DS Stage I Plasma Cell MyelomaPhase 2NCT01251172
RO4929097 After Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma
A Phase 2 study testing Autologous Hematopoietic Stem Cell Transplantation, Gamma-Secretase Inhibitor RO4929097 and Hematopoietic Stem Cell Mobilization in people with DS Stage I Plasma Cell Myeloma and DS Stage II Plasma Cell Myeloma, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
This phase II clinical trial is studying how well gamma-secretase/Notch signalling pathway inhibitor RO4929097 (RO4929097) after autologous stem cell transplant works in treating patients with multiple myeloma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- DS Stage I Plasma Cell Myeloma, DS Stage II Plasma Cell Myeloma, DS Stage III Plasma Cell Myeloma
- What is being tested?
- Autologous Hematopoietic Stem Cell Transplantation, Gamma-Secretase Inhibitor RO4929097 and Hematopoietic Stem Cell Mobilization
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2010to Jul 2012
- Phase
- Phase 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
- Patients must have a diagnosis of multiple myeloma as defined below with staging based on the International Staging System:
- Multiple myeloma - all three required (Note: these criteria identify stage 1B and stages II and IIIA/B myeloma by Durie/Salmon stage; stage 1A becomes smoldering or indolent myeloma):
- Monoclonal plasma cells in the bone marrow ≥ 10% and/or presence of a biopsy-proven plasmacytoma
- Monoclonal protein present in the serum and/or urine (if no monoclonal protein is detected [non-secretory disease], then >= 30% monoclonal bone marrow plasma cells and/or biopsy-proven plasmacytoma is required)
- Myeloma-related organ dysfunction (1 or more) (a variety of other types of end organ dysfunctions can occasionally occur and lead to a need for therapy; such dysfunction is sufficient to support classification as myeloma if proven to be myeloma related):
- Calcium elevation in the blood (serum calcium > 10.5 mg/L or upper limit of normal)
Show all 54Show fewer
- Renal insufficiency (serum creatinine > 2mg/dL)
- Anemia (hemoglobin < 10 g/dL or 2 g < normal)
- Lytic bone lesions or osteoporosis (if a solitary [biopsy-proven] plasmacytoma or osteoporosis alone [without fractures] are the sole defining criteria, then > 30% plasma cells are required in the bone marrow)
- Patients must have measurable disease
- Patients with non-secretory multiple myeloma will be eligible only if the baseline serum free light chain level is elevated (>= 10 mg/dL)
- For therapeutic treatment with RO4929097, patients must have peripheral blood stem cell collection of >= 4.0 x 10^6 cells/kg (patient's ideal body weight)
- ECOG performance status =< 2, Karnofsky >= 60%
- Estimated life expectancy of at least 12 weeks
- Absolute neutrophil count (ANC) >= 1,500/mcL
- Platelets >= 100,000/mcL
- Hemoglobin >= 9.0 g/dL
- NOTE: Patients should not require chronic supportive growth factor administration or transfusions to meet treatment eligibility criteria (e.g., G-CSF for ANC eligibility)
- Serum creatinine =< 2.0 mg/dL OR a measured creatinine clearance by 24-hour urine collection >= 40 mL/min (a calculated creatinine clearance by Cockcroft-Gault formula is acceptable in lieu of a measured value)
- Total bilirubin within normal institutional limits
- AST (SGOT)/ALT SGPT) =< 2.5 X institutional ULN
- Patients with uncontrolled electrolyte abnormalities including hypocalcemia, hypomagnesemia, hyponatremia, hypophosphatemia, or hypokalemia, defined as less than the lower limit of normal for the institution despite adequate electrolyte supplementation, are excluded from this study
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use two forms of contraception (i.e., barrier contraception and one other method of contraception) at least 4 weeks prior to study entry, for the duration of study participation, and for at least 12 months post-treatment
- No requirement for routine use of hematopoietic growth factors (including granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, or interleukin-11) or platelet transfusions to maintain absolute neutrophil counts or platelet counts above the required thresholds for study entry
- Use of erythropoietin-stimulating agents and red cell transfusions are also not permitted prior to initiation of RO4929097
- Patient must meet the institutional standard criteria for undergoing high-dose chemotherapy and autologous stem cell transplant
- No serious concomitant systemic disorders (including active infections) that would compromise the safety of the patient or compromise the patient's ability to complete the study, at the discretion of the investigator
- No uncontrolled intercurrent illness including, but not limited to:
- Ongoing or active infection
- Symptomatic congestive heart failure
