MDACC Study Summary 2006-0780

Study Summary
No. 2006-0780:.......Blood And Marrow Transplantation......Partow Kebriaei......Stem Cell Transplantation and Cellular Therapy

Study Summary Title



Study Summary Number:	2006-0780

Study Title:	A Phase III Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

Physician

New Patient Referral



Name:	Partow Kebriaei	Patients Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Dept:	Stem Cell Transplantation and Cellular Therapy	Referring MD Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Phone:	713-745-0663 Contact us about clinical trials

General Information



Disease Group:	Blood And Marrow Transplantation	Supported By:	N/A

Phase of Study:	Phase III	Return Visit:	Patients will be assessed weekly until day 63, and then on days 100 (+/- 3), 130 (+/- 7), 160 (+/- 7), and then at study completion on day 180 (+/- 7) from time of first infusion of investigational agent.

Treatment Agents:	Prochymal	Home Care:	Intravenous fluids, antibiotics may be necessary through home care agencies.

Treatment Loc:	Independent Multicenter Arrangements

Estimated Length of Stay in Houston:	Depends on how quickly the patient's GVHD symptoms improve; may range from 1 to several weeks.


Description/ Intervention:	The goal of this clinical research study is to learn if infusing Prochymal in patients with acute GVHD can help to control the disease. The safety of Prochymal will also be studied.

Study Objectives / Outcomes

1. To evaluate the efficacy of Prochymal™ in subjects with Grades B-D acute graft versus host disease (GVHD), who have failed to respond to steroid treatment.
2. To gather additional information on the safety of Prochymal™ in subjects with Grades B-D acute GVHD, who have failed to respond to steroid treatment.

Study Status Information



Study Activation / Registration Date:

IRB Review and Approval Date:	10/18/2006

Study Type:	Phase Iii

Recruitment Status:	Closed

Projected Accrual:	240

Enrollment Eligibility

If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:

1) Subjects must be 6 months to 70 years of age inclusive.

2) Subjects who have failed to respond to steroid treatment as defined by any Grade B-D (IBMTR grading) of acute GVHD that shows a) no improvement after 3 days and b) a duration of no greater than 2 weeks while receiving treatment with methylprednisone (greater than or equal to 1mg/kg/d) or equivalent.

3) Subjects must be able to be treated within 4 days of randomization. In urgent situations 2nd line therapy may be started 24 hours prior to randomization, and Prochymal must be initiated within the following 3 days. Subjects who may have received an increase in their steroid dose treatment prior to randomization will be eligible for enrollment. An increase in steroid dose will not be considered as second line therapy.

4) Subjects must have adequate renal function as defined by: Calculation for Adults: Calculated creatinine clearance (CrCl) of >30 mL/min using the Cockroft-Gault equation: Calculated Cr Cl= (140-age in years) x (weight in kg)/ 72 x (serum creatinine value in mg/dL). Calculation for Pediatrics: Schwartz equation: CrCl (ml/min) = [length (cm) x k]/Scr (Patient population:infants over 1 week old through adolescence (<18 years old) k=0.45 for infants 1 to 52 weeks old k=0.55 for childrn 1 to 13 years old k=0.55 for adolescent females 13-18 years old k=0.7 for adolescent males 13-18 years old

5) Subjects who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male subjects must use adequate contraception.

6) Subjects must have a minimum Karnofsky/Lansky Performance Level of at least 30 at the time of study entry.

7) Subject (or legal representative where appropriate) must be capable of providing written informed consent.

8) Subjects with persistent GI GVHD manifested by diarrhea with stool volume <500mL/day in the absence of nausea or vomiting may be deemed as having grade B GVHD if other causes of diarrhea have been ruled out (e.g., C. difficile or CMV infection, oral magnesium administration) and if the low stool volume reflects the effects of fasting or administration of narcotics or anti-diarrheal medications.

Exclusion Criteria:

1) Subject has started treatment with second line therapy greater than 24 hours prior to randomization.

2) Subject has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the subject, including uncontrolled infection, heart failure, pulmonary hypertension, etc.

3) Subjects may not receive any other investigational agents (not approved by the FDA for any indication) concurrently during study participation or within 30 days of randomization.

4) Subject has a known allergy to bovine or porcine products.

5) Subject has received a transplant for a solid tumor disease.

6) Subject has received agents other than steroids for primary treatment of acute GVHD.

7) Subject is participating in the CTN Protocol 0302.

8) Subject has evidence of a pulmonary infiltrate or diffuse alveolar hemorrhage and is likely to require more than 2L of oxygen via face mask or an estimated FiO2 of 28% via other delivery methods in order to sustain an O2 saturation of 92% during the next 3 days.

Links

Registration Number: NCT00366145
Study Information on Clinical Trials Registry (clinicaltrials.gov)