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CompletedNCT02009332Updated Jun 8, 2021Results posted

Phase 1/2 Study of ABI-009 in Nonmuscle Invasive Bladder Cancer

A Phase 1/2 interventional study of ABI-009 and Gemcitabine in Non-muscle Invasive Bladder Cancer (NMIBC), sponsored by Aadi Bioscience, Inc.. Completed at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-06-08.

Sponsored by Aadi Bioscience, Inc. · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
21
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

Purpose of this study is to determine appropriate dosing of ABI-009 and evaluate the safety and anti-tumor activity of ABI-009 in treatment of non-muscle invasive bladder cancer

02

Conditions studied

  • Non-muscle Invasive Bladder Cancer (NMIBC)
03

In context

Urinary Bladder Neoplasms

1,616 studies on the registry are indexed under Urinary Bladder Neoplasms; 421 are open to participants now.

This study's enrollment of 21 is below the median of 60 across 1,163 interventional studies indexed under Urinary Bladder Neoplasms.

Browse Urinary Bladder Neoplasms studies →

Lead sponsor

Aadi Bioscience, Inc. is the lead sponsor of 11 studies on the registry; 2 are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

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

Inclusion criteria

  1. Patients must have a diagnosis of transitional cell carcinoma (TCC) of the urinary bladder confirmed at the study institution. The patient must have demonstrated nonmuscle-invasive bladder cancer refractory or recurrent to standard intravesical therapy. Refractory disease is defined as failure to achieve tumor-free status by 6 months of initiation of adequate BCG therapy. Recurrent disease is defined as reappearance of disease after achieving a tumor-free status by 6 months of initiation of adequate BCG therapy. Adequate BCG therapy includes at least 6 weeks induction plus 3 additional doses of either induction or maintenance. Patients with a history of other intravesical agents (except nab-rapamycin or gemcitabine) in addition to standard BCG will also be allowed to enroll. All grossly visible disease must be fully resected and pathologic stage will be confirmed at the institution where the patient is enrolled. This will include stage Ta, T1, Tis and exclude all patients with muscle invasion (T2).

    1. For phase 1, patients with multifocal low-grade Ta histology will be eligible for participation
    2. For phase 2, individuals with Ta disease only must have documentation of high-grade histology
    3. For phase 2, prior intravesical treatment with nab-rapamycin or gemcitabine is not allowed
  2. Age >18 and must be able to read, understand, and sign informed consent
  3. Performance Status: ECOG 0, 1, and 2 (See Appendix III)

  4. Hematologic inclusion within 2 weeks of start of treatment

    1. Absolute neutrophil count >1,500/mm3
    2. Hemoglobin >9.0 g/dl
    3. Platelet count >100,000/mm3

  5. Hepatic inclusion within 2 weeks of entry

    1. Total bilirubin must be within normal limits.
    2. Adequate renal function with serum creatinine ≤2.5 mg/dL
    3. Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 x ULN for the institution, alkaline phosphatase ≤ 2.5 x ULN for the institution, unless bone metastasis is present in the absence of liver metastasis
  6. Women of childbearing potential must have a negative pregnancy test.
  7. All patients of childbearing potential must be willing to consent to using effective contraception, ie, intrauterine device, birth control pills, depo-provera, and condoms while on treatment and for 3 months after their participation in the study ends.

Exclusion criteria

Exclusion Criteria:

  1. Any other malignancy diagnosed within 1 year of study entry (except basal or squamous cell skin cancers or noninvasive cancer of the cervix) is excluded
  2. Concurrent treatment with any chemotherapeutic agent
  3. Women who are pregnant or lactating
  4. History of vesicoureteral reflux or an indwelling urinary stent
  5. Participation in any other research protocol involving administration of an investigational agent within 1 month prior to study entry
  6. History of radiation to the pelvis
  7. History of interstitial lung disease and/or pneumonitis
  8. Evidence of metastatic disease
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    Phase 1: ABI-009 100 mg/week

