Dichloroacetate Treatment of Congenital Lactic Acidosis P... | NCT01797276

Studies
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access

NCT01797276

Sponsor

University of Florida

Status

No longer available

Last Update Posted

Aug 8, 2017Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Mitochondrial Enzyme Deficiencies

Interventions

Dichloroacetate

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2017

Overall Recruitment Status or Expanded Access Status

No longer available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Feb 15, 2013

First Submission Date that Met QC Criteria

Feb 20, 2013

First Posted Date

Feb 22, 2013Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 7, 2017

Last Update Posted Date

Aug 8, 2017Actual

Description Module

Brief Summary

Expanded access to DCA as continued treatment for congenital lactic acidosis.

Detailed Description

Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day for expanded access. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated).

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Individual PatientsTreatment IND/Protocol

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
Dichloroacetate	Drug	Subjects are given DCA 25mg/kg in a divided dose twice a day.		DCA

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Diagnosis of congenital lactic acidosis
Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
Intolerance to DCA

Accepts Healthy Volunteers

Sex

Female

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

50 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Peter W. Stacpoole, PhD, MD	University of Florida	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Florida Box 100226		Gainesville	Florida	32610-0226	United States

Condition Browse Module

MeSH Terms

ID	Term
D000138	Acidosis
D035583	Rare Diseases

Ancestor Terms

ID	Term
D000137	Acid-Base Imbalance
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D020969	Disease Attributes

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D003999	Dichloroacetic Acid

Ancestor Terms

ID	Term
D062845	Chloroacetates
D000085	Acetates
D000144	Acids, Acyclic
D002264	Carboxylic Acids

Browse Leaves

Not provided

Browse Branches

Not provided