Hydroxychloroquine for the First Thrombosis Prevention in Antiphospholipid Antibody Positive Patients

• Sponsor: Hospital for Special Surgery, New York (Other)
• Overall Status: Terminated
• CT.gov ID: NCT01784523
• Collaborator: AntiPhospholipid Syndrome Alliance For Clinical Trials and InternatiOnal Networking (Other), The New York Community Trust (Other)
• Enrollment: 11
• Location: 1
• Arms: 2
• Duration (Months): 31.9
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

In this multi-center international study, our aim is to determine the effectiveness of HCQ for primary thrombosis prophylaxis in persistently aPL-positive but thrombosis-free patients without systemic autoimmune diseases.

Condition or Disease	Intervention/Treatment	Phase
Antiphospholipid Syndrome	Drug: Hydroxychloroquine	Phase 3

Detailed Description

Randomized to receive HCQ or no treatment in addition to their standard regimen. 11 study visits and 10 phone visits over 5 years.

This study was terminated at 2 years due to low recruitment rate exacerbated by manufacturing shortage and price increase of hydroxychloroquine.

Study Design

• Study Type: Interventional
• Actual Enrollment: 11 participants
• Allocation: Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: A Prospective Randomized Controlled Trial of Hydroxychloroquine in the Primary Thrombosis Prophylaxis of aPL Positive But Thrombosis-free Patients.
• Study Start Date: Feb 1, 2013
• Actual Primary Completion Date: Sep 30, 2015
• Actual Study Completion Date: Sep 30, 2015

Arms and Interventions

Arm	Intervention/Treatment
No Intervention: Standard treatment patients randomized to standard treatment will not receive hydroxychloroquine.
Experimental: Hydroxychloroquine Patients will be randomized to receive standard of care or standard of care + hydroxychloroquine. Dose will be weight-adjusted: 200 mg daily for patients weighing <60kg; and 400 mg daily (200 mg twice a day)for patients weighing >60kg.	Drug: Hydroxychloroquine; Other Names: plaquenil

Outcome Measures

Primary Outcome Measures

1. Number of Participants With an Acute Thrombosis Event [2 years]

   To determine the efficacy of Hydroxychloroquine in the primary thrombosis prevention of persistently aPL-positive but thrombosis free patients with no other systemic autoimmune diseases over the five year study period.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 60 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Persistent(at least 12 weeks apart)aPL-positivity within 12 months prior to the screening defined as:

• aCL IgG/M (>40U,medium-to-high titer,and/or greater than the 99th percentile)and/or

• aβ2GPI IgG/M(>40U, medium-to-high titer, and/or greater than the 99th percentile)and/or

• Positive LA test based on the International Society of Thrombosis & Haematosis Recommendations

Selected Exclusion Criteria:

• History of thrombosis (arterial, venous, and/or biopsy proven microthrombosis

• History of Transient Ischemic Attack Confirmed by a Neurologist

• SLE Diagnosis based on the ACR Classification Criteria > 4/11

• Other Systemic Autoimmune Diseases diagnosed based on ACR Classification Criteria

• Current Hydroxychloroquine or another antimalarial treatment (-3 months)

• Current warfarin treatment (-3 months)

• Current heparin therapy( -3 months)

• Current pregnancy

• History of Hydroxychloroquine eye toxicity

• History of Hydroxychloroquine allergy

• Known glucose-6-phosphate dehydrogenase deficiency

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hospital for Special Surgery	New York	New York	United States	10021

Sponsors and Collaborators

• Hospital for Special Surgery, New York
• AntiPhospholipid Syndrome Alliance For Clinical Trials and InternatiOnal Networking
• The New York Community Trust

Investigators

• Principal Investigator: Doruk Erkan, MD, Hospital for Special Surgery, New York

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• APS ACTION

Publications

• Erkan D, Derksen R, Levy R, Machin S, Ortel T, Pierangeli S, Roubey R, Lockshin M. Antiphospholipid Syndrome Clinical Research Task Force report. Lupus. 2011 Feb;20(2):219-24. doi: 10.1177/0961203310395053.
• Erkan D, Lockshin MD; APS ACTION members. APS ACTION--AntiPhospholipid Syndrome Alliance For Clinical Trials and InternatiOnal Networking. Lupus. 2012 Jun;21(7):695-8. doi: 10.1177/0961203312437810.

