Barth SyndromePhase 2/3TAZPOWERNCT03098797
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
A Phase 2/3 study testing Elamipretide in people with Barth Syndrome, run by Stealth BioTherapeutics Inc. at 1 site in United States.
Trial summary
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Barth Syndrome
- What is being tested?
- Elamipretide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jul 2017to 11 Oct 2021
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed Barth Syndrome
- Male aged 12 and above
- At the screening visit, eGFR must meet the following:
- Body weight >30 kg AND eGFR ≥ 90mL/min at screening
- Body weight >40kg AND eGFR ≥60 but <90mL/min/ 1.73m² at screening
- Ambulatory and impaired during the baseline 6MWT
- On stable medication for 30 days prior to the baseline visit
Who cannot join
- Participated in another interventional clinical trial within 30 days of or is currently enrolled in a non-interventional clinical trial at the baseline visit potentially confounding with this trial
- Prior or current medical condition that would prevent the subject from safely participating in the trial
- Undergone any inpatient hospitalizations within 30 days of the baseline visit
- Is undergoing an apparent pubertal growth spurt
- Has uncontrolled hypertension
- History of substance abused within the year before the baseline visit or is likely to be uncompliant
Show all 11Show fewer
- History of heart transplantation or current placement on the waiting list for a heart transplant
- For subjects with an ICD: known occurrence of ICD discharge in the 3 months prior to the baseline visit
- For subjects without an ICD: expected to undergo an implantation of an ICD during the conduct of the study
- Currently receiving treatment with chemotherapeutic agents or immunosuppressant agents or has received prior radiation therapy to the chest
- Recipient of stem cell or gene therapy or is currently being treated by a therapeutic investigational device
Trial locations and contacts
1 site in 1 country.
United States1 site
McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine
Baltimore, Maryland, 21287
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety and Efficacy of Subcutaneous Injections of Elamipretide in Subjects With Genetically Confirmed Barth Syndrome Followed by Open-Label Treatment
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.
Read the detailed description
A phase 2 randomized, double-blind, placebo-controlled crossover trial to evaluate the safety, tolerability, and efficacy of subcutaneous injections of elamipretide in subjects with genetically confirmed Barth syndrome followed by open-label treatment extension. Part 1 was a randomized, double-blind, placebo-controlled, crossover trial to assess safety, tolerability, and efficacy single daily subcutaneous (SC) doses of 40 mg elamipretide administered for 12 weeks in subjects with Barth syndrome. Part 2: This was an open-label extension trial to assess the long-term safety, tolerability, and longitudinal trends in efficacy single daily SC doses of 40 mg elamipretide for up to 192 weeks.
- Conditions
- Barth Syndrome
- Being tested
- Elamipretide, Placebo
- Keywords
- Barth Syndrome, elamipretide, MTP-131, Stealth, Stealth BT, BTHS
- Medical subject headings
- Barth Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElamipretideDrug
Also called MTP-131
40 mg daily subcutaneous injection for 12 weeks
PlaceboDrug
Also called Placebo Comparator
daily subcutaneous injection for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Elamipretide, then Placebo | Experimental | Experimental Part 1: 12 weeks of single daily SC doses of 40 mg elamipretide in Treatment Period 1 followed by 12 weeks of treatment with placebo in Treatment Period 2 (separated by a 4 week washout period).
Experimental Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks Receives: Drug: Elamipretide; Drug: Placebo |
| Placebo , then Elamipretide | Placebo comparator | Placebo Comparator Part 1: 12 weeks of single daily SC doses of placebo in Treatment Period 1 followed by 12 weeks of treatment with 40 mg elamipretide in Treatment Period 2 (separated by a 4 week washout period).
Placebo Comparator Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks Receives: Drug: Elamipretide; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit
Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Measured: Pre-dose to Week 12 (end of treatment)
Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.
Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12)
Measured: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)
Secondary outcomes (12)
Part 2: Distance Walked During the 6MWT
Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit
Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Part 1: Muscle Strength as Measured by HHD by Visit
Muscle strength as measured by handheld dynamometry (HHD) in Newtons, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Handheld dynamometry assesses the average strength of the knee extensors of both legs.
