PROVe: Clinical Study Assessing Outcomes, Adverse Events, Treatment Patterns, and Quality of Life in Patients Diagnosed With Mycosis Fungoides Cutaneous T-cell Lymphoma

• Sponsor: Helsinn Therapeutics (U.S.), Inc (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02296164
• Collaborator: (none)
• Enrollment: 300
• Locations: 36
• Actual Duration (Months): 47.1
• Patients Per Site: 8.3
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The Valchlor PROVe study is a multi-center, prospective, observational, US-based drug study that longitudinally follows patients with Mycosis Fungoides Cutaneous T-cell Lymphoma (MF-CTCL) who are receiving therapy with Valchlor. Patients will be followed prospectively for a maximum of 2 years from the date of signed informed consent (enrollment) until end of study. Continuation in the study is not contingent on continuation of Valchlor.

Condition or Disease	Intervention/Treatment	Phase
Mycosis Fungoides	Drug: Valchlor

Detailed Description

This is a multi-center, prospective, observational, US-based drug study. All consecutive Mycosis Fungoides Cutaneous T-cell Lymphoma (MF-CTCL) patients being treated with Valchlor will be invited to enroll in this study.Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient completed questionnaires for symptoms and QOL, there are no specific or mandated clinical assessments to be performed. Patients will be followed prospectively for a maximum of 2 years

Study Design

• Study Type: Observational [Patient Registry]
• Actual Enrollment: 300 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: A PROspective, Observational, US-based Study Assessing Outcomes, Adverse Events, Treatment Patterns, and Quality of Life in Patients Diagnosed With Mycosis Fungoides Cutaneous T-cell Lymphoma and Treated With Valchlor®
• Actual Study Start Date: Nov 12, 2014
• Actual Primary Completion Date: Oct 17, 2018
• Actual Study Completion Date: Oct 17, 2018

Arms and Interventions

Arm	Intervention/Treatment
MF-CTCL Patients receiving Valchlor Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient completed questionnaires for symptoms and Quality Of Life.	Drug: Valchlor; Valchlor gel 0.016%; Other Names: Mechlorethamine gel

Outcome Measures

Primary Outcome Measures

1. Treatment Responders Using Body Surface Area (BSA) at 12 Months [12 Months]

   The primary efficacy endpoint was the proportion of patients who are responders to treatment at the 12-month timepoint using a ≥50% reduction from baseline in BSA as the definition of a responder in the group of patients who used mechlorethamine plus corticosteroids and possibly another treatment.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• All adult patients (≥ 18 years of age) diagnosed with MF CTCL and being treated with Valchlor. This includes patients newly initiating Valchlor OR patients continuing treatment with Valchlor:

• Patients newly initiating Valchlor are patients who have their first office visit after having initiated Valchlor.

• Patients continuing treatment with Valchlor includes patients who are actively taking Valchlor on the day of enrollment.

• Signed patient informed consent.

Exclusion Criteria:

• None

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site	Little Rock	Arkansas	United States	72205
2	Investigator Site	Duarte	California	United States	91010
3	Investigator Site	La Mesa	California	United States	91941
4	Investigator Site	San Francisco	California	United States	94115
5	Investigator Site	Santa Ana	California	United States	92701
6	Investigator Site	Aurora	Colorado	United States	80045
7	Investigator Site	New Haven	Connecticut	United States	06520
8	Investigator Site	Coral Gables	Florida	United States	33134
9	Investigator Site	Hollywood	Florida	United States	33021
10	Investigator Site	Naples	Florida	United States	34102
11	Investigator Site	Tampa	Florida	United States	33612
12	Investigator Site	Atlanta	Georgia	United States	30328
13	Investigator Site	Chicago	Illinois	United States	60611
14	Investigator Site	Chicago	Illinois	United States	60612
15	Investigator Site	Chicago	Illinois	United States	60637
16	Investigator Site	New Orleans	Louisiana	United States	70112
17	Investigator Site	Boston	Massachusetts	United States	02215
18	Investigator Site	Ann Arbor	Michigan	United States	48103
19	Investigator Site	Jackson	Mississippi	United States	39216
20	Investigator Site	Saint Louis	Missouri	United States	63104
21	Investigator Site	Saint Louis	Missouri	United States	63110
22	Investigator Site	Henderson	Nevada	United States	89074
23	Investigator Site	East Windsor	New Jersey	United States	08520
24	Investigator Site	Bronx	New York	United States	10467
25	Investigator Site	Brooklyn	New York	United States	11203
26	Investigator Site	Fairport	New York	United States	14450
27	Investigator Site	New York	New York	United States	10032
28	Investigator Site	Cleveland	Ohio	United States	44106
29	Investigator Site	Exton	Pennsylvania	United States	19341
30	Investigator Site	Philadelphia	Pennsylvania	United States	19104
31	Investigator Site	Philadelphia	Pennsylvania	United States	19107
32	Investigator Site	Pittsburgh	Pennsylvania	United States	15213
33	Investigator Site	Charleston	South Carolina	United States	29414
34	Investigator Site	Dallas	Texas	United States	75231
35	Investigator Site	Dallas	Texas	United States	75390
36	Investigator Site	Fairfax	Virginia	United States	22031