• Responsible Party: Hospital for Special Surgery, New York
• ClinicalTrials.gov Identifier: NCT01784523
• Other Study ID Numbers: 2014-253; NCT02635126
• First Posted: Feb 6, 2013
• Last Update Posted: Sep 14, 2021
• Last Verified: Aug 1, 2021

• Keywords provided by Hospital for Special Surgery, New York: antiphospholipid autoantibodies; antiphospholipid positive
• Additional relevant MeSH terms: Thrombosis; Antiphospholipid Syndrome; Hydroxychloroquine

Study Results

Participant Flow

Arm/Group Title	Standard Treatment	Hydroxychloroquine
Arm/Group Description	patients randomized to standard treatment will not receive hydroxychloroquine.	Patients will be randomized to receive standard of care or standard of care + hydroxychloroquine. Dose will be weight-adjusted: 200 mg daily for patients weighing <60kg; and 400 mg daily (200 mg twice a day)for patients weighing >60kg. Hydroxychloroquine
Period Title: Overall Study
STARTED	7	4
COMPLETED	0	0
NOT COMPLETED	7	4

Baseline Characteristics

Arm/Group Title	Standard Treatment	Hydroxychloroquine	Total
Arm/Group Description	patients randomized to standard treatment will not receive hydroxychloroquine.	Patients will be randomized to receive standard of care or standard of care + hydroxychloroquine. Dose will be weight-adjusted: 200 mg daily for patients weighing <60kg; and 400 mg daily (200 mg twice a day)for patients weighing >60kg. Hydroxychloroquine	Total of all reporting groups
Overall Participants	7	4	11
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	7 100%	4 100%	11 100%
>=65 years	0 0%	0 0%	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	44 (8.98)	45.75 (7.89)	44.64 (8.24)
Sex: Female, Male (Count of Participants)
Female	7 100%	3 75%	10 90.9%
Male	0 0%	1 25%	1 9.1%
Region of Enrollment (Count of Participants)
United States	7 100%	4 100%	11 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With an Acute Thrombosis Event
Description	To determine the efficacy of Hydroxychloroquine in the primary thrombosis prevention of persistently aPL-positive but thrombosis free patients with no other systemic autoimmune diseases over the five year study period.
Time Frame	2 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Standard Treatment	Hydroxychloroquine
Arm/Group Description	patients randomized to standard treatment will not receive hydroxychloroquine.	Patients will be randomized to receive standard of care or standard of care + hydroxychloroquine. Dose will be weight-adjusted: 200 mg daily for patients weighing <60kg; and 400 mg daily (200 mg twice a day)for patients weighing >60kg. Hydroxychloroquine
Measure Participants	7	4
Count of Participants [Participants]	0 0%	0 0%

Adverse Events

Arm/Group Title	Standard Treatment		Hydroxychloroquine
Arm/Group Description	patients randomized to standard treatment will not receive hydroxychloroquine.		Patients will be randomized to receive standard of care or standard of care + hydroxychloroquine. Dose will be weight-adjusted: 200 mg daily for patients weighing <60kg; and 400 mg daily (200 mg twice a day)for patients weighing >60kg. Hydroxychloroquine
All Cause Mortality
	Standard Treatment		Hydroxychloroquine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/7 (0%)		0/4 (0%)
Serious Adverse Events
	Standard Treatment		Hydroxychloroquine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/7 (0%)		0/4 (0%)
Other (Not Including Serious) Adverse Events
	Standard Treatment		Hydroxychloroquine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/7 (0%)		0/4 (0%)

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.