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Trial record 22 of 42 for:    Microcephaly

Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome

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ClinicalTrials.gov Identifier: NCT00114634
Recruitment Status : Completed
First Posted : June 16, 2005
Results First Posted : April 9, 2014
Last Update Posted : January 29, 2016
Sponsor:
Information provided by (Responsible Party):
Forbes Porter, M.D., National Institutes of Health Clinical Center (CC)

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition: Smith-Lemli-Opitz Syndrome
Interventions: Dietary Supplement: Egg yolk preparation with cholesterol
Dietary Supplement: Egg substitute, without cholesterol

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
October to July 2008

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
no exclusions

Reporting Groups
  Description
Egg Yolk or no Egg Yolk This was a double-blind, placebo-controlled, cross-over design. The trial was composed of five 2-week phases. Two phases, phases 2 and 4, were double-blinded. During these phases, subjects were either on placebo (egg substitute with no cholesterol) or cholesterol supplementation (pasteurized egg yolk). During the interval phases, 1, 3 and 5, subjects were maintained on baseline therapy of 150 mg/kg/day of crystalline cholesterol suspended in Ora-Plus. Order of placebo versus cholesterol was random with 6 subjects receiving placebo first (in phase 2) and 4 subjects receiving cholesterol containing pasteurized egg yolk first. 13 subjects were enrolled, only 10 subjects completed the study.

Participant Flow:   Overall Study
    Egg Yolk or no Egg Yolk
STARTED   13 
COMPLETED   10 [1] 
NOT COMPLETED   3 
failure to return forms                3 
[1] Assessment forms lost by one failure to complete and return forms by two



  Baseline Characteristics

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
Egg Yolk or no Egg Yolk All subjects were on cholesterol suspension or egg yolk versus no egg yolk.

Baseline Measures
   Egg Yolk or no Egg Yolk 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age 
[Units: Participants]
 
<=18 years   9 
Between 18 and 65 years   1 
>=65 years   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 11.4  (4.9) 
Gender 
[Units: Participants]
 
Female   6 
Male   4 
Region of Enrollment 
[Units: Participants]
 
United States   10 
Severity score [1] 
[Units: Units on a scale]
Median (Full Range)
 17 
 (6 to 28) 
[1] Severity score ranges from 0-100; the higher the score the greater the severity


  Outcome Measures

1.  Primary:   Hyperactivity Sub-scale of the Aberrant Behavior Checklist-Community (ABC-C).   [ Time Frame: 2 weeks ]

2.  Secondary:   ABC Irritability Sub-scale   [ Time Frame: 2 weeks ]

3.  Secondary:   ABC Lethargy Sub-scale   [ Time Frame: 2 weeks ]

4.  Secondary:   ABC Stereotypy Sub-scale   [ Time Frame: 2 weeks ]

5.  Secondary:   ABC Inappropriate Behavior Sub-scale   [ Time Frame: 2 weeks ]

6.  Secondary:   ABC Total Score   [ Time Frame: 2 weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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
Poor power due to low enrollment


  More Information

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


Results Point of Contact:  
Name/Title: Forbes D Porter
Organization: NICHD, NIH
phone: 301-435-4432
e-mail: fdporter@mail.nih.gov


Publications:

Responsible Party: Forbes Porter, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00114634     History of Changes
Other Study ID Numbers: 050168
05-CH-0168 ( Other Identifier: NIH CC )
First Submitted: June 15, 2005
First Posted: June 16, 2005
Results First Submitted: March 19, 2010
Results First Posted: April 9, 2014
Last Update Posted: January 29, 2016