    Phase 1, Cohort 1: ABI-009 injectable suspension, 100 mg in 80 mL 0.9% saline, administered intravesically and retained for 2 hours, once per week for 6 weeks

    Drug: ABI-009

  • Experimental
    Phase 1: ABI-009 200 mg/week

    Phase 1, Cohort 2: ABI-009 injectable suspension, 200 mg in 80 mL 0.9% saline, administered intravesically and retained for 2 hours, once per week for 6 weeks

    Drug: ABI-009

  • Experimental
    Phase 1: ABI-009 100 mg 2×/week

    Phase 1, Cohort 2b: ABI-009 injectable suspension, 100 mg in 80 mL 0.9% saline, administered intravesically and retained for 2 hours, twice per week (total dose 200 mg per week) for 6 weeks

    Drug: ABI-009

  • Experimental
    Phase 1: ABI-009 300 mg/week

    Phase 1, Cohort 3: ABI-009 injectable suspension, 300 mg in 80 mL 0.9% saline, administered intravesically and retained for 2 hours, once per week for 6 weeks

    Drug: ABI-009

  • Experimental
    Phase 1: ABI-009 400 mg/week

    Phase 1, Cohort 4: ABI-009 injectable suspension, 400 mg in 80 mL 0.9% saline, administered intravesically and retained for 2 hours, once per week for 6 weeks

    Drug: ABI-009

  • Experimental
    Phase 2: ABI-009 400 mg/week + Gemcitabine 2000 mg/week

    ABI-009 injectable suspension, 200 mg in 80 mL 0.9% saline, administered intravesically and retained for 1 hour, once per week for 6 weeks; Gemcitabine, 2000 mg in 100 mL saline, administered intravesically after voiding of ABI-009 and retained for 1 hour, once per week for 6 weeks

    Drug: ABI-009 · Drug: Gemcitabine

Interventions

  • DrugABI-009

    ABI-009 is a nanoparticle albumin-bound (nab®) formulation of the mammalian Target of Rapamycin ( mTOR) inhibitor, sirolimus. Specifically, ABI-009 is a sterile lyophilized powder of albumin-bound sirolimus nanoparticles with a mean particle size of less than 100 nm.

    Also known as: nab-sirolimus, nab-rapamycin

  • DrugGemcitabine

    Gemcitabine is administered after ABI-009 in the Phase 2 study.

06

What researchers measure

Primary outcomes

  1. Phase 1: Dose Limiting Toxicities (DLT) Following Intravesical Administration of ABI-009

    The primary endpoint of the Phase 1 study is DLT following intravesical administration of ABI-009 in patients with BCG refractory or recurrent nonmuscle-invasive transitional cell carcinoma (TCC) of the bladder to identify maximum deliverable dose (MDD). Systemic DLT will be defined as any grade systemic toxicity using the NCI CTCAE version 4.0. Local dose limiting toxicity was defined as grade 3 or 4 bladder toxicity (hematuria, dysuria, urinary retention, urinary frequency/urgency, or bladder spasms) using the NCI CTCAE version 4.0.

    Time frame: Duration of treatment (6 weeks) plus 30 days follow up (up to 2.5 months)

  2. Phase 2: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009 and Gemcitabine

    The primary objective of the Phase 2 study is to evaluate the utility (potential for clinical efficacy) of ABI-009 in combination with gemcitabine in the treatment of BCG refractory or recurrent nonmuscle-invasive TCC of the bladder. Response rate will be measured and documented at the 6-week post-treatment assessment, including cystoscopy with biopsy. A complete response is defined as a cancer-negative biopsy at the 6-week post-treatment cystoscopy. No response will be defined as positive cystoscopic biopsy.

    Time frame: End of Study [EOS, 3 months]

Secondary outcomes

  1. Phase 1: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009

    Response rate will be measured and documented at the 6-week post-treatment assessment, including cystoscopy with biopsy. A complete response is defined as a cancer-negative biopsy at the 6-week post-treatment cystoscopy. No response will be defined as positive cystoscopic biopsy.

