational drug trial.
  • Pregnant females or females who have given birth within the past 90 days or who are breastfeeding.
  • Any documented history of a stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months
  • Any conditional TCD within the past 12 months
  • Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet therapy (other than aspirin) within the 10 days prior to Week 1 Day 1 dosing
  • Hospitalized at Screening
  • Planning to undergo a major surgical procedure during the duration of the study
  • Planning to initiate or terminate HU/HC while on study, other than for safety reasons
  • Patient with active HIV infection (detectable viral load)
  • Patients with known active Hepatitis B infection.
  • Patients with known Hepatitis C history.
  • Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator.
  • Malignant disease. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 2 years prior to study treatment; any completely resected carcinoma in situ.
  • Has a serious mental or physical illness, which, in the opinion of the Investigator would compromise participation in the study.
  • Resting QTcF ≥450 msec at pretreatment (baseline) for patients under 12 years of age and ≥450 msec for males and ≥460 msec for female patients 12 years and older.
  • Cardiac or cardiac repolarization abnormality
  • Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome
  • Sexually active females who are unwilling to comply with reliable method of birth control until 15 weeks following last dose of study drug.
  • Current drug or alcohol abuse:
  • Has a positive qualitative urine drug test at Screening for cocaine, phencyclidine (PCP), or amphetamines (opioids are permitted).
  • Consumes >12 (for males) or >8 (for females) standard alcoholic beverages per week.
  • Not able to understand and to comply with study instructions and requirements.
  • Subjects, who are an employee of the sponsor or investigator or otherwise dependent on them.
  • Subjects, who are committed to an institution by virtue of an order issued either by the judicial or the administrative authorities.
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study team will discuss the risks associated with taking part in this research study.
Compensation
If you choose to take part in this study, you will be given a debit card. $125 will be automatically loaded onto the card at each regularly scheduled study visit.
Resources
Schedule
Study duration and period
You will be in the Treatment Phase of this study for up to two years.
Recruitment period
From June 24, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Beatrice Ark-Majiyagbe
Research Topic
Conditions:
  • Sickle Cell Disease

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