Trial Outcomes & Findings for Creatine Safety & Tolerability in Huntington's Disease (NCT NCT01412151)

NCT ID: NCT01412151

Last Updated: 2018-03-09

Results Overview

Proportion of subjects able to complete treatment

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: 306 Weeks
• Results posted on: 2018-03-09

Participant Flow

Participant milestones

Measure	Creatine Monohydrate Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Week 0 - 306 - Treatment Period STARTED	10
Week 0 - 306 - Treatment Period COMPLETED	5
Week 0 - 306 - Treatment Period NOT COMPLETED	5
Week 306 - 310 - Washout Period STARTED	5
Week 306 - 310 - Washout Period COMPLETED	3
Week 306 - 310 - Washout Period NOT COMPLETED	2

Reasons for withdrawal

Measure	Creatine Monohydrate Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Week 0 - 306 - Treatment Period Death	1
Week 0 - 306 - Treatment Period Withdrawal by Subject	1
Week 0 - 306 - Treatment Period Withdrawal by Investigator	3
Week 306 - 310 - Washout Period Withdrawal by Subject	2

Baseline Characteristics

Creatine Safety & Tolerability in Huntington's Disease

Baseline characteristics by cohort

Measure	Creatine Monohydrate n=10 Participants Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	48.2 years STANDARD_DEVIATION 8.4 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 306 Weeks

Proportion of subjects able to complete treatment

Outcome measures

Measure	Creatine Monohydrate n=10 Participants Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Tolerability	5 Participants

SECONDARY outcome

Timeframe: 310 Weeks

Population: data was not collected or analyzed for this outcome measure.

Components of the UHDRS (Unified Huntington Disease Rating Scale)

data was not collected or analyzed for this outcome measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 310 Weeks

Population: data was not collected or analyzed for this outcome measure.

Biological indicators that creatine treatment might affect the progression of HD: serum creatine levels, neuroimaging, metabolomic and gene expression analysis

Outcome measures

Outcome data not reported

Adverse Events

Creatine Monohydrate

Serious events: 4 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Measure	Creatine Monohydrate n=10 participants at risk Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Psychiatric disorders Agitation	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Infections and infestations Cellulitis	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Psychiatric disorders Depression with Suicidal Ideation	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Infections and infestations Pneumonia	20.0% 2/10 • Number of events 2 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Respiratory, thoracic and mediastinal disorders Pneumonia Aspiration	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Infections and infestations Sepsis	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Infections and infestations Skin Infection	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Gastrointestinal disorders Small Intestinal Obstruction	10.0% 1/10 • Number of events 1 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.

Other adverse events

Measure	Creatine Monohydrate n=10 participants at risk Twice daily with a meal mixed in a liquid for a total daily dosage of 30 grams. Daily dosage adjustments (e.g. reductions, suspensions, rechallenges) were allowed to manage adverse events.
Psychiatric disorders Agitation	50.0% 5/10 • Number of events 9 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Injury, poisoning and procedural complications Fall	70.0% 7/10 • Number of events 9 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Gastrointestinal disorders Diarrhea	50.0% 5/10 • Number of events 7 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.
Infections and infestations Infection	10.0% 1/10 • Number of events 5 • 310 Weeks Adverse events were assessed clinically (symptoms reported by the subject or signs detected on examination/through ancillary testing - vital signs, EKG, laboratory tests etc). Stable chronic conditions (e.g. diabetes, arthritis) present prior to the start of the study that did not worsen during the trial were not considered adverse events.

Additional Information

Steven M. Hersch, MD, PhD

Massachusetts General Hospital

Phone: 617-726-1254

Email: hersch@helix.mgh.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place