    Time frame: End of Study [EOS, 3 months]

07

Results

Posted Jun 8, 2021

Participant flow

Phase 1: Dose Level 1 (Weeks 1-10)
Participant flow — Phase 1: Dose Level 1 (Weeks 1-10)
MilestonePhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 200 mg/Week + Gemcitabine 2000 mg/Week
Started300000
Completed300000
Not completed000000
Phase 1: Dose Level 2/2b (Wks 11-29)
Participant flow — Phase 1: Dose Level 2/2b (Wks 11-29)
MilestonePhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 200 mg/Week + Gemcitabine 2000 mg/Week
Started041000
Completed031000
Not completed010000
Withdrew: Physician decision010000
Phase 1: Dose Level 3 (Weeks 30-47)
Participant flow — Phase 1: Dose Level 3 (Weeks 30-47)
MilestonePhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 200 mg/Week + Gemcitabine 2000 mg/Week
Started000300
Completed000300
Not completed000000
Phase 1: Dose Level 4 (Weeks 48-77)
Participant flow — Phase 1: Dose Level 4 (Weeks 48-77)
MilestonePhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 200 mg/Week + Gemcitabine 2000 mg/Week
Started000040
Completed000030
Not completed000010
Withdrew: Withdrawal by subject000010
Phase 2 (Weeks 206-252)
Participant flow — Phase 2 (Weeks 206-252)
MilestonePhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 200 mg/Week + Gemcitabine 2000 mg/Week
Started000006
Completed000005
Not completed000001
Withdrew: Withdrawal by subject000001

Outcome measures

PrimaryPhase 1: Dose Limiting Toxicities (DLT) Following Intravesical Administration of ABI-009

The primary endpoint of the Phase 1 study is DLT following intravesical administration of ABI-009 in patients with BCG refractory or recurrent nonmuscle-invasive transitional cell carcinoma (TCC) of the bladder to identify maximum deliverable dose (MDD). Systemic DLT will be defined as any grade systemic toxicity using the NCI CTCAE version 4.0. Local dose limiting toxicity was defined as grade 3 or 4 bladder toxicity (hematuria, dysuria, urinary retention, urinary frequency/urgency, or bladder spasms) using the NCI CTCAE version 4.0.

Time frame:
Duration of treatment (6 weeks) plus 30 days follow up (up to 2.5 months)
Reported as:
Count of participants · Participants
Phase 1: Dose Limiting Toxicities (DLT) Following Intravesical Administration of ABI-009
ParticipantsPhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/Week
Phase 1: Dose Limiting Toxicities (DLT) Following Intravesical Administration of ABI-00900000
Statistical analysis
  • Phase 1: ABI-009 100 mg/Week vs Phase 1: ABI-009 200 mg/Week vs Phase 1: ABI-009 100 mg 2×/Week vs Phase 1: ABI-009 300 mg/Week vs Phase 1: ABI-009 400 mg/Week · Maximum deliverable dose (mdd): 400Maximum deliverable dose (MDD) was not reached in the Phase 1 study as no DLTs were observed in any of the dose groups up to ABI-009 400 mg/week.
PrimaryPhase 2: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009 and Gemcitabine

The primary objective of the Phase 2 study is to evaluate the utility (potential for clinical efficacy) of ABI-009 in combination with gemcitabine in the treatment of BCG refractory or recurrent nonmuscle-invasive TCC of the bladder. Response rate will be measured and documented at the 6-week post-treatment assessment, including cystoscopy with biopsy. A complete response is defined as a cancer-negative biopsy at the 6-week post-treatment cystoscopy. No response will be defined as positive cystoscopic biopsy.

