High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy

NCT01236456 | Withdrawn Phase 2

• 1 other identifier: 65865
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The primary endpoint of this study is to determine what percentage of patients receiving high-dose Cyclophosphamide may experience a halt in the worsening of their disease or experience improvement of their disease and for how long the benefit may last.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy

Keywords

chronic inflammatory demyelinating polyneuropathy; cyclophosphamide; autoimmune

Outcome Measures

Primary Outcomes (1)

• The primary endpoint of this study is to evaluate the response rate of CIDP patients as determined by functional score, change in Summated compound motor action potential and strength, after high-dose cyclophosphamide therapy.

Secondary Outcomes (1)

• The secondary endpoint of this study is to determine remission duration.

Interventions

Cyclophosphamide DRUG

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of CIDP according to the American Academy of Neurology clinical and electrophysiologic criteria
• Age \>18 but \< 75 years
• Modified Rankin Scale score of \>3 after two standard treatment regimens
• Patient must have a left ventricular ejection fraction of \>45%
• Serum Creatinine \<3mg/dL
• Willingness to participate in a clinical trial

You may not qualify if:

• Patients who are preterminal or moribund
• Patients with active malignancies
• Patients with chromosomal abnormalities or peripheral blood counts suggestive of myelodysplastic syndrome
• Patients with active bacterial or fungal infections requiring oral or intravenous antimicrobials are not eligible until resolution of the infection
• Pregnant women and breast-feeding women

Sponsors & Collaborators

• Stony Brook University: lead

Related Publications (2)

• Gladstone DE, Prestrud AA, Brannagan TH 3rd. High-dose cyclophosphamide results in long-term disease remission with restoration of a normal quality of life in patients with severe refractory chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2005 Mar;10(1):11-6. doi: 10.1111/j.1085-9489.2005.10104.x.

  PMID: 15703014 RESULT

• Brannagan TH 3rd, Pradhan A, Heiman-Patterson T, Winkelman AC, Styler MJ, Topolsky DL, Crilley PA, Schwartzman RJ, Brodsky I, Gladstone DE. High-dose cyclophosphamide without stem-cell rescue for refractory CIDP. Neurology. 2002 Jun 25;58(12):1856-8. doi: 10.1212/wnl.58.12.1856.

  PMID: 12084892 RESULT

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Interventions

Cyclophosphamide

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 5, 2010
• First Posted: November 8, 2010
• Study Start: October 1, 2003
• Study Completion: November 1, 2006
• Last Updated: November 9, 2021

Record last verified: 2021-11