Sponsors and Collaborators

• Helsinn Therapeutics (U.S.), Inc

Investigators

• Study Director: Bill Bailey, Helsinn Therapeutics (U.S.), Inc

Study Documents (Full-Text)

• Study Protocol - Dec 13, 2016
• Statistical Analysis Plan - Nov 22, 2019

More Information

Publications

None provided.

• Responsible Party: Helsinn Therapeutics (U.S.), Inc
• ClinicalTrials.gov Identifier: NCT02296164
• Other Study ID Numbers: AC-079A501
• First Posted: Nov 20, 2014
• Last Update Posted: Mar 4, 2020
• Last Verified: Feb 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Helsinn Therapeutics (U.S.), Inc: PROVe; CTCL; Mechlorethamine; Cutaneous T-cell lymphoma; Mycosis fungoides cutaneous T-cell lymphoma; Valchlor; Dermatology; MF-CTCL; Mycosis Fungoides; Oncology
• Additional relevant MeSH terms: Mycoses; Lymphoma; Lymphoma, T-Cell; Lymphoma, T-Cell, Peripheral; Mycosis Fungoides; Lymphoma, T-Cell, Cutaneous; Mechlorethamine

Study Results

Participant Flow

Recruitment Details	The study registered 301 consecutive adult patients (Only registered/not enrolled:1; Enrolled: 300) diagnosed with MF-CTCL and being treated with Valchlor, regardless of other MF-CTCL therapies received before or at enrollment. All patients were enrolled from 46 participating centers/clinics in university-affiliated or community hospitals in the US
Pre-assignment Detail	All consecutive MF-CTCL patients being treated with Valchlor were invited to enroll in this study. Two patients were not eligible as they were not on Valchlor at time of study start

Arm/Group Title	MF-CTCL Patients Receiving Valchlor
Arm/Group Description	Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient-completed questionnaires for symptoms and QOL, there were no specific or mandated clinical assessments performed, and patients did not receive experimental intervention or treatment as a consequence of their participation in this study. Continuation in the study was not contingent on continuation of Valchlor. Valchlor: Valchlor gel 0.016%
Period Title: Overall Study
STARTED	298
COMPLETED	188
NOT COMPLETED	110

Baseline Characteristics

Arm/Group Title	MF-CTCL Patients Receiving Valchlor
Arm/Group Description	Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient completed questionnaires for symptoms and Quality Of Life. Valchlor: Valchlor gel 0.016%
Overall Participants	298
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	162 54.4%
>=65 years	136 45.6%
Sex: Female, Male (Count of Participants)
Female	119 39.9%
Male	179 60.1%
Race/Ethnicity, Customized (Count of Participants)
Caucasian	11 3.7%
African American	45 15.1%
Hispanic or Latino	29 9.7%
Asian	2 0.7%
Native Hawaiian/other Pacific Islander	6 2%
Unknown or ≥2 races/ethnicities	2 0.7%
Not Disclosed	203 68.1%
Region of Enrollment (Count of Participants)
United States	298 100%
diagnosed with MF-CTCL and being treated with Valchlor. (Count of Participants)
Count of Participants [Participants]	298 100%

Outcome Measures

1. Primary Outcome

Title	Treatment Responders Using Body Surface Area (BSA) at 12 Months
Description	The primary efficacy endpoint was the proportion of patients who are responders to treatment at the 12-month timepoint using a ≥50% reduction from baseline in BSA as the definition of a responder in the group of patients who used mechlorethamine plus corticosteroids and possibly another treatment.
Time Frame	12 Months

Outcome Measure Data

Analysis Population Description

The primary efficacy endpoint was the proportion of patients who are responders to treatment at the 12-month timepoint using a ≥50% reduction from baseline in BSA as the definition of a responder in the group of patients who used mechlorethamine plus corticosteroids and possibly another treatment.

