Double-blind, placebo-controlled, parallel group, phase III study comparing dichlorphenamide vs. placebo for the treatment of periodic paralysis

Phase 1

• Conditions: Patients with hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis.; MedDRA version: 12.1 Level: LLT Classification code 10016208 Term: Familial periodic paralysis

• Registration Number: EUCTR2008-002306-19-GB
• Lead Sponsor: niversity of Rochester

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2010-03-15
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 140

Inclusion Criteria

Inclusion criteria for Hyperkalemic Periodic Paralysis (HYP):
1.Genetically definite, clinically definite or clinically probable HYP.
2.Male and female participants, age 18 and older who are able to comply with the study procedures.
3.Participants will be required to have distinct regular episodes of weakness by history with an average frequency of >1 /week and <3/day either on or off treatment, whichever is higher.
4.Normal TSH and T4.
5.Pregnancy: Women: non-childbearing potential (i.e., postmenopausal or surgically sterile) or must meet all of the following conditions:
Use a medically accepted contraceptive regimen for at least 30 days before the baseline visit, and agree to continue such use throughout the duration of the study and for 30 days after the final dose of study drug.
Reliable forms of contraception include oral, implanted, or injected contraceptives; intrauterine devices in place for at least 3 months; or adequate barrier methods in conjunction with spermicide (abstinence is considered an acceptable contraceptive regimen).
Women must be given a pregnancy test unless they are at least 2 years postmenopausal or surgically sterile.

Inclusion criteria for Hypokalemic Periodic Paralysis (HOP):
1.Genetically definite, clinically definite or clinically probable HOP.
2.Male and female participants, age 18 and older who are able to comply with the study procedures.
3.Participants will be required to have distinct regular episodes of weakness by history with an average frequency of >1 /week and <3/day either on or off treatment, whichever is higher.
4.Normal TSH and T4.
5.Pregnancy: Women: nonchildbearing potential (i.e., postmenopausal or surgically sterile) or must meet all of the following conditions:
Use a medically accepted contraceptive regimen for at least 30 days before the baseline visit, and agree to continue such use throughout the duration of the study and for 30 days after the final dose of study drug.
Reliable forms of contraception include oral, implanted, or injected contraceptives; intrauterine devices in place for at least 3 months; or adequate barrier methods in conjunction with spermicide (abstinence is considered an acceptable contraceptive regimen).
Women must be given a pregnancy test unless they are at least 2 years postmenopausal or surgically sterile.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria for Hyperkalemic Periodic Paralysis:
None of the following can be present:

1.Evidence for Andersen-Tawil syndrome (any one of the following 3 criteria)
•Prolonged QT interval or complex ventricular ectopy between attacks
•KIR 2.1 gene mutation
•Distinctive physical features (2 out of 5)
oLow set ears
oShort stature
oHypo-/micro-gnathia
oClinodactyly
oHypo-/hypertelorism
2.Coincidental renal, hepatic, restrictive or obstructive lung disease, active thyroid, or heart disease
3.Chronic, non-congestive, angle-closure glaucoma
4.Use of any of the following medications for reasons other than treatment of periodic paralysis: diuretics, antiarrhythmics, corticosteroids, beta-blockers, calcium channel blockers, antiepileptics, magnesium
5.History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks (prior to treatment)
6.History of worsening symptoms with the use of CAI’s
7.Any other neuromuscular disease

Exclusion criteria for Hypokalemic Periodic Paralysis:
None of the following can be present:

1. Known mutation in the a subunit of sodium channel.

2. Evidence for Andersen-Tawil syndrome (any one of the following 3 criteria)
•Prolonged QT interval or complex ventricular ectopy between attacks
•Distinctive physical features (2 out of 5)
oLow set ears
oShort stature
oHypo-/micrognathia
oClinodactyly
oHypo-/hypertelorism
•KIR 2.1 gene mutation

3.Coincidental renal, hepatic, active thyroid disease, restrictive or obstructive lung disease, or heart disease
4.Chronic, non-congestive, angle-closure glaucoma
5.Use of any of the following medications for reasons other than treatment of periodic paralysis: diuretics, antiarrhythmics, corticosteroids, beta-blockers, calcium channel blockers, antiepileptics, magnesium
6.History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks (prior to treatment)
7.History of worsening symptoms with the use of CAI’s
8. Any other neuromuscular disease

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified