ClinicalTrials.gg
TerminatedNCT020929098400-401Updated Sep 10, 2019Results posted

Phase 1/2 Dose Escalation Study in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia

A Phase 1/2 interventional study of IMO-8400 in Waldenstrom's Macroglobulinemia, sponsored by Idera Pharmaceuticals, Inc.. Terminated at 10 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-09-10.

Sponsored by Idera Pharmaceuticals, Inc. · Phase 1/2, Interventional, and Treatment

Why this study was terminated
Lack of efficacy

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Phase
Phase 1/2
Study type
Interventional
Enrollment
31
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 419 studies in Waldenstrom Macroglobulinemia with a site in United States — 34 open to participants now, 57 under way, 1 of them phase 3. Waldenstrom Macroglobulinemia · United States.

Enrollment
208th of 365
31 · median 37
Started
2014
with 15 others
Phase 1/2
48 of 419
2 open now
Registered
322nd of 419
by date

Among the 419 studies in Waldenstrom Macroglobulinemia with a site in United States, this one is the 208th largest by enrollment of the 365 interventional ones that give a count, is one of 16 that started in 2014, is one of 48 in phase 1/2, and was the 322nd registered.

Waldenstrom Macroglobulinemia

524 studies on the registry are indexed under Waldenstrom Macroglobulinemia; 62 are open to participants now.

Browse Waldenstrom Macroglobulinemia studies →

Open to participants now

34 of the 419 studies in Waldenstrom Macroglobulinemia with a site in United States are open now. Recruiting first, the newest first:

29 more — sign in →

All 34 open studies →

A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →

Who these studies take

Of the 48 Waldenstrom Macroglobulinemia studies recruiting now, 44 (92%) set no upper age limit and 1 (2%) stops at 75 or younger; 2 take children under 18; none take healthy volunteers.

This study: 18 Years and older · all sexes · no healthy volunteers.

The full table on the hub →

This tab as a report to print or save as PDF → With an account.

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Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 419 studies in Waldenstrom Macroglobulinemia with a site in United States, or all of them as a file.
02

Study summary

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.

Read the detailed description

Eligible subjects will be enrolled and assigned to escalating dose cohorts. Treatment will be administered by subcutaneous injection.

03

Conditions studied

  • Waldenstrom's Macroglobulinemia
04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Inclusion Criteria:

Patients must have a diagnosis of Waldenstrom's Macroglobulinemia.

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
  3. ≥ 7.5 g/dL, - ≥ 1.0 x 109/L (1000/mm3), - ≥ 50,000/μL

Exclusion Criteria:

  1. Is nursing or pregnant
  2. Has > 34.9 kg/m2.
  3. Has a positive test for (HIV-1 or -2) virus (HCV) or surface antigen (HBsAg).
  4. Receiving chronic systemic therapy > 20 mg of prednisone daily.
  5. Being treated with other anti-cancer therapies (approved or ).
  6. Has, at the initiation of study drug, received cytotoxic chemotherapy or a Bruton's tyrosine kinase (BTK)-inhibitor (e.g. ibrutinib) within the past 3 weeks or rituximab within the past 2 months
  7. Has an active infection requiring systemic antibiotics.
  8. Has had surgery requiring general anesthesia within 4 weeks of starting the study.
  9. Has cytopenia (anemia, thrombocytopenia, leukopenia).
  10. Has heart failure of Class III or IV.
  11. Has sensory or motor neuropathy limiting daily activities.
Terms in these criteria, in plain language(12)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
31 participants (actual)

Study arms

  • Experimental
    IMO-8400 at escalating dose levels

    IMO-8400 at escalating dose levels by subcutaneous injection

    Drug: IMO-8400

Interventions

  • IMO-8400 at escalating dose levels by subcutaneous injection

06

What researchers measure

Primary outcomes

  1. Safety and Tolerability of IMO-8400 in Patients With Waldenstrom's Macroglobulinemia

    Safety and tolerability of IMO-8400 in patients with Waldenstrom's Macroglobulinemia: Assessment of adverse events

    Time frame: Up to 24 weeks

Secondary outcomes

  1. Best Overall Response

    Best Overall Response using criteria from the VIth International Workshop in Waldenstrom's Macroglobulinemia

    Time frame: Up to 24 weeks

  2. Identify the Number of Patients Experiencing DLTs at Each Dose Level

    To identify an appropriate dose of IMO-8400 for further clinical evaluation via evaluation of DLT at each dose level

    Time frame: 28 days

  3. Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - Cmax.

    Pharmacokinetics of escalating dose levels of IMO 8400 administered by SC injection - Cmax.