- Unstable angina pectoris
- A history of torsades de pointes
- Cardiac arrhythmia other than chronic, stable atrial fibrillation
- Psychiatric illness/social situations that would limit compliance with study requirements
- No baseline QTcF > 450 msec (male) or QTcF > 470 msec (female)
- Patients with malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel are excluded
- Subjects with ulcerative colitis, inflammatory bowel disease, or a partial or complete small bowel obstruction are also excluded
- No condition (e.g., gastrointestinal tract disease resulting in an inability to take oral medication or a requirement for IV alimentation, prior surgical procedure affecting absorption, or active peptic ulcer disease) that impairs the ability to swallow and retain RO4929097
- Patients who have active hepatitis B or C, or have a history of liver disease, other forms of hepatitis or cirrhosis are ineligible
- HIV-positive patients on combination antiretroviral therapy are ineligible
- No second primary malignancy except most situ carcinoma (e.g., in situ carcinoma of the of the cervix, adequately treated non-melanomatous carcinoma of the skin) or other malignancy treated at least 3 years previously with no evidence of recurrence
- No history of allergic reactions attributed to compounds of similar chemical or biologic composition to RO4929097or other agents used in the study
- There are no limitations on type and amount of prior therapy with the following exception: patients who have had a solid organ transplant are ineligible
- Acute toxicities of high-dose melphalan therapy must have resolved to the NCI-CTCAE version 4.0 grade 1 or less (with the exception of alopecia)
- Patients taking medications with narrow therapeutic indices that are metabolized by cytochrome P450 (CYP450), including warfarin sodium (Coumadin®) are ineligible
- No concurrent medications that are strong inducers/inhibitors or substrates of CYP3A4
- No concurrent drugs, food, supplements, or over-the-counter medications that may interfere with the metabolism of RO4929097, including ketoconazole and fresh-squeezed grapefruit juice
- Patients may not be receiving any other investigational agents
- Patients may not be receiving antiarrhythmics or other medications known to prolong QTc
- No other investigational or commercial agents or therapies may be administered with the intent to treat the patient's malignancy
- No patients who have had any chemotherapy or radiotherapy post-autologous stem cell transplant prior to entering the therapeutic arm of the study or those who have not recovered from the high-dose melphalan and autologous stem cell transplant
- For therapeutic treatment with RO4929097, the acute toxicities of the high-dose melphalan therapy must have resolved to the NCI-CTCAE version 4.0 Grade 1 or less (with the exception of alopecia)
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of RO4929097 to Eradicate Residual Disease in Patients With Multiple Myeloma Post Single Autologous Stem Cell Transplant
This phase II clinical trial is studying how well gamma-secretase/Notch signalling pathway inhibitor RO4929097 (RO4929097) after autologous stem cell transplant works in treating patients with multiple myeloma. Giving chemotherapy, such as melphalan, before autologous stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Before treatment, stem cells are collected from the patient's blood and stored. After chemotherapy, the stem cells are returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. RO4929097 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving RO4929097 after autologous stem cell transplant may kill more cancer cells.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the efficacy of RO4929097 in eradicating residual/persistent disease (conversion of VGPR to CR or sCR status) in multiple myeloma patients status post single high-dose melphalan/autologous stem cell rescue with residual/persistent disease post-transplant.
SECONDARY OBJECTIVES:
- To determine the safety of single agent RO4929097 post-autologous stem cell transplant in multiple myeloma patients.
II. To analyze Notch receptor and ligand protein expression in pre-transplant, pre-treatment with RO4929097, and post-treatment bone marrow biopsy samples.
III. To isolate and identify clonogenic multiple myeloma progenitor cells from bone marrow aspirate and/or peripheral blood at the following time points: pre-transplant, day 100 post-transplant, and post-treatment staging bone marrow biopsy.
IV. To analyze Notch receptor and ligand protein expression on isolated clonogenic progenitor myeloma cells.
- To characterize the tumor xenograft-initiating potential of clonogenic myeloma cells in adult Casper zebrafish and SCID-hu model and evaluate the drug toxicity and therapeutic potential of RO4929097 with this model.
