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Recurrent Adult Acute Myeloid LeukemiaPhase 1NCT01159028

Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDS

A Phase 1 study testing BP1001 and BP1001 in combination with LDAC in people with Recurrent Adult Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Bio-Path Holdings, Inc. at 1 site 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 first goal of this clinical research study is to find the highest safe dose of BP1001, a liposomal Growth Factor Receptor Bound Protein-2 antisense oligodeoxynucleotide (L-Grb2 AS), for patients with Philadelphia Chromosome positive CML, AML, ALL and MDS. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
BP1001 and BP1001 in combination with LDAC
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
1 siteUnited States
Study dates
Jun 2010to 30 Mar 2017
Phase
Phase 1
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 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female patients 18 years of age or older
  • A diagnosis of refractory or relapsed AML, or Ph+ CML (in chronic, accelerated or blast phase, or acute lymphoblastic leukemia, or myelodysplastic syndrome.
  • One of the following parameters is required to meet criteria for accelerated phase CML:
  • Blasts in Peripheral Blood or Bone Marrow ≥15%
  • Promyelocytes and Blasts in Peripheral Blood or Bone Marrow ≥30%
  • PB or BM basophils ≥20%
Show all 21Show fewer
  • Thrombocytopenia <100 x 103/ml, not resulting from therapy
  • Blast phase is defined as ≥30% blasts in peripheral blood or bone marrow, or presence of extramedullary disease, except for liver or spleen.
  • Patients with CML must have demonstrated resistance and/or intolerance to therapy with at least 2 tyrosine kinase inhibitors (TKI)
  • Patients with AML and ALL should have received at least 1 prior treatment regimen and either failed to achieve response or relapsed on treatment
  • Patients with MDS should have failed prior therapy with a hypomethylating agent or, if associated with a 5q- chromosomal abnormality, lenalidomide. NOTE: Patients with 5q- unable to receive or intolerant to lenalidomide are also eligible.
  • Have clinically adequate hepatic and renal functions as defined by:
  • ALT<2x ULN
  • Serum creatinine concentration <2x ULN
  • Serum bilirubin <2x ULN
  • Patients must sign an informed consent
  • Women of childbearing age must have a negative serum or urine pregnancy test prior to the initiation of study drug.
  • Barrier contraceptive precautions are to be used throughout the trial by all study participants of child bearing potential.
  • Have not received anti-cancer therapy for at least 2 weeks prior to study entry, with the exception of low dose ara-C (LDAC) given as subcutaneous injections (no less than 15 days prior), hydroxyurea or anagrelide (no less than 24 hours prior), TKI (no less than 5 days prior), and interferon (no less than 2 weeks prior)
  • Have an ECOG Performance of 0-2
  • Have a life-expectancy ≥3 months

Who cannot join

  • Serious intercurrent medical illnesses which would interfere with the ability of the patient to carry out the treatment program
  • Pregnant or breastfeeding women
  • Patients who have uncontrolled active infection
  • Patients who have received another investigational product within the longer of 14 days or 5 half-lives of the previous product
  • Any history of adverse reaction or hypersensitivity to LDAC
  • Part B: BP1001 with Concurrent LDAC Dose-Expansion Cohorts
  • Enrollment in the dose-expansion cohorts (DEC) will be limited to only those patients with a diagnosis of refractory or relapsed AML(except acute promyelocytic leukemia) or those who are refractory to at least 1 prior therapy regimen and no more than 1 prior salvage regimen.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M. D. Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: A Phase I Clinical Trial to Study the Safety, Pharmacokinetics, and Efficacy of BP1001 (L-Grb-2 Antisense Oligonucleotide) in Patients With Refractory or Relapsed Acute Myeloid Leukemia, Philadelphia Chromosome Positive Chronic Myelogenous Leukemia, or Acute Lymphoblastic Leukemia, and Myelodysplastic Syndrome

The first goal of this clinical research study is to find the highest safe dose of BP1001, a liposomal Growth Factor Receptor Bound Protein-2 antisense oligodeoxynucleotide (L-Grb2 AS), for patients with Philadelphia Chromosome positive CML, AML, ALL and MDS. The response of the leukemia to this treatment will also be studied. The second goal of this clinical research study is to evaluate the safety and toxicity of the combination of BP1001 and concurrent low-dose ara-C (LDAC) in patients with AML.

Read the detailed description

The Philadelphia Chromosome is an unusual genetic trait found in 90-95% of patients with CML and approximately 20-25% of patients with ALL. The protein created by this unusual trait causes normal cells within the body to become cancer cells, and then causes these cells to grow and divide at a rapid rate. Researchers think that the protein "Growth Factor Receptor Bound Protein-2 (Grb-2)" plays an important role in the rapid growth of leukemic cells. The study drug (BP1001) may be able to inhibit the cells from making Grb-2. Researchers hope that without this protein, the leukemia cells will die.