    Time frame: Cycle 1 Week 1 Day 1: Samples were obtained pre-dose (within 1 hr prior to injection) and post-dose at 1 hr (+/-5 min), 2 hrs (+/-10 min) and 4 hrs (+/-15 min)

  4. Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - AUC0-t (hr*ng/mL)

    Pharmacokinetics of escalating dose levels of IMO 8400 administered by SC injection - AUC0-t (hr\*ng/mL) .

    Time frame: Cycle 1 Week 1 Day 1: Samples were obtained pre-dose (within 1 hr prior to injection) and post-dose at 1 hr (+/-5 min), 2 hrs (+/-10 min) and 4 hrs (+/-15 min)

07

Results

Posted Aug 29, 2019
Limitations and caveats
Early termination leading to small numbers of subjects analyzed.

Participant flow

Patients were enrolled from 02 April 2014 through 31 March 2017 at 14 study sites in the United States.

Participant flow — Overall Study
MilestoneIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Started65146
Completed5250
Not completed1396
Withdrew: Adverse event0042
Withdrew: Lack of efficacy0210
Withdrew: Withdrawal by subject0021
Withdrew: Study terminated by sponsor0002
Withdrew: Miscellaneous1121

Outcome measures

PrimarySafety and Tolerability of IMO-8400 in Patients With Waldenstrom's Macroglobulinemia

Safety and tolerability of IMO-8400 in patients with Waldenstrom's Macroglobulinemia: Assessment of adverse events

Time frame:
Up to 24 weeks
Reported as:
Count of participants · Participants
Safety and Tolerability of IMO-8400 in Patients With Waldenstrom's Macroglobulinemia
ParticipantsIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
At Least 1 TEAE65146
At Least 1 Related TEAE52126
At Least 1 Grade >=3 TEAE1273
At Least 1 SAE0012
At Least 1 DLT0010
At Least 1 TEAE Leading to Death0000
At Least 1 TEAE Leading to Premature Treatment DC0042
SecondaryBest Overall Response

Best Overall Response using criteria from the VIth International Workshop in Waldenstrom's Macroglobulinemia

Time frame:
Up to 24 weeks
Reported as:
Count of participants · Participants
Best Overall Response
ParticipantsIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Complete Response0000
Very Good Partial Response0000
Partial Response1020
Minor Response2041
Standard Deviation2343
Progressive Disease0111
Not Evaluable0010
SecondaryIdentify the Number of Patients Experiencing DLTs at Each Dose Level

To identify an appropriate dose of IMO-8400 for further clinical evaluation via evaluation of DLT at each dose level

Time frame:
28 days
Reported as:
Count of participants · Participants
Identify the Number of Patients Experiencing DLTs at Each Dose Level
ParticipantsIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Identify the Number of Patients Experiencing DLTs at Each Dose Level0010
SecondaryPharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - Cmax.

Pharmacokinetics of escalating dose levels of IMO 8400 administered by SC injection - Cmax.

Time frame:
Cycle 1 Week 1 Day 1: Samples were obtained pre-dose (within 1 hr prior to injection) and post-dose at 1 hr (+/-5 min), 2 hrs (+/-10 min) and 4 hrs (+/-15 min)
Reported as:
Geometric mean · Cmax (ng/mL)
Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - Cmax.
Cmax (ng/mL)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - Cmax.807.6 ± 70.32137.9 ± 68.72421.2 ± 50.84433.1 ± 29.9
SecondaryPharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - AUC0-t (hr*ng/mL)

Pharmacokinetics of escalating dose levels of IMO 8400 administered by SC injection - AUC0-t (hr\*ng/mL) .

Time frame:
Cycle 1 Week 1 Day 1: Samples were obtained pre-dose (within 1 hr prior to injection) and post-dose at 1 hr (+/-5 min), 2 hrs (+/-10 min) and 4 hrs (+/-15 min)
Reported as:
Geometric mean · AUC0-t (hr*ng/mL)
Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - AUC0-t (hr*ng/mL)
AUC0-t (hr*ng/mL)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Pharmacokinetics of Escalating Dose Levels of IMO 8400 Administered by SC Injection - AUC0-t (hr*ng/mL)2565.8 ± 64.66493.0 ± 61.77229.3 ± 54.513691.7 ± 32.3

