Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises (SUSTAIN)

Reprixys Pharmaceutical

Status and phase: Completed; Phase 2

Conditions: Sickle Cell Disease

Treatments: Drug: Placebo; Drug: SelG1

Study type: Interventional

Funder types: Industry; Other U.S. Federal agency; NIH

Identifiers

NCT01895361

R01FD004805

R44HL093893 (U.S. NIH Grant/Contract)

SelG1-00005

Details and patient eligibility

About

The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream.

Funding Source - FDA Office of Orphan Products Development (OOPD)

Enrollment: 198 patients

Sex: All

Ages: 16 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Sickle Cell Disease (HbSS, HbSC, HbSβ⁰-thalassemia, or HbSβ⁺-thalassemia)
• If receiving hydroxyurea or erythropoietin, treatment must have been prescribed for at least 6 months, with the dose stable for at least 3 months
• Between 2 and 10 sickle cell-related pain crises in the past 12 months

Key Exclusion Criteria:

• On a chronic transfusion program or planning on exchange transfusion during the study
• Hemoglobin <4.0 g/dL
• Planned initiation, termination, or dose alteration of hydroxyurea during the study
• Receiving chronic anticoagulation therapy (e.g. warfarin, heparin) other than aspirin

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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198 participants in 3 patient groups, including a placebo group

High-dose SelG1 (Selg1 5.0 mg/kg)

Experimental group

Description:

IV Infusion, once every 4 weeks through Week 50

Treatment:

Drug: SelG1

Low-dose SelG1 (Selg1 2.5 mg/kg)

Experimental group

Description:

IV Infusion, once every 4 weeks through Week 50

Treatment:

Drug: SelG1

Placebo

Placebo Comparator group

Description:

IV Infusion, once every 4 weeks through Week 50

Treatment:

Drug: Placebo