VI. To analyze anti-angiogenic effects of RO4929097 as measured by reduction in microvessel density and VEGFR-1 expression in pre-treatment and post-treatment bone marrow biopsy samples.
VII. To determine whether measurement of soluble surrogate markers of angiogenesis prior to and following treatment with RO4929097 provide an early marker of disease response to Notch inhibition.
OUTLINE: This is a multicenter study.
STEM CELL TRANSPLANTATION AND CHEMOTHERAPY: Patients undergo standard mobilization and collection of autologous peripheral stem cells (>= 4.0 x 10^6 CD34+ cells/kg). Patients then receive high-dose melphalan IV on days -3 and -2 and undergo autologous stem cell transfusion on day 0. Patients with progressive disease, stable disease, partial response, stringent complete response, or complete response are taken off study; patients with residual/persistent disease (very good partial response [VGPR]*) continue to therapeutic treatment.
NOTE: *Patients who meet the criteria for VGPR but have findings of abnormal bone marrow cytogenetic study demonstrating >= 10% metaphase cells positive for either high-risk chromosomal abnormalities or persistent disease-related chromosomal abnormalities (e.g., chromosome del 13; del17p, t(4;14), t(14;16), t(6;14) or complex cytogenetics) will have a repeat bone marrow biopsy assessment with cytogenetics within 2-3 weeks. If the repeat biopsy findings suggest persistent high-risk cytogenetic findings, then the patient is considered eligible to receive RO4929097 to eradicate residual disease.
THERAPEUTIC TREATMENT: Beginning 100-110 days after transplantation, patients receive oral RO4929097 once daily on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for at least 4 weeks.
- Conditions
- DS Stage I Plasma Cell Myeloma, DS Stage II Plasma Cell Myeloma, DS Stage III Plasma Cell Myeloma, Refractory Plasma Cell Myeloma
- Being tested
- Autologous Hematopoietic Stem Cell Transplantation, Gamma-Secretase Inhibitor RO4929097, Hematopoietic Stem Cell Mobilization, In Vitro-Treated Peripheral Blood Stem Cell Transplantation, Laboratory Biomarker Analysis
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called Autologous Stem Cell Transplantation
Undergo in vitro treated autologous peripheral blood stem cell transplant
Gamma-Secretase Inhibitor RO4929097Drug
Also called RO4929097
Given orally
Hematopoietic Stem Cell MobilizationProcedure or surgery
Undergo standard mobilization
In Vitro-Treated Peripheral Blood Stem Cell TransplantationProcedure or surgery
Also called in vitro-treated PBPC transplantation, in vitro-treated peripheral blood progenitor cell transplantation
Undergo in vitro treated autologous peripheral blood stem cell transplant
Laboratory Biomarker AnalysisOther
Correlative studies
MelphalanDrug
Also called Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (RO4929097 after autologous stem cell transplant) | Experimental | STEM CELL TRANSPLANTATION AND CHEMOTHERAPY: Patients undergo standard mobilization and collection of autologous peripheral stem cells (>= 4.0 x 10^6 CD34+ cells/kg). Patients then receive high-dose melphalan IV on days -3 and -2 and undergo autologous stem cell transfusion on day 0. Patients with progressive disease, stable disease, partial response, stringent complete response, or complete response are taken off study; patients with residual/persistent disease (VGPR) continue to therapeutic treatment.
THERAPEUTIC TREATMENT: Beginning 100-110 days after transplantation, patients receive oral gamma-secretase inhibitor RO4929097 once daily on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Drug: Gamma-Secretase Inhibitor RO4929097; Procedure: Hematopoietic Stem Cell Mobilization; Procedure: In Vitro-Treated Peripheral Blood Stem Cell Transplantation; Other: Laboratory Biomarker Analysis; Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate of gamma-secretase/Notch signalling pathway inhibitor RO4929097 in eradicating residual/persistent disease, defined as conversion from VGPR to CR or sCR
A Simon's 2-stage optimal Phase II design will be used to monitor the study.
Measured: Up to 42 days
Secondary outcomes (2)
Clonogenic myeloma progenitor cells
Logistic regression models will be used to study the association of clinical response (relapse) and the myeloma stem cell kinetics (present and absent of cell, and/or percentage of cells).