Measured: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit
Muscle strength as measured by handheld dynamometry (HHD) in Newtons. Handheld dynamometry assesses the average strength of the knee extensors of both legs. Change from baseline where Baseline= Part 1 Period 1 Pre-dose: the greater positive change, the better the outcome, the smaller positive change (or negative change) the number the worse the outcome.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Part 1: 5XSST by Visit
Time (in seconds) to complete the Five Times Sit-To-Stand, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Measured: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: 5X Sit to Stand by Visit
Change in baseline in time (in seconds), where Baseline= Part 1 Period 1 Pre-dose to complete the Five Times Sit-To-Stand by visit. More time means worse outcome, less time means better outcome.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Part 1: SWAY Application Balance Assessments by Visit
SWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability from Pre-dose to Week 12 (end of treatment), where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Measured: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: SWAY Application Balance Assessment
Change from Baseline: SWAY Application Balance Assessment,where Baseline= Part 1 Period 1 Pre-doseSWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability. An increase from baseline is a better outcome and a decrease in score is a worse outcome.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Part 1: Patient Global Impression Scales of Symptoms
Patient-reported health status over the past week, by visit, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 0=None, 1=Mild, 2=Moderate, 3=Severe, and 4=Very Severe. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Measured: Pre-dose, Week 1, Week 12 (end of treatment)
Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit
Part 2: Change from Baseline: Patient Global Impression Scales of Symptoms by Visit Patient-reported health status over the past week where Baseline= Part 1 Period 1 Pre-dose, administered at Week 12 for Period 1 and Period 2. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. Change from baseline: Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Part 1: Part 1: Clinician Global Impression
Clinician Global Impression Scale by visit: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. End of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Measured: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: CGI Symptom Scale
Change from Baseline: CGI Symptom Scale by visit where Baseline= Part 1 Period 1 Pre-dose. Clinician Global Impression Scale: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Change from baseline: higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Measured: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2017
- Study start5 Jul 2017
- Primary completion5 Oct 2018
- Study completion11 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Barth SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 28 weeks, double-blinded crossover followed by a 168-week open label long term safety \& tolerability trial.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hilary Vernon, MD, PhD, Principal investigator, McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03098797
- Sponsor's protocol ID
- SPIBA-201
- Acronym
- TAZPOWER
- Status last verified
- Mar 2024
- First submitted
- 21 Mar 2017
- Last updated
- 16 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C. Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study. Orphanet J Rare Dis. 2025 Apr 25;20(1):199. doi: 10.1186/s13023-025-03693-5.
PubMed 40281531Thompson WR, Manuel R, Abbruscato A, Carr J, Campbell J, Hornby B, Vaz FM, Vernon HJ. Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER. Genet Med. 2024 Jul;26(7):101138. doi: 10.1016/j.gim.2024.101138. Epub 2024 Apr 8.
PubMed 38602181Kim AY, Vernon H, Manuel R, Almuqbil M, Hornby B. Quality of life in Barth syndrome. Ther Adv Rare Dis. 2022 Jun 11;3:26330040221093743. doi: 10.1177/26330040221093743. eCollection 2022 Jan-Dec.
PubMed 37180415
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety and Efficacy of Subcutaneous Injections of Elamipretide in Subjects With Genetically Confirmed Barth Syndrome Followed by Open-Label Treatment
- ClinicalTrials.gov ID
- NCT03098797
- Acronym / sponsor ID
- TAZPOWER / SPIBA-201
- Conditions
- Barth Syndrome
- Keywords
- Barth Syndrome, elamipretide, MTP-131, Stealth, Stealth BT, BTHS
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jul 2017 (actual)
- Primary completion
- 5 Oct 2018 (actual)
- Study completion
- 11 Oct 2021 (actual)
- First posted
- 4 Apr 2017 (actual)
- Results
- Posted 16 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.
