A Study of SHP655 (rADAMTS13) in Sickle Cell Disease

Completed · Phase 1 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants. Study participants will receive TAK-755 or placebo on Day 1. Their SCD will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be asked to follow-up on 13 days following SHP655 or placebo administration for safety assessment. Maximum duration of participation is expected to be about 2 months.

Key facts

Study ID

NCT03997760

Run by

Shire

People needed

19

Starts

2019-10-21

Expected to finish

2022-10-26

Last updated by the study team

2024-04-23

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 18 to 65 years at the time of signing the informed consent.
• An understanding, ability, and willingness to fully comply with study procedures and requirements.
• Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
• Male or female with a documented history of HbSS or HbSβo thalassemia (based on clinical record of genetic, electrophoresis, or high-performance liquid chromatography testing).
• Participant currently taking hydroxyurea must be on a stable dosing for 3 months at screening.

You may not qualify if…

• The participant was diagnosed with acute VOC in the 21 days before dosing on Day 1.
• The participant has undergone blood transfusion within the last 30 days or blood transfusion on greater than or equal to (>=) 2 occasions in the last 90 days, at Screening Visit.
• The participant has a history of acquired or congenital thrombotic thrombocytopenic purpura.
• The participant has serum creatinine level greater than (>) 1.2 milligrams per deciliter (mg/dL).
• The participant has alanine transaminase >3* upper limit of normal (based on clinical laboratory normal range), direct bilirubin level >2 mg/dL, or indirect bilirubin level >5 mg/dL at the Screening Visit.
• The participant has a hemoglobin level <5 grams per deciliter (g/dL) at the Screening Visit.
• The participant has a platelet count of <100 000/cubic millimeter (mm\^3) at the Screening Visit.
• Signs or symptoms of infection requiring treatment with IV antibiotics during the Screening Period.
• The participant has fever with body temperature of >=38.5 degree Celsius (ºC) (101.3 degree Fahrenheit [ºF]) at the Screening Visit or before dosing on Day 1.
• The participant has Acute Chest Syndrome (ACS), diagnosed or strongly suspected, as evidenced by a new infiltrate on chest radiograph, and one or more of the following criteria:
• Fever with body temperature >39°C (102.2°F)
• Hypoxia (confirmed by arterial blood gases with partial pressure of arterial oxygen (PaO2) <70 millimeter of mercury [mmHg])
• Chest pain
• Suspicious findings on physical examination (tachypnea, intercostal retraction, wheezing, and/or rales)
• The participant has recently (within the past 28 days, from Screening Visit) undergone major surgery, requires hospitalization, documented serious bacterial infection requiring antibiotic treatment, or significant bleeding.
• The participant has had a recent (within the past 90 days, from Screening Visit) episode of stroke, transient ischemic attack, symptomatic pulmonary hypertension, or seizure.
• Any history of hemorrhagic stroke or bleeding diathesis.
• The participant has received any of the following protocol-restricted medicines: a) systemic steroid therapy within 48 hours before dosing, or there is the expectation that such therapy may be given during the study (inhaled or topical steroids are allowed); b) Anticoagulant or antiplatelet therapy within the past 3 weeks before dosing; c) crizanlizumab within the past 30 days before dosing; d) voxelotor within the past 14 days before dosing.
• For participants receiving chronic or long-acting opioids, a change in dose or pain requiring medical attention in the past 14 days before dosing.
• The participant has a medical or psychiatric condition that, in the opinion of the investigator, may pose a risk to the participant for participation or interfere with the conduct or results of the study.
• The participant has received or plans to receive any other investigational agent within the 4 weeks prior to the study screening visit or during the course of the study.
• There is the expectation that the participant will not be able to be followed for the duration of the study.
• The participant is pregnant or lactating or a female of childbearing potential or male unable or unwilling to comply with birth control methods or abstinence until the end of study visit.
• The participant with active use of illicit drugs (excluding marijuana) and/or alcohol dependence, as determined by the investigator.
• The participant has been administered SHP655 previously.

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• University of Colorado Sickle Cell Treatment and Research Center — Aurora, Colorado, United States
• Sickle Cell Center — Denver, Colorado, United States
• University of Illinois — Chicago, Illinois, United States
• Ochsner Health System — New Orleans, Louisiana, United States
• Johns Hopkins University School Of Medicine — Baltimore, Maryland, United States
• Levine Cancer Institute — Charlotte, North Carolina, United States
• Duke University Medical Center — Durham, North Carolina, United States
• East Carolina University — Greenville, North Carolina, United States
• Ohio State University Medical Center — Columbus, Ohio, United States
• Medical University of South Carolina (MUSC) — Charleston, South Carolina, United States
• University of Tennessee -- Memphis — Memphis, Tennessee, United States
• VCU Health - Research Parent — Richmond, Virginia, United States

Full record on ClinicalTrials.gov

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