Arm/Group Title	MF-CTCL Patients Receiving Valchlor
Arm/Group Description	Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient completed questionnaires for symptoms and Quality Of Life. Valchlor: Valchlor gel 0.016%
Measure Participants	298
Count of Participants [Participants]	44 14.8%

Adverse Events

Time Frame	The definition of an on-study event is one that occurs on the day of enrollment or later, but before the date of study discontinuation or completion ( 2 years).
Adverse Event Reporting Description	The frequency and percentage of patients experiencing any on-study Adverse Events (AEs), Serious Adverse Events (SAEs), or death. Additional detail for Adverse Events (AEs) of special interest (e.g., dermatitis). Serious Adverse Events (SAEs) both including and excluding events that were not concurrent with Valchlor exposure.
Arm/Group Title	MF-CTCL Patients Receiving Valchlor
Arm/Group Description	Patients will undergo clinical assessments and receive standard medical care, as determined by the patients' physician, in the real world setting. With the exception of protocol-required patient completed questionnaires for symptoms and Quality Of Life. Valchlor: Valchlor gel 0.016%
All Cause Mortality
	MF-CTCL Patients Receiving Valchlor
	Affected / at Risk (%)	# Events
Total	0/298 (0%)
Serious Adverse Events
	MF-CTCL Patients Receiving Valchlor
	Affected / at Risk (%)	# Events
Total	23/298 (7.7%)
Blood and lymphatic system disorders
Anaemia	1/298 (0.3%)	1
Cardiac disorders
Atrial fibrillation	1/298 (0.3%)	1
Bradycardia	1/298 (0.3%)	1
Cardiac arrest	1/298 (0.3%)	1
Cardiac failure congestive	1/298 (0.3%)	1
Gastrointestinal disorders
Abdominal pain	1/298 (0.3%)	1
General disorders
Asthenia	1/298 (0.3%)	1
Death	2/298 (0.7%)	2
Fatigue	1/298 (0.3%)	1
Multi-organ failure	1/298 (0.3%)	1
Non-cardiac chest pain	1/298 (0.3%)	1
Pyrexia	1/298 (0.3%)	1
Infections and infestations
Bacteraemia	1/298 (0.3%)	1
Bronchitis	1/298 (0.3%)	1
Clostridium difficile colitis	1/298 (0.3%)	1
Device related sepsis	1/298 (0.3%)	1
Endocarditis bacterial	1/298 (0.3%)	1
Osteomyelitis	1/298 (0.3%)	1
Pneumonia	1/298 (0.3%)	1
Sepsis	3/298 (1%)	3
Staphylococcal bacteraemia	1/298 (0.3%)	1
Injury, poisoning and procedural complications
Fall	1/298 (0.3%)	1
Pulmonary contusion	1/298 (0.3%)	1
Rib fracture	1/298 (0.3%)	1
Wrist fracture	1/298 (0.3%)	1
Investigations
Liver function test abnormal	1/298 (0.3%)	1
Metabolism and nutrition disorders
Decreased appetite	1/298 (0.3%)	1
Failure to thrive	1/298 (0.3%)	1
Musculoskeletal and connective tissue disorders
Spinal column stenosis	1/298 (0.3%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Anaplastic large cell lymphoma T- and null-cell types	1/298 (0.3%)	1
Basal cell carcinoma	1/298 (0.3%)	1
Bladder cancer	1/298 (0.3%)	1
Cholangiocarcinoma	1/298 (0.3%)	1
Mycosis fungoides	1/298 (0.3%)	1
Skin cancer with HLT skin neoplasm malignant and unspecified (exc. Melanoma)	2/298 (0.7%)	2
Nervous system disorders
Cerebrovascular accident	1/298 (0.3%)	1
Depressed level of consciousness	1/298 (0.3%)	1
Renal and urinary disorders
Renal failure acute	1/298 (0.3%)	1
Respiratory, thoracic and mediastinal disorders
Dyspnoea	1/298 (0.3%)	1
Hypoxia	1/298 (0.3%)	1
Pleural effusion	1/298 (0.3%)	1
Pneumonia aspiration	1/298 (0.3%)	1
Pneumothorax	1/298 (0.3%)	1
Skin and subcutaneous tissue disorders
Skin ulcer	2/298 (0.7%)	2
Surgical and medical procedures
Packed red blood cell transfusion	1/298 (0.3%)	1
Vascular disorders
Aortic aneurysm	1/298 (0.3%)	1
Deep vein thrombosis	1/298 (0.3%)	1
Haematoma	1/298 (0.3%)	1
Jugular vein thrombosis	1/298 (0.3%)	1
Venous thrombosis limb	1/298 (0.3%)	1
Other (Not Including Serious) Adverse Events
	MF-CTCL Patients Receiving Valchlor
	Affected / at Risk (%)	# Events
Total	104/298 (34.9%)
Skin and subcutaneous tissue disorders
Dermatitis	38/298 (12.8%)	38
Erythema	15/298 (5%)	15
Pruritus	29/298 (9.7%)	29
Skin irritation	22/298 (7.4%)	22

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.