Detailed description
A phase 2 randomized, double-blind, placebo-controlled crossover trial to evaluate the safety, tolerability, and efficacy of subcutaneous injections of elamipretide in subjects with genetically confirmed Barth syndrome followed by open-label treatment extension. Part 1 was a randomized, double-blind, placebo-controlled, crossover trial to assess safety, tolerability, and efficacy single daily subcutaneous (SC) doses of 40 mg elamipretide administered for 12 weeks in subjects with Barth syndrome. Part 2: This was an open-label extension trial to assess the long-term safety, tolerability, and longitudinal trends in efficacy single daily SC doses of 40 mg elamipretide for up to 192 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 28 weeks, double-blinded crossover followed by a 168-week open label long term safety \& tolerability trial.
| Arm | Type | Receives |
|---|---|---|
| Elamipretide, then Placebo | Experimental | Drug: Elamipretide, Drug: PlaceboExperimental Part 1: 12 weeks of single daily SC doses of 40 mg elamipretide in Treatment Period 1 followed by 12 weeks of treatment with placebo in Treatment Period 2 (separated by a 4 week washout period). Experimental Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks |
| Placebo , then Elamipretide | Placebo comparator | Drug: Elamipretide, Drug: PlaceboPlacebo Comparator Part 1: 12 weeks of single daily SC doses of placebo in Treatment Period 1 followed by 12 weeks of treatment with 40 mg elamipretide in Treatment Period 2 (separated by a 4 week washout period). Placebo Comparator Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Genetically confirmed Barth Syndrome
- Male aged 12 and above
- At the screening visit, eGFR must meet the following:
- Ambulatory and impaired during the baseline 6MWT
- On stable medication for 30 days prior to the baseline visit
Key exclusion criteria (11 listed)
- Participated in another interventional clinical trial within 30 days of or is currently enrolled in a non-interventional clinical trial at the baseline visit potentially confounding with this trial
- Prior or current medical condition that would prevent the subject from safely participating in the trial
- Undergone any inpatient hospitalizations within 30 days of the baseline visit
- Is undergoing an apparent pubertal growth spurt
- Has uncontrolled hypertension
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ElamipretideAlso: MTP-13140 mg daily subcutaneous injection for 12 weeks | Drug | Elamipretide, then Placebo, Placebo , then Elamipretide |
| PlaceboAlso: Placebo Comparatordaily subcutaneous injection for 12 weeks | Drug | Elamipretide, then Placebo, Placebo , then Elamipretide |
Primary outcome
Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit
Time frame: Pre-dose to Week 12 (end of treatment)
Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.
Time frame: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)
Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12)
12 secondary outcomes
Part 2: Distance Walked During the 6MWT
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Part 1: Muscle Strength as Measured by HHD by Visit
Time frame: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Part 1: 5XSST by Visit
Time frame: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: 5X Sit to Stand by Visit
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Part 1: SWAY Application Balance Assessments by Visit
Time frame: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: SWAY Application Balance Assessment
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Part 1: Patient Global Impression Scales of Symptoms
Time frame: Pre-dose, Week 1, Week 12 (end of treatment)
Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Part 1: Part 1: Clinician Global Impression
Time frame: Pre-dose to Week 12 (end of treatment)
Part 2: Change From Baseline: CGI Symptom Scale
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40281531Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C. Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study. Orphanet J Rare Dis. 2025 Apr 25;20(1):199. doi: 10.1186/s13023-025-03693-5.
- Derived from this study: PubMed 38602181Thompson WR, Manuel R, Abbruscato A, Carr J, Campbell J, Hornby B, Vaz FM, Vernon HJ. Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER. Genet Med. 2024 Jul;26(7):101138. doi: 10.1016/j.gim.2024.101138. Epub 2024 Apr 8.
- Derived from this study: PubMed 37180415Kim AY, Vernon H, Manuel R, Almuqbil M, Hornby B. Quality of life in Barth syndrome. Ther Adv Rare Dis. 2022 Jun 11;3:26330040221093743. doi: 10.1177/26330040221093743. eCollection 2022 Jan-Dec.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hilary Vernon, MD, PhD principal investigator
McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Elamipretide in people with Barth Syndrome, run by Stealth BioTherapeutics Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit (time frame: Pre-dose to Week 12 (end of treatment)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03098797, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2024.