Measured: At baseline, at 100 days post-transplant, and at 6 weeks
Incidence of adverse events and abnormal laboratory variables as assessed according to the NCI-CTCAE version 4.0
Adverse events will be reported in frequency tables overall, by intensity, and by relationship.
Measured: Up to 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2010 (estimated)
- Study startDec 2010
- Primary completionJul 2012
Why it stopped: Lack of Drug Supply
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DS Stage I Plasma Cell MyelomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Mecide Gharibo, Principal investigator, Rutgers Cancer Institute of New Jersey
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01251172
- Sponsor's protocol ID
- NCI-2011-02555
- Other IDs
- NCI-2011-02555, CDR0000689644, 011003, 8540, P30CA072720, U01CA132194
- Status last verified
- Sep 2017
- First submitted
- 30 Nov 2010
- Last updated
- 28 Sep 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of RO4929097 to Eradicate Residual Disease in Patients With Multiple Myeloma Post Single Autologous Stem Cell Transplant
- ClinicalTrials.gov ID
- NCT01251172
- Acronym / sponsor ID
- NCI-2011-02555
- Conditions
- DS Stage I Plasma Cell Myeloma, DS Stage II Plasma Cell Myeloma, DS Stage III Plasma Cell Myeloma, Refractory Plasma Cell Myeloma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of Drug Supply
- Enrolment
- 0 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Dec 2010
- Primary completion
- Jul 2012 (actual)
- First posted
- 1 Dec 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II clinical trial is studying how well gamma-secretase/Notch signalling pathway inhibitor RO4929097 (RO4929097) after autologous stem cell transplant works in treating patients with multiple myeloma. Giving chemotherapy, such as melphalan, before autologous stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Before treatment, stem cells are collected from the patient's blood and stored. After chemotherapy, the stem cells are returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. RO4929097 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving RO4929097 after autologous stem cell transplant may kill more cancer cells.
Detailed description
PRIMARY OBJECTIVES:
- To determine the efficacy of RO4929097 in eradicating residual/persistent disease (conversion of VGPR to CR or sCR status) in multiple myeloma patients status post single high-dose melphalan/autologous stem cell rescue with residual/persistent disease post-transplant.
SECONDARY OBJECTIVES:
- To determine the safety of single agent RO4929097 post-autologous stem cell transplant in multiple myeloma patients.
II. To analyze Notch receptor and ligand protein expression in pre-transplant, pre-treatment with RO4929097, and post-treatment bone marrow biopsy samples.
III. To isolate and identify clonogenic multiple myeloma progenitor cells from bone marrow aspirate and/or peripheral blood at the following time points: pre-transplant, day 100 post-transplant, and post-treatment staging bone marrow biopsy.
IV. To analyze Notch receptor and ligand protein expression on isolated clonogenic progenitor myeloma cells.
- To characterize the tumor xenograft-initiating potential of clonogenic myeloma cells in adult Casper zebrafish and SCID-hu model and evaluate the drug toxicity and therapeutic potential of RO4929097 with this model.
VI. To analyze anti-angiogenic effects of RO4929097 as measured by reduction in microvessel density and VEGFR-1 expression in pre-treatment and post-treatment bone marrow biopsy samples.
VII. To determine whether measurement of soluble surrogate markers of angiogenesis prior to and following treatment with RO4929097 provide an early marker of disease response to Notch inhibition.
OUTLINE: This is a multicenter study.
STEM CELL TRANSPLANTATION AND CHEMOTHERAPY: Patients undergo standard mobilization and collection of autologous peripheral stem cells (>= 4.0 x 10^6 CD34+ cells/kg). Patients then receive high-dose melphalan IV on days -3 and -2 and undergo autologous stem cell transfusion on day 0. Patients with progressive disease, stable disease, partial response, stringent complete response, or complete response are taken off study; patients with residual/persistent disease (very good partial response [VGPR]*) continue to therapeutic treatment.
NOTE: *Patients who meet the criteria for VGPR but have findings of abnormal bone marrow cytogenetic study demonstrating >= 10% metaphase cells positive for either high-risk chromosomal abnormalities or persistent disease-related chromosomal abnormalities (e.g., chromosome del 13; del17p, t(4;14), t(14;16), t(6;14) or complex cytogenetics) will have a repeat bone marrow biopsy assessment with cytogenetics within 2-3 weeks. If the repeat biopsy findings suggest persistent high-risk cytogenetic findings, then the patient is considered eligible to receive RO4929097 to eradicate residual disease.