Adverse events

Collected over From obtaining informed consent through EOS visit. (up to 24 weeks). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
IMO-8400 0.6 mg/kg 1x/wk0/6 (0%)0/6 (0%)6/6 (100%)
IMO-8400 1.2 mg/kg 1x/wk0/5 (0%)0/5 (0%)5/5 (100%)
IMO-8400 1.2 mg/kg 2x/wk0/14 (0%)1/14 (7.1%)13/14 (92.9%)
IMO-8400 2.4 mg/kg 1x/wk0/6 (0%)2/6 (33.3%)6/6 (100%)
Most frequent serious events
Most frequent serious events
EventIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Lower limb fractureInjury, poisoning and procedural complications0/60/50/141/6
Pulmonary embolismRespiratory, thoracic and mediastinal disorders0/60/50/141/6
SepsisInfections and infestations0/60/50/141/6
ArthralgiaMusculoskeletal and connective tissue disorders0/60/50/141/6
ArthritisMusculoskeletal and connective tissue disorders0/60/51/140/6
Most frequent other events
Showing 10 of 109
Most frequent other events
EventIMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wk
Injection site reactionGeneral disorders1/61/55/144/6
DiarrhoeaGastrointestinal disorders0/60/53/143/6
NauseaGastrointestinal disorders1/61/53/143/6
FatigueGeneral disorders1/61/55/143/6
Injection site erythemaGeneral disorders3/60/50/140/6
PyrexiaGeneral disorders0/61/51/143/6
ArthralgiaMusculoskeletal and connective tissue disorders0/61/51/143/6
Back painMusculoskeletal and connective tissue disorders1/60/50/143/6
Pain in extremityMusculoskeletal and connective tissue disorders2/60/51/143/6
HeadacheNervous system disorders0/62/53/142/6

Baseline characteristics

Safety Population

Age, Continuous
Age, Continuous(years)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Mean74.8 ± 12.9270.4 ± 9.7166.5 ± 11.1762.7 ± 11.3668.0 ± 11.53
Sex: Female, Male
Sex: Female, Male(Participants)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Female23319
Male4211522
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Hispanic or Latino00000
Not Hispanic or Latino6514631
Unknown or Not Reported00000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
American Indian or Alaska Native00000
Asian00112
Native Hawaiian or Other Pacific Islander00000
Black or African American00101
White6512528
More than one race00000
Unknown or Not Reported00000
Region of Enrollment
Region of Enrollment(participants)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
United States6514631
Time Since WM Diagnosis (yrs)
Time Since WM Diagnosis (yrs)(years)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Mean7.03 ± 4.6210.16 ± 6.907.66 ± 3.555.78 ± 5.227.58 ± 4.66
Baseline IPSSWM Score
Baseline IPSSWM Score(Participants)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Low215210
Intermediate338418
High11103
Baseline Total IgM
Baseline Total IgM(mg/dL)IMO-8400 0.6 mg/kg 1x/wkIMO-8400 1.2 mg/kg 1x/wkIMO-8400 1.2 mg/kg 2x/wkIMO-8400 2.4 mg/kg 1x/wkTotal
Mean1090 ± NA2410 ± 9732857 ± 15672910 ± 17272715 ± 1473

1 further baseline measures are reported on the registry.

08

Study locations

10 sites
  • Cancer Centers of Excellence
    Fayetteville, Arkansas 72758, United States
  • UCLA
    Los Angeles, California 90404, United States
  • Mayo Clinic Jacksonville
    Jacksonville, Florida 32224, United States
  • Emory Winship Cancer Institute
    Atlanta, Georgia 30322, United States
  • Horizon BioAdvance
    Lafayette, Indiana 47905, United States
  • Mayo Clinic
    Rochester, Minnesota 55902, United States
  • Hackensack University
    Hackensack, New Jersey 07601, United States
  • Memorial Sloan-Kettering Cancer Center
    New York, New York 10065, United States
  • MD Anderson Cancer Center
    Houston, Texas 77030, United States
  • Seattle Cancer Care Alliance
    Seattle, Washington 98109, United States
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Mar 2014, a primary completion of Apr 2017 and a completion of Nov 2017. It does not say whether these dates are actual or estimated.

Results were first posted in Aug 2019, 2 years 4 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 18, 2014
First posted
Mar 20, 2014
Start
Mar 2014
Primary completionActual
Apr 2017
CompletionActual
Nov 2017
Results first posted
Aug 29, 2019
Last update
Sep 10, 2019
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Sep 10, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02092909
Lead sponsor
Idera Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Mar 20, 2014
Start date
Mar 2014
Primary completion
Apr 2017
Completion
Nov 2017
Results posted
Aug 29, 2019
Last update
Sep 10, 2019

Study contacts

Mark Cornfeld, MD, MPH
study director · Idera Pharmaceuticals, Inc.
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Aug 2019. You cannot join it, but the record below documents what was studied.

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