NCT00001880

Stem Cell Transplantation for Metastatic Solid Tumors

Terminated Phase 2 Results posted Last updated 10 November 2021

What this trial tests

Phase 2 trial testing methotrexate in Neoplasm Metastasis in 42 participants. Terminated before completion.

Timeline

12 March 1999

Primary endpoint
14 August 2008

13 August 2020

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	42
Start date	12 March 1999
Primary completion	14 August 2008
Estimated completion	13 August 2020
Sites	1 location across United States

Drugs / interventions tested

• methotrexate — full drug profile →
• Cyclosporin — full drug profile →

Conditions studied

• Neoplasm Metastasis — all drugs for Neoplasm Metastasis →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 10 to 80, any sex, with Neoplasm Metastasis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants Based on Tumor Response Criteria With Anti-tumor Effect Induce by Graft-versus-tumor Effect. Primary · one year

To identify an anti-tumor effect of allogenic PNSC transplantation by induction of a graft-versus-tumor (GVT) effect in patients with a diversity of metastatic solid tumors, which are refractory to standard therapy. Tumor response assessed as follows: Complete response (CR): disappearance of all signs and symptoms of metastatic disease for a period of at least one month. Partial response (PR): a 50% or greater decrease in the sum of the products of the longest perpendicular diameters of all measured lesions lasting for a period of at least one month. No new metastatic lesions may appear. S

Group	Value	95% CI
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	0
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	9
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	5
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	28

Number of Participants That Achieved Engraftment Secondary · Day 100

Number of participants that achieved engraftment based on blood Chimerism Cluster of differentiation 3 (CD3) analysis that is greater than or equal to 95%.

Group	Value	95% CI
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	31

Number of Participants Who Received Donor Lymphocyte Infusion to Achieve Tumor Regression or Prevent Graft Failure Secondary · 2 years

To evaluate the effects of donor lymphocyte infusion (DLI) and cyclosporine A (CSA) withdrawal on tumor regression in participants who show progressive disease off of CSA and in the absence of grade \> II GVHD, or who are at risk for graft failure due to incomplete donor T-cell engraftment will receive one or more DLI. Tumor response assessed as follows: Complete response (CR): disappearance of signs \& symptoms of metastatic disease at least one month. Partial response (PR): a 50% or greater decrease in the sum of the products of the longest perpendicular diameters of all measured lesions

Group	Value	95% CI
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	16

Number of Participants Who Developed Acute GVHD Grade 2 and Higher Secondary · Day 100

Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD. Grades are defined as: Grade I: Skin = Maculopapular rash\< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day. Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Gra

Group	Value	95% CI
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	9
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	11
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	22

Number of Participants Who Developed Chronic GVHD Secondary · Day 100 to year 2

Number of participants who developed Chronic Graft vs Host Disease (GVHD). Chronic GVHD is defined as symptoms that persist or appear after 100 days since the time of stem cell transplantation.

Group	Value	95% CI
Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors	18

Adverse events — posted to ClinicalTrials.gov

Time frame: 8.5 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors

Serious: 21/42 (50%)

Deaths: 14/42

Serious adverse events (15 terms)

Reaction	System	Stem Cell Transplantation …
Progressive disease	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
Acute Graft VS Host Disease of GI Tract	Gastrointestinal disorders	—
Mucositis with fever	Gastrointestinal disorders	—
Pseudomonas sepsis	Infections and infestations	—
Parainfluenza pneumonia and septic shock	Infections and infestations	—
Pulmonary aspergillus	Infections and infestations	—
Dyspnea with progressive mets to lungs	Respiratory, thoracic and mediastinal disorders	—
Fecal impaction	Gastrointestinal disorders	—
Community acquired pneumonia	Infections and infestations	—
Pneumonia	Infections and infestations	—
Cytomegalovirus colitis	Gastrointestinal disorders	—
Invasive fungal infection	Infections and infestations	—
Acute nephrotoxicity	Renal and urinary disorders	—
Acute Respiratory Distress	Infections and infestations	—
Liver Failure	Hepatobiliary disorders	—

Other adverse events (2 terms — click to expand)

Reaction	System	Stem Cell Transplantation …
Hemorrhagic cystitis	Renal and urinary disorders	—
Cytomegalovirus disease	Infections and infestations	—

Most-reported serious reactions: Progressive disease, Acute Graft VS Host Disease of GI Tract, Mucositis with fever, Pseudomonas sepsis, Parainfluenza pneumonia and septic shock, Pulmonary aspergillus, Dyspnea with progressive mets to lungs, Fecal impaction.

Data from ClinicalTrials.gov NCT00001880 adverse events section.

Sponsor's own description

The goal of this research study is to identify other types of cancer (malignant neoplasms) that may be treatable with stem cell transplantation (allogenic peripheral blood stem cell transplantation.

Patients with a variety of different types of cancerous tumors that have spread (metastasized) and whose conditions have not improved with stand therapy, will be eligible to participate. Those patients selected to participate in the study will undergo a procedure known as a "mini-transplant". The mini-transplant is a transplantation of stem-cells collected from a sibling (brother or sister) of the patient. Unlike traditional bone marrow transplants, the mini-transplant does not require intense chemotherapy or radiation therapy. Because of this, patients experience fewer and less severe side effects.

This study is open to patients diagnosed with a variety of metastatic solid tumors including esophageal, gastric (stomach), colon, rectal, liver tumors (hepatoma), cancer of the biliary system (cholangiocarcinoma), cancer of the pancreas, lung, breast, prostate, bone (sarcoma), adrenal basal cell, bladder, and adenocarcinomas of unk primary origin.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.