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Myelodysplastic SyndromePhase 1/2NCT02363491

A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome

A Phase 1/2 study testing OPN-305 in people with Myelodysplastic Syndrome, run by Opsona Therapeutics Ltd. at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The dose-confirming part of this study, comprising at least 10 patients is designed as a single center, prospective, single arm, open label in patients who have failed or are unresponsive to Azacitidine (AZA) or Decitabine (they may also have additionally failed an Erythropoiesis Stimulating Agent (ESA) followed by a… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome
What is being tested?
OPN-305
Could I receive a placebo?
No, everyone receives an active treatment
How many people
96 participants
Where
4 sitesUnited States
Study dates
Jan 2015to Dec 2018
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Written informed consent
  • Age ≥ 18 years
  • Diagnosis of MDS (de novo or secondary) by bone marrow aspirate based on the World Health Organization (WHO) classification - Low and Intermediate-1 risk categories MDS using the IPSS (International Prognostic Scoring System)
  • AZA/decitabine (this applies to standard of care and investigational drugs) failure (Dose confirming and Dose expansion parts):
  • defined as discontinuation due to any of the following:
  • Lack of response after at least 4 cycles
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  • Loss of response (patient must have received therapy for at least 4 cycles)
  • Progressive disease
  • Adverse events
  • Note: Patients are eligible if additionally they have failed an ESA
  • HMA Naïve group:
  • Never received a hypomethylating agent for MDS
  • Failed or ceased to respond to ESA(s)
  • ESA ineligible; defined as endogenous serum erythropoietin level > 200 U/L for subjects not previously treated with ESAs
  • Red blood cell transfusion dependent defined as ≥ 2 Red blood cells (RBC) units required in the 8 weeks prior to starting in the study. In addition, there should be no 8 consecutive weeks without red blood cell transfusions in the 16 weeks prior to enrolment.
  • Life expectancy ≥ 3 months
  • Eastern Cooperative Oncology Group (ECOG) performance status Grade 0-2
  • Serum bilirubin levels ≤2 x upper limits of normal (ULN)
  • Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels ≤2.5 x ULN
  • Del 5q patients who have failed or are not eligible for Revlimid
  • Creatinine clearance >30 ml/min calculated by the Cockcroft-Gault formula
  • Willingness to comply with the protocol procedures for the duration of the study, including scheduled follow-up visits and examinations
  • Negative urine β-human chorionic gonadotropin (β-HCG) pregnancy test for fertile women at screening and confirmed by serum pregnancy test in the 48 hours prior to OPN-305 administration
  • If sexually active female, patient must be/have one of the following:
  • Post-menopausal defined as the absence of menses for at least one year (serum Follicle-stimulating hormone (FSH) ≥20IU/L can also be measured according to local practice),OR
  • Surgically sterile defined as a bilateral tubal ligation at least 6 months prior to administration of study drug, bilateral oophorectomy, or complete hysterectomy, OR
  • Using an effective means of contraception that is planned to continue for the duration of treatment and for a further 3 months.
  • If sexually active male, patient must: Agree to use an effective means of contraception (per site-specific guidelines) that is planned to continue until 6 months after the last dose of OPN-305.Agree not to donate sperm until 6 months after the last dose of OPN-305

Who cannot join

  • Diagnosis of MDS by bone marrow aspirate of Intermediate-2 and High risk category MDS based on the World Health Organization (WHO) classification using the IPSS (International Prognostic Scoring System)
  • Patients with 5q deletion (del) MDS eligible for Revlimid (lenalidomide)
  • Hypomethylating agent (HMA) Naïve group:
  • Have received a hypomethylating agent for MDS
  • Have not failed or ceased to respond to an ESA
  • Are not ESA ineligible as defined in inclusion criteria
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  • Prior history of acute leukemia or AML
  • Unable/unwilling to undergo bone marrow sampling
  • Prior history of bone marrow transplantation
  • Prior malignancy (other than non-invasive malignancy including in situ cervical cancer, Bowen's disease, basal cell cancer of the skin and non-invasive or excised skin squamous cell carcinoma) unless treated with curative intent and without evidence of disease for 3 years before randomization
  • Active viral or bacterial infections: this includes any infections that are being actively treated even if the signs and symptoms appear to have resolved. Courses of antibiotics or anti-viral treatment should be completed before the patients is enrolled
  • Unstable angina, congestive heart failure [NYHA (New York Heart Association) >class II], uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction, uncontrolled diabetes mellitus
  • Clinical Evidence of Central Nervous System (CNS) disease
  • Less than 4 weeks since any therapy for MDS
  • Prior history of anaphylaxis to similar products
  • History or presence of a medical condition or disease or substance abuse that in the investigator's assessment would place the patient at an unacceptable risk for study participation
  • Lactating or pregnant woman

