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Phase 1/2 Dose Escalation Study in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia (8400-401)

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2015 by Idera Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Idera Pharmaceuticals, Inc. Identifier:
First received: March 18, 2014
Last updated: November 19, 2015
Last verified: November 2015
Recent reports have identified a specific oncogenic mutation L265P of the MYD88 gene in approximately 90% of the patients with Waldenström's macroglobulinemia. MYD88 is a key linker protein in the signaling pathway of Toll Like Receptors (TLRs) 7, 8, and 9, and IMO-8400 is an oligonucleotide specifically designed to inhibit TLRs 7,8, and 9. The scientific hypothesis for use of IMO-8400 to treat patients with Waldenström's macroglobulinemia depends on the inhibition of mutant MYD88 signaling in the TLR pathway, thereby interrupting the proliferation of cell populations responsible for the propagation of the disease.

Condition Intervention Phase
Waldenstrom's Macroglobulinemia
Drug: IMO-8400
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 1/2 Open-Label, Multiple-dose, Dose-escalation Study to Evaluate the Safety and Tolerability of IMO-8400 in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia

Resource links provided by NLM:

Further study details as provided by Idera Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Safety and tolerability of IMO-8400 in patients with Waldenstrom's Macroglobulinemia [ Time Frame: 29 weeks (24 week treatment + 5 week follow up) ] [ Designated as safety issue: Yes ]
    Assessment of adverse events, injection site reactions and concomitant medications in escalating dose levels of IMO-8400.

Secondary Outcome Measures:
  • Assess the treatment effect [ Time Frame: 29 weeks (24 week treatment + 5 week follow up) ] [ Designated as safety issue: No ]
    To assess the treatment effect (clinical activity) of escalating dose levels of IMO-8400 using disease-specific international guidelines for classifying clinical response.

  • Identify an appropriate dose of IMO-8400 [ Time Frame: 29 weeks (24 weeks treatment + 5 week follow-up) ] [ Designated as safety issue: No ]
    To identify an appropriate dose of IMO-8400 for further clinical evaluation.

  • Characterize PK [ Time Frame: 29 weeks (24 weeks treatment + 5 week follow-up) ] [ Designated as safety issue: No ]
    To characterize the pharmacokinetics of escalating dose levels of IMO-8400 administered once weekly by SC injection.

Estimated Enrollment: 24
Study Start Date: March 2014
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohorts A1, A2 and A3
IMO-8400 at one of three dose levels/cohorts
Drug: IMO-8400
IMO-8400 0.6 mg/kg q wk x 24 wk by subcutaneous injection (Cohort A1)
Drug: IMO-8400
IMO-8400 1.2 mg/kg q wk x 24 wk by subcutaneous injection (Cohort A2)
Drug: IMO-8400
IMO-8400 2.4 mg/kg q wk x 24 wk by subcutaneous injection (Cohort A3)

Detailed Description:
Eligible subjects will be enrolled and assigned to one of three dose cohorts. Treatment will be administered once weekly by subcutaneous injection. Subjects will receive treatment for 24 weeks and then be followed for an additional 5 weeks.

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Patients must have a diagnosis of Waldenstrom's Macroglobulinemia that has relapsed after a previous treatment or has shown partial or no response to previous treatment.

In addition to the above, key inclusion and exclusion criteria are listed below.

Inclusion Criteria:

  1. At least 18 years of age.
  2. Agree to use contraception

Exclusion Criteria:

  1. Is nursing or pregnant
  2. Has body weight < 50 kg.
  3. Has BMI > 34.9 kg/m2.
  4. Has a positive test for human immunodeficiency virus (HIV-1 or -2) hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg).
  5. Receiving chronic systemic corticosteroid therapy > 20 mg of prednisone daily.
  6. Being treated with other anti-cancer therapies (approved or investigational).
  7. Has an active infection requiring systemic antibiotics.
  8. Has had surgery requiring general anesthesia within 4 weeks of starting the study.
  9. Has autoimmune cytopenia (anemia, thrombocytopenia, leukopenia).
  10. Has heart failure of Class III or IV.
  11. Has sensory or motor neuropathy limiting daily activities.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02092909

Contact: Lindsey Simov 617-679-5500

United States, Arkansas
Cancer Centers of Excellence Recruiting
Fayetteville, Arkansas, United States, 72758
United States, California
UCLA Recruiting
Los Angeles, California, United States, 90404
Stanford University Recruiting
Palo Alto, California, United States, 94304
United States, Florida
University of Miami Sylvester Comprehensive Center Recruiting
Miami, Florida, United States, 33136
UF Health Cancer Center - Orlando Health Recruiting
Orlando, Florida, United States, 32806
United States, Georgia
Emory Winship Cancer Institute Recruiting
Atlanta, Georgia, United States, 30322
United States, Indiana
Horizon BioAdvance Recruiting
Lafayette, Indiana, United States, 47905
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55902
United States, New Jersey
Hackensack University Recruiting
Hackensack, New Jersey, United States, 07601
United States, New York
Memorial Sloan-Kettering Cancer Center Recruiting
New York, New York, United States, 10065
United States, Ohio
Gabrail Cancer Center Recruiting
Canton, Ohio, United States, 02139
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
United States, Washington
Seattle Cancer Care Alliance Recruiting
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Idera Pharmaceuticals, Inc.
Study Director: James O'Leary, MD Idera Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Idera Pharmaceuticals, Inc. Identifier: NCT02092909     History of Changes
Other Study ID Numbers: 8400-401
Study First Received: March 18, 2014
Last Updated: November 19, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Waldenstrom Macroglobulinemia
Blood Protein Disorders
Cardiovascular Diseases
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Neoplasms, Plasma Cell
Vascular Diseases processed this record on November 27, 2015