Up to 60 patients are expected to be enrolled on this study.

Part A: Dose escalation: Each cohort will receive BP1001 at a dose higher than the previous group.

Part B: Dose-expansion Cohorts: Subjects with relapsed or refractory AML will receive escalating doses of BP1001 concurrently with fixed low-dose ara-C (LDAC)

The study drug is an antisense molecule complementary to the messenger RNA (mRNA) code for the cell's expression of the protein Grb-2. The study drug is incorporated into lipid (fat) particles known as liposomes. This incorporation process is part of the manufacturing process and is done before the study drug is administered. The liposomes (which carry the study drug) will be administered intravenously twice a week for 28 days. Subjects enrolled in Part B of the study will receive study drug twice a week for 28 days concurrently with low dose ara-C, self administered twice daily for 10 consecutive days.

Being tested
BP1001, BP1001 in combination with LDAC
Keywords
Liposomal Grb-2 treatment of CML, AML, CLL, MDS, Liposomal Grb-2 with LDAC for AML
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

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

  • BP1001Drug

    Also called Liposomal Grb-2, L-Grb-2, BP-100-1.01

    Study drug (BP1001) is constituted in normal saline, administered by IV on twice weekly for 28 days.

  • BP1001 in combination with LDACDrug

    Also called Liposomal Grb-2, L-Grb-2, BP-100-1.01, low dose ara-C

    Study drug (BP1001) is constituted in normal saline, administered by IV twice weekly for 28 days. Low dose ara-C (LDAC) is self administered twice daily for 10 consecutive days during the 28 day cycle.

Study groups in NCT01159028
Study groupRoleWhat they receive
BP1001ExperimentalSubjects are treated with open-label study drug (BP1001) in a dose-escalation model.
Receives: Drug: BP1001
BP1001 in combination with LDACExperimentalAML subjects are treated with open-label escalating study drug (BP1001) in combination with low dose ara-C (LDAC)
Receives: Drug: BP1001 in combination with LDAC

What is being measured

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

  1. Safety of BP1001

    Evaluate toxicity, tolerance, and MTD of escalating doses of BP1001

    Measured: 30 days

  2. Safety of BP1001 in combination with LDAC

    Evaluate safety and toxicity of the combination of BP1001 and concurrent LDAC using non-hematologic and hematologic measures per NCI CTCAE criteria.

    Measured: 30 days

Secondary outcomes (3)

  1. Optimal biologically active dose

    Determine the optimal biologically active dose of BP1001 defined as a 50% reduction in Grb-2 expression in circulating leukemia cells

    Measured: 30 days

  2. In vivo pharmacokinetics

    Evaluate the in vivo PK of BP1001 in all subjects using plasma and urine to compute half life and elimination

    Measured: 30 days

  3. Correlate PK data with historical experience

    Correlate the in vivo PK data with historical experience to demonstrate the liposomal delivery performs as expected for all subjects by comparing PK data (half life and elimination) obtained from each subject with historical experience

    Measured: 30 days

Timeline

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

  1. First posted9 Jul 2010 (estimated)
  2. Study startJun 2010
  3. Primary completion30 Mar 2017
  4. Study completion30 Mar 2017

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01159028
Sponsor's protocol ID
2003-0578 (v) 08-8
Status last verified
May 2020
First submitted
7 Jul 2010
Last updated
28 May 2020
Results posted
No
Data monitoring committee
Yes

Publications

  • Ohanian M, Tari Ashizawa A, Garcia-Manero G, Pemmaraju N, Kadia T, Jabbour E, Ravandi F, Borthakur G, Andreeff M, Konopleva M, Lim M, Pierce S, O'Brien S, Alvarado Y, Verstovsek S, Wierda W, Kantarjian H, Cortes J. Liposomal Grb2 antisense oligodeoxynucleotide (BP1001) in patients with refractory or relapsed haematological malignancies: a single-centre, open-label, dose-escalation, phase 1/1b trial. Lancet Haematol. 2018 Apr;5(4):e136-e146. doi: 10.1016/S2352-3026(18)30021-8. Epub 2018 Mar 14.

    PubMed 29550383

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BP1001 and BP1001 in combination with LDAC in people with Recurrent Adult Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Bio-Path Holdings, Inc. at 1 site 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 21 inclusion and 7 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 May 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 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.

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?

Safety of BP1001 (time frame: 30 days). The study lists 2 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 NCT01159028, which also lists 1 linked publication.

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 May 2020; AzTrials last refreshed its copy on 7 Nov 2026.

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