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Hyper- and Hypokalemic Periodic Paralysis Study (HYP-HOP)

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Robert Griggs, MD, University of Rochester
ClinicalTrials.gov Identifier:
NCT00494507
First received: June 27, 2007
Last updated: June 5, 2014
Last verified: June 2014
Results First Received: April 30, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Conditions: Hyperkalemic Periodic Paralysis
Hypokalemic Periodic Paralysis
Interventions: Drug: Dichlorphenamide (double-blind)
Drug: Placebo (double-blind)
Drug: Dichlorphenamide (open-label)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The original trial design included an Acetazolamide (ACZ) drug arm which was subsequently removed. Five participants randomized to ACZ and one ineligible participant are not included in the trial results reported here.

Reporting Groups
  Description
HYP Dichlorphenamide

Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HYP Placebo

Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HOP Dichlorphenamide

Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HOP Placebo

Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day


Participant Flow for 2 periods

Period 1:   Double-Blind Phase
    HYP Dichlorphenamide     HYP Placebo     HOP Dichlorphenamide     HOP Placebo  
STARTED     12     9     24     20  
Reached Endpoint of Acute Worsening     0     2     0     5  
COMPLETED     9     9     22     19  
NOT COMPLETED     3     0     2     1  
Adverse Event                 2                 0                 1                 0  
Withdrawal by Subject                 1                 0                 0                 0  
Subject Non-compliance                 0                 0                 1                 0  
Negative DNA test                 0                 0                 0                 1  

Period 2:   Open-Label Phase
    HYP Dichlorphenamide     HYP Placebo     HOP Dichlorphenamide     HOP Placebo  
STARTED     9     8 [1]   22     19  
COMPLETED     9     7     17     14  
NOT COMPLETED     0     1     5     5  
Adverse Event                 0                 1                 5                 3  
Worsening Disease                 0                 0                 0                 2  
[1] 1 subject randomized to Acetazolamide in this phase is not included-drug arm was removed from trial.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
HYP Dichlorphenamide

Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HYP Placebo

Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HOP Dichlorphenamide

Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

HOP Placebo

Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.

Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet

Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day

Total Total of all reporting groups

Baseline Measures
    HYP Dichlorphenamide     HYP Placebo     HOP Dichlorphenamide     HOP Placebo     Total  
Number of Participants  
[units: participants]
  12     9     24     20     65  
Age  
[units: years]
Mean ± Standard Deviation
  40.6  ± 10.3     45.2  ± 17.7     44.8  ± 14.6     44.0  ± 15.6     44.2  ± 14.3  
Gender  
[units: participants]
         
Female     6     6     8     4     24  
Male     6     3     16     16     41  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   HYP Attack Rate   [ Time Frame: 8 weeks ]

2.  Primary:   HOP Attack Rate   [ Time Frame: 8 weeks ]

3.  Secondary:   HYP Severity-weighted Attack Rate   [ Time Frame: 8 weeks ]

4.  Secondary:   HOP Severity-weighted Attack Rate   [ Time Frame: 8 weeks ]

5.  Secondary:   HYP Attack Duration   [ Time Frame: 8 weeks ]

6.  Secondary:   HOP Attack Duration   [ Time Frame: 8 weeks ]

7.  Secondary:   HYP Endpoint of Acute Worsening   [ Time Frame: 0-9 weeks ]

8.  Secondary:   HOP Endpoint of Acute Worsening   [ Time Frame: 0-9 weeks ]

9.  Secondary:   HYP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

10.  Secondary:   HOP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

11.  Secondary:   HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

12.  Secondary:   HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

13.  Secondary:   HYP Change From Baseline to Week 9 in Lean Body Mass   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

14.  Secondary:   HOP Change From Baseline to Week 9 in Lean Body Mass   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

15.  Secondary:   HYP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

16.  Secondary:   HOP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

17.  Secondary:   HYP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No

18.  Secondary:   HOP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score   [ Time Frame: Baseline and 9 weeks ]
Results not yet reported.   Anticipated Reporting Date:   06/2014   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Sample sizes in the trial were limited by slow recruitment and the trial was concluded before attainment of the target numbers of subjects. Statistical analyses were conducted using smaller group sizes than planned, particularly for HYP subjects.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Robert Griggs, MD
Organization: University of Rochester
phone: 585-275-3707
e-mail: robert_griggs@urmc.rochester.edu


No publications provided


Responsible Party: Robert Griggs, MD, University of Rochester
ClinicalTrials.gov Identifier: NCT00494507     History of Changes
Other Study ID Numbers: R01NS045686-02, CRC
Study First Received: June 27, 2007
Results First Received: April 30, 2014
Last Updated: June 5, 2014
Health Authority: United States: Food and Drug Administration