DDrare: Database of Drug Development for Rare Diseases

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 13 valent conjugated pneumococcal vaccine; 150 mg GSK1265744B; 300mg Gemcabene; 3TC; 600mg Gemcabene; ABC; ACETYL- L- CARNITINE; AKCEA-ANGPTL3-LRX; ATV; AZT; AZT/3TC; Abacavir; Abacavir 300mg twice daily; Abacavir sulfate; Abacavir sulfate, Lamivudine and Zidovudine; Acetate; Acetylcarnitine; Acipimox; Amprenavir; Antiretroviral/Anti HIV; Aramchol; Atazanavir; Atazanavir (ATV) + ritonavir (RTV), continuation of backbone 2 nucleoside reverse transcriptase inhibitor (NRTIs); Atazanavir (Reyataz); Atazanavir/Ritonavir; Atorvastatin; Atorvastatin calcium; Avandia administration for 6-12 weeks; BMS-232632; Baricitinib; Behavioral: 60-minutes of steady state exercise; Behavioral: Diet; Behavioral: Exercise; Behavioral: Exercise Training; Behavioral: Exercise training; CD; CD-4 guided therapy interruption; COMBIVIR; Caffeine; Calcium; Carnitine; Cholic Acid; CoFactor; Cofactor supplementation (thiamine, riboflavin, L-carnitine); Combivir (zidovudine [AZT] / lamivudine [3TC]); Continuation of current HAART (boosted protease inhibitor [PI] combination + 2 NRTIs); Cyclophosphamide; DIOSMIN; Device: Venus Versa; Device: poly-L-lactic acid; Didanosine; Different HAART regimens; Diosmin; Diosminex, 500 mg, tabletki powlekane; Dolutegravir; Dolutegravir 50 MG; Efavirenz; Efavirenz (EFV); Emtricitabine; Emtricitabine/tenofovir; Enfuvirtide; Enfuvirtide (T20); Fenofibrate; GSK1265744B (sodium salt) containing 14C-GSK1265744B; Gemcabene; Genetic: Amplification by PCR and direct sequencing on the entire coding sequence and intron-exons boundaries of the candidate gene; Genotropin (human recombinant Growth hormone); Glycerine; Growth Hormone Releasing Hormone; Growth hormone; High-carbohdyrate breakfast; High-dose REGN4461; Hu Leptin (A-100); Human growth hormone; Human recombinant leptin (metreleptin); ISIS 304801; Indinavir; Kaletra (lopinavir [LPVr]); L-Carnitine; L-Lactic acid; Lactic acid; Lamivudine; Lamivudine/Zidovudine; Leptin; Lopinavir; Lopinavir/Ritonavir; Lopinavir/ritonavir + nevirapine; Lopinavir/ritonavir + zidovudine + lamivudine; Low-Dose REGN4461; METRELEPTIN; Mecasermin; Metformin; Metformin hydrochloride; Metreleptin; Metrelpetin; Monotherapy (Lopinavir/ritonavir); Monotherapy (Lopinavir/ritonavir) + ABC/3TC; Myalept; Nelfinavir; Nelfinavir mesylate; Nevirapine; Niacin; Non-nucleoside reverse transcriptase inhibitors; Norvir; NucleomaxX; Nucleoside analogue sparing HAART regimen; Nucleoside reverse transcriptase inhibitors; Obeticholic Acid; Obeticholic acid; Other: Glycerine gel; Other: Observation; POLY-L-LACTIC ACID; Perform blood cells and fibroblasts biochemical and immuno-labeled investigations; Pioglitazone; Pioglitazone or metformin; Pneumococcal polysaccharide vaccine 23 valent; Pravastatin; Pravastatin sodium; Prevenar13; Procedure: testosterone injection; Protease inhibitor; Protease inhibitors; R-metHuLeptin; REYATAZ 150MG 60 CPS BL; RTV; Raltegravir; Recombinant human growth hormone; Recombinant human growth hormone + rosiglitazone; Reyataz; Riboflavin; Ritonavir; Ritonavir (Norvir); Rosiglitazone; Rosiglitazone maleate; ST200; SUSTIVA; Saquinavir; Serostim®[somatropin (rDNA origin) for injection]; Setmelanotide; Simvastatin; Somatropin; Standard continuous antiretroviral therapy; Stavudine; Sulfate; TAT4 Gel concentration A; TAT4 Gel concentration B; TH9507; TRUVADA; Tenofovir; Tenofovir DF; Tenofovir Disoproxil Fumarate; Tenofovir disoproxil; Tesamorelin; Tesamorelin (Egrifta); Testosterone; Thiamine; Tipranavir; Total Dietary Replacement; Use of lipid-lowering drugs.; VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide; Viral load driven treatment interruption; Volanesorsen; Zidovudine; [N-trans-3-Hexenoyl] Human Growth Hormone Releasing Factor (1-44) Acetate