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Research Site

    Houston, Texas, 77030

  • Research Site

    New York, New York, 10021

  • Research Site

    Tampa, Florida, 33613

  • Research Site

    The Bronx, New York, 10467

Other trials in United States

About this study

Official title: A Prospective, Open Label Phase I/II Study to Assess the Safety and Efficacy of Cycles of Intravenously Infused Doses of OPN-305 in Second-line or Third-line Lower (Low and Intermediate-1) Risk Myelodysplastic Syndrome (MDS)

The dose-confirming part of this study, comprising at least 10 patients is designed as a single center, prospective, single arm, open label in patients who have failed or are unresponsive to Azacitidine (AZA) or Decitabine (they may also have additionally failed an Erythropoiesis Stimulating Agent (ESA) followed by a dose expansion part with at least 44 patients; the objective of the whole study being to assess the safety, efficacy, pharmacokinetics and pharmacodynamics of intravenously infused multiple doses of OPN-305 in low and intermediate-1 risk myelodysplastic syndrome (second and third line Lower risk MDS).

Being tested
OPN-305
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OPN-305Drug

    For the dose confirming part of the study, patients will receive a starting dose of 5 mg/kg OPN-305.

Study groups in NCT02363491
Study groupRoleWhat they receive
OPN-305ExperimentalOPN-305
Receives: Drug: OPN-305

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Establishment of the dose and dose frequency based on dose-limiting toxicity and bone marrow receptor occupancy of OPN-305 in low and intermediate -1 (Lower) risk MDS

    Measured: 8 weeks

  2. Tolerability of OPN-305 as monotherapy based on adverse events

    Measured: 16 weeks/32 weeks (if there is no AZA add-back)

  3. Tolerability of OPN-305 as monotherapy and in combination with AZA based on adverse events

    Measured: 32 weeks

Secondary outcomes (9)

  1. Hematological response based on International Working Group (IWG) 2000/2006

    Measured: week 36

  2. Cytokine levels in serum (TNFα, IL-1β, IL-6, IL-10, IL-12, IL-18, IL-23 and IFN-γ)

    Measured: day 1 and week 4

  3. Immunogenicity of OPN-305 (Measurement of anti drug antibodies and neutralizing antibodies)

    Measured: day 1, weeks 4, 8, 16, 24 and 32

  4. Incidence of infections

    Measured: 36 weeks

  5. Pharmacokinetic profile of OPN-305 (maximum concentration (Cmax))

    Measured: day 1, weeks 4, 8, 12, 16, 20, 24, 28, 32

  6. Pharmacokinetic profile of OPN-305 (time at which Cmax is attained (tmax))

    Measured: day 1, weeks 4, 8, 12, 16, 20, 24, 28, 32

  7. OPN-305 receptor occupancy in peripheral monocytes, bone marrow cells and stroma

    Measured: screening (bone marrow only), day 1 (blood only), wks 4 (blood only), 8, 12 (blood only), 16, 20 (blood only), 24 (blood only), 28 (blood only), 32 and 36 (blood only)

  8. Correlation of clinical response with cytogenical observations

    Measured: wk 36

  9. Quality of life MD Anderson Symptom Inventory (MDASI) - Acute Myeloid Leukemia (AML)/Myelodysplastic syndrome (MDS) questionnaire

    MDASI is MD Anderson symptom inventory. It has two scales

    1. Severity of symptoms scale 0-10 with 0 being not present and 10 being as bad as you can imagine
    2. How symptoms interfere with life scale 0-10 with 0 did not interfere and 10 interfere completely

    Measured: wk 36

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Feb 2015 (estimated)
  2. Study startJan 2015
  3. Primary completionDec 2018
  4. Study completionDec 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
96 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02363491
Sponsor's protocol ID
OPN-305-106
Status last verified
Jan 2019
First submitted
21 Jan 2015
Last updated
28 Jan 2019
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing OPN-305 in people with Myelodysplastic Syndrome, run by Opsona Therapeutics Ltd. at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 28 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Establishment of the dose and dose frequency based on dose-limiting toxicity and bone marrow receptor occupancy of OPN-305 in low and intermediate -1 (Lower) risk MDS (time frame: 8 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02363491.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jan 2019.

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