Time frame:
End of Study [EOS, 3 months]
Reported as:
Count of participants · Participants
Phase 2: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009 and Gemcitabine
ParticipantsPhase 2: ABI-009 400 mg/Week + Gemcitabine 2000 mg/Week
Phase 2: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009 and Gemcitabine1
SecondaryPhase 1: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009

Response rate will be measured and documented at the 6-week post-treatment assessment, including cystoscopy with biopsy. A complete response is defined as a cancer-negative biopsy at the 6-week post-treatment cystoscopy. No response will be defined as positive cystoscopic biopsy.

Time frame:
End of Study [EOS, 3 months]
Reported as:
Count of participants · Participants
Phase 1: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-009
ParticipantsPhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/Week
Phase 1: Number of Participants Achieving a Complete Response Following Intravesical Administration of ABI-00901001

Adverse events

Collected over From the signing of the informed consent through the 6-week induction treatment period and the 30-day follow-up period (up to 2.5 months). An additional 4 months for patients who received 3 additional monthly maintenance treatments (up to 6.5 months).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Phase 1: ABI-009 100 mg/Week0/3 (0%)0/3 (0%)2/3 (66.7%)
Phase 1: ABI-009 200 mg/Week0/4 (0%)0/4 (0%)1/4 (25%)
Phase 1: ABI-009 100 mg 2×/Week0/1 (0%)0/1 (0%)1/1 (100%)
Phase 1: ABI-009 300 mg/Week0/3 (0%)0/3 (0%)3/3 (100%)
Phase 1: ABI-009 400 mg/Week0/4 (0%)0/4 (0%)3/4 (75%)
Phase 2: ABI-009 400 mg/Week + Gemcitabine 2000 mg/Week0/6 (0%)0/6 (0%)6/6 (100%)
Most frequent other events
Showing 10 of 33
Most frequent other events
EventPhase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2×/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2: ABI-009 400 mg/Week + Gemcitabine 2000 mg/Week
HematuriaRenal and urinary disorders0/30/41/12/31/40/6
Urinary tract infectionRenal and urinary disorders0/30/41/11/31/42/6
Kidney stoneRenal and urinary disorders0/30/41/10/30/40/6
AnemiaBlood and lymphatic system disorders1/30/41/11/30/41/6
ThrombocytopeniaBlood and lymphatic system disorders1/30/41/10/30/40/6
Decreased absolute neutrophilBlood and lymphatic system disorders0/30/41/10/30/41/6
Decreased white blood cellBlood and lymphatic system disorders0/30/41/10/30/40/6
Bladder spasmRenal and urinary disorders0/30/40/10/30/45/6
Urinary tract painRenal and urinary disorders0/30/40/10/30/43/6
Penis Lesion/rashReproductive system and breast disorders1/30/40/11/30/40/6

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Phase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2x/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2, EfficacyTotal
<=18 years0000000
Between 18 and 65 years0300003
>=65 years31134618
Sex: Female, Male
Sex: Female, Male(Participants)Phase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2x/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2, EfficacyTotal
Female1000012
Male24134519
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Phase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2x/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2, EfficacyTotal
American Indian or Alaska Native0000000
Asian0000000
Native Hawaiian or Other Pacific Islander0000000
Black or African American0000000
White34134621
More than one race0000000
Unknown or Not Reported0000000
Region of Enrollment
Region of Enrollment(participants)Phase 1: ABI-009 100 mg/WeekPhase 1: ABI-009 200 mg/WeekPhase 1: ABI-009 100 mg 2x/WeekPhase 1: ABI-009 300 mg/WeekPhase 1: ABI-009 400 mg/WeekPhase 2, EfficacyTotal
United States34134621
08

Study locations

2 sites
  • Columbia University Medical Center
    New York, New York 10032, United States
  • Vanderbilt University Medical Center
    Nashville, Tennessee 37232, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Apr 20, 2016

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 8, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02009332
Lead sponsor
Aadi Bioscience, Inc.
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Dec 12, 2013
Start date
Apr 9, 2014
Primary completion
Dec 1, 2019
Completion
Dec 1, 2019
Results posted
Jun 8, 2021
Last update
Jun 8, 2021

Study contacts

James McKiernan, MD
principal investigator · Columbia University

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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