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2015-000493-35-DE (EUCTR)	17/02/2016	14/08/2015	Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy	A Randomized, Double-Blind, Placebo-Controlled, with an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Familial Partial Lipodystrophy - The BROADEN Study	Partial lipodystrophy MedDRA version: 20.0;Level: PT;Classification code 10053857;Term: Partial lipodystrophy;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide Product Code: ISIS 304801 INN or Proposed INN: ISIS 304801 Other descriptive name: ISIS 304801	Akcea Therapeutics, Inc.	NULL	Not Recruiting	Female: yes Male: yes	60	Phase 3	United States;Portugal;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Germany;Netherlands
2	EUCTR2015-000493-35-NL (EUCTR)	15/12/2015	30/10/2015	Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy	A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Partial Lipodystrophy - The BROADEN Study	Partial lipodystrophy MedDRA version: 18.1;Level: PT;Classification code 10053857;Term: Partial lipodystrophy;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide Product Code: ISIS 304801 INN or Proposed INN: ISIS 304801 Other descriptive name: ISIS 304801	Isis Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	125	Phase 2;Phase 3	Portugal;United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Germany
3	EUCTR2015-000493-35-ES (EUCTR)	04/12/2015	15/10/2015	Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy	A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Partial Lipodystrophy - The BROADEN Study	Partial lipodystrophy MedDRA version: 18.1;Level: PT;Classification code 10053857;Term: Partial lipodystrophy;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide Product Code: ISIS 304801 INN or Proposed INN: ISIS 304801 Other descriptive name: ISIS 304801	Isis Pharmaceuticals, Inc.	NULL	Not Recruiting	Female: yes Male: yes	125	Phase 2;Phase 3	Portugal;United States;Greece;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Germany;Netherlands

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2015-000493-35-DE
(EUCTR)

17/02/2016

14/08/2015

Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy

A Randomized, Double-Blind, Placebo-Controlled, with an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Familial Partial Lipodystrophy - The BROADEN Study

Partial lipodystrophy
MedDRA version: 20.0;Level: PT;Classification code 10053857;Term: Partial lipodystrophy;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide
Product Code: ISIS 304801
INN or Proposed INN: ISIS 304801
Other descriptive name: ISIS 304801

Akcea Therapeutics, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Portugal;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Germany;Netherlands

EUCTR2015-000493-35-NL
(EUCTR)

15/12/2015

30/10/2015

Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy

A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Partial Lipodystrophy - The BROADEN Study

Partial lipodystrophy
MedDRA version: 18.1;Level: PT;Classification code 10053857;Term: Partial lipodystrophy;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide
Product Code: ISIS 304801
INN or Proposed INN: ISIS 304801
Other descriptive name: ISIS 304801

Isis Pharmaceuticals, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

125

Phase 2;Phase 3

Portugal;United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Germany

EUCTR2015-000493-35-ES
(EUCTR)

04/12/2015

15/10/2015

Study of ISIS 304801 versus placebo Administered Subcutaneously to Patients with Partial Lipodystrophy

A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients with Partial Lipodystrophy - The BROADEN Study

Product Name: VOLANESORSEN SODIUM, ApoC-III Antisense Oligonucleotide
Product Code: ISIS 304801
INN or Proposed INN: ISIS 304801
Other descriptive name: ISIS 304801

Isis Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

125

Phase 2;Phase 3

Portugal;United States;Greece;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Germany;Netherlands