THERAPEUTIC TREATMENT: Beginning 100-110 days after transplantation, patients receive oral RO4929097 once daily on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for at least 4 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (RO4929097 after autologous stem cell transplant) | Experimental | Procedure: Autologous Hematopoietic Stem Cell Transplantation, Drug: Gamma-Secretase Inhibitor RO4929097, Procedure: Hematopoietic Stem Cell Mobilization, Procedure: In Vitro-Treated Peripheral Blood Stem Cell Transplantation, Other: Laboratory Biomarker Analysis, Drug: MelphalanSTEM CELL TRANSPLANTATION AND CHEMOTHERAPY: Patients undergo standard mobilization and collection of autologous peripheral stem cells (>= 4.0 x 10^6 CD34+ cells/kg). Patients then receive high-dose melphalan IV on days -3 and -2 and undergo autologous stem cell transfusion on day 0. Patients with progressive disease, stable disease, partial response, stringent complete response, or complete response are taken off study; patients with residual/persistent disease (VGPR) continue to therapeutic treatment. THERAPEUTIC TREATMENT: Beginning 100-110 days after transplantation, patients receive oral gamma-secretase inhibitor RO4929097 once daily on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (54 listed)
- Patients must have a diagnosis of multiple myeloma as defined below with staging based on the International Staging System:
- Patients must have measurable disease
- For therapeutic treatment with RO4929097, patients must have peripheral blood stem cell collection of >= 4.0 x 10^6 cells/kg (patient's ideal body weight)
- ECOG performance status =< 2, Karnofsky >= 60%
- Estimated life expectancy of at least 12 weeks
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Hematopoietic Stem Cell TransplantationAlso: Autologous Stem Cell TransplantationUndergo in vitro treated autologous peripheral blood stem cell transplant | Procedure or surgery | Treatment (RO4929097 after autologous stem cell transplant) |
| Gamma-Secretase Inhibitor RO4929097Also: RO4929097Given orally | Drug | Treatment (RO4929097 after autologous stem cell transplant) |
| Hematopoietic Stem Cell MobilizationUndergo standard mobilization | Procedure or surgery | Treatment (RO4929097 after autologous stem cell transplant) |
| In Vitro-Treated Peripheral Blood Stem Cell TransplantationAlso: in vitro-treated PBPC transplantation, in vitro-treated peripheral blood progenitor cell transplantationUndergo in vitro treated autologous peripheral blood stem cell transplant | Procedure or surgery | Treatment (RO4929097 after autologous stem cell transplant) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (RO4929097 after autologous stem cell transplant) |
| MelphalanAlso: Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine MustardGiven IV | Drug | Treatment (RO4929097 after autologous stem cell transplant) |
Primary outcome
Overall response rate of gamma-secretase/Notch signalling pathway inhibitor RO4929097 in eradicating residual/persistent disease, defined as conversion from VGPR to CR or sCR
Time frame: Up to 42 days
A Simon's 2-stage optimal Phase II design will be used to monitor the study.
2 secondary outcomes
Clonogenic myeloma progenitor cells
Time frame: At baseline, at 100 days post-transplant, and at 6 weeks
Incidence of adverse events and abnormal laboratory variables as assessed according to the NCI-CTCAE version 4.0
Time frame: Up to 30 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2017.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mecide Gharibo principal investigator
Rutgers Cancer Institute of New Jersey
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous Hematopoietic Stem Cell Transplantation, Gamma-Secretase Inhibitor RO4929097 and Hematopoietic Stem Cell Mobilization in people with DS Stage I Plasma Cell Myeloma and DS Stage II Plasma Cell Myeloma, run by National Cancer Institute (NCI) 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 54 inclusion and 0 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: withdrawn, as of the sponsor's last update on 28 Sep 2017. 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 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 was withdrawn before enrolling participants, so there are no results.
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?
Overall response rate of gamma-secretase/Notch signalling pathway inhibitor RO4929097 in eradicating residual/persistent disease, defined as conversion from VGPR to CR or sCR (time frame: Up to 42 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Sep 2017; AzTrials last refreshed its copy on 8 Nov 2026.