Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo

NCT01419977 | Completed Phase 2 Results DMC

• 1 other identifier: Pro00023305
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

Sickle cell disease (SCD) is one of the most common inherited diseases worldwide and exhibits highest frequency in people of African descent. Patients with SCD currently have few treatment options, with hydroxyurea being the only medication approved to reduce the frequency of vaso-occlusive crisis (VOC) and prevent other SCD complications such as acute chest syndrome. Once patients develop VOC, hospitalizations aim to alleviate pain; no specific therapy is currently available to otherwise affect the course of the VOC. However, there has been increasing interest in the role of coagulation in the pathogenesis of SCD. The investigators hypothesize that low dose anticoagulant therapy, such as prophylactic dose low-molecular-weight heparin (LMWH), could be a novel way to ameliorate the vaso-occlusive process and thereby hasten the resolution of pain.

Conditions

Sickle Cell Disease; Vaso-occlusive Crisis

Keywords

sickle cell disease; vaso-occlusive crisis; anticoagulation

Outcome Measures

Primary Outcomes (4)

• Change in D-dimer

  Patients will have D-dimer,for samples drawn on Day 1 and Day 3

  Day 1 and Day 3

• Change in Clinical Pain Scores

  The primary pain assessment tool will be a 10-cm horizontal visual analog scale (VAS), with "0" corresponding to no pain at one end and "10" indicating the worst pain at the other.

  Baseline to day 1

• Change in Thrombin Generation Assay - Endogenous Thrombin Potential

  Patients will have thrombin generation assay samples drawn on Day 1 and 3

  Day 1 and Day 3

• Change in Clinical Pain Scores

  The primary pain assessment tool will be a 10-cm horizontal visual analog scale (VAS), with "0" corresponding to no pain at one end and "10" indicating the worst pain at the other.

  Baseline to day 3

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Normal saline solution

Drug: Placebo

Dalteparin

EXPERIMENTAL

5000 unites subcutaneously, Other Name: Fragmin

Drug: Dalteparin

Interventions

Placebo DRUG

Normal saline solution, administered by nursing staff once daily

Placebo

Dalteparin DRUG

Low molecular weight heparin (LMWH), 5000 unites subcutaneously, administered by nursing staff once daily, Other Name: Fragmin

Dalteparin

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented HgbSS or HgbS-beta0 thalassemia by previous hemoglobin electrophoresis,
• age greater than 18 years old, and
• admit diagnosis of vaso-occlusive crisis.
• Labs must be drawn within 36 hours of admission and randomization to treatment arm must occur during this time.

You may not qualify if:

• End stage renal disease (creatinine \>3.0 mg/dL),
• use of antiplatelet or anticoagulation medication for an alternative indication,
• use of steroids or immunosuppressive medications,
• platelet count less than 100 X 109/L,
• history or development of heparin induced thrombocytopenia, packed red blood cell transfusion in the past one month, or
• recent hospitalization with discharge within the past 1 week.
• Patients with re-admissions will not be enrolled again and will have no further samples drawn.

Sponsors & Collaborators

• Duke University: lead
• Eisai Limited: collaborator

Study Sites (1)

Duke University

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises

Interventions

Dalteparin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Heparin, Low-Molecular-Weight; Heparin; Glycosaminoglycans; Polysaccharides; Carbohydrates

Limitations and Caveats

Limitations to our pilot study include the small study sample size, which leads to difficulty in determining significant differences in the outcome measurements.

Results Point of Contact

• Title: Nirmish Shah, MD
• Organization: Duke University Medical Center

nirmish.shah@duke.edu

919-684-5350

Study Officials

• Nirmish Shah, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2011
• First Posted: August 19, 2011
• Study Start: May 1, 2011
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2014
• Last Updated: February 16, 2015
• Results First Posted: February 16, 2015

Record last verified: 2015